| Primary | Percentage of Participants Who Achieved Complete Remission (CR) With a Corticosteroids Dose of Less Than or Equal to (<=) 10 mg/Day From Week 29 to Week 37: Pemphigus Vulgaris Participants in Modified Intent-to-Treat (PV mITT) Population | Complete remission was defined as absence of new and established lesions and was intended to mean "no disease activity" for 2 consecutive weeks, with Pemphigus Disease Area Index (PDAI) activity score =0 and CS dose <=10 mg. PDAI score weighted for number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). PDAI total score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. In this outcome measure (OM), percentage of participants who achieved CR while on a daily corticosteroids dose of <=10 mg/day were reported. | Analysis was performed on PV mITT which included randomized PV participants who had received at least one dose of study medication and were analyzed according to the treatment they were allocated to at randomization. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
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| P-value and 95% confidence interval (CI) were based on Cochran-Mantel-Haenszel general association test stratified by disease type (PV or PF) and disease history (newly diagnosed [<=6 months prior to screening] or relapsing [diagnosed greater than [>] 6 months prior to screening]). | Cochran-Mantel-Haenszel | | 0.4469 | | Difference in percentage | 5.73 | Standard Error of the Mean | 7.535 | 2-Sided | 95 | -9.037 | 20.500 | | | | | Superiority | | |
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| Primary | Percentage of Participants Who Achieved Complete Remission With a Corticosteroids Dose of <=10 mg/Day From Week 29 to Week 37: Modified Intent-to-Treat (mITT) Population | Complete remission was defined as absence of new and established lesions and was intended to mean "no disease activity" for 2 consecutive weeks, with PDAI activity score =0 and CS dose <=10 mg. PDAI score weighted for number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). PDAI total score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. In this OM, percentage of participants who achieved CR while on a daily corticosteroids dose of <=10 mg/day were reported. | Analysis was performed on mITT population which included all randomized participants (i.e. PV + PF participants) who had received at least one dose of study medication and were analyzed according to the treatment they were allocated to at randomization. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Oral Corticosteroid Dose From Baseline to Week 37: PV mITT Population | The cumulative dose (in milligrams) of sponsor-provided oral CS (prednisone or prednisolone) received by the participants during the BT period was calculated from Baseline to Week 37 and is reported in this OM. | Analysis was performed on PV mITT population. | Posted | | Mean | Standard Deviation | milligrams | | Baseline to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Oral Corticosteroid Dose From Baseline to Week 37: mITT Population | The cumulative dose (in milligrams) of sponsor-provided oral CS (prednisone or prednisolone) received by the participants during the BT period was calculated from Baseline to Week 37 and is reported in this OM. | Analysis was performed on mITT population. | Posted | | Mean | Standard Deviation | milligrams | | Baseline to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Week 37: PV mITT Population | Cumulative duration (in days) of CR post first CS dose of <= 10 mg/day from Baseline to Week 37 was analyzed with a zero-inflated negative binomial model with terms of treatment group and disease history, and an offset based on the number of days on blinded treatment period and were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on PV mITT population. | Posted | | Mean | Standard Deviation | days | | Baseline to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Week 37: mITT Population | Cumulative duration (in days) of CR post first CS dose of <= 10 mg/day from Baseline to Week 37 was analyzed with a zero-inflated negative binomial model with terms of treatment group and disease history, and an offset based on the number of days on blinded treatment period and were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on mITT population. | Posted | | Mean | Standard Deviation | days | | Baseline to Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Time to First Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Week 37: PV mITT Population | Time to first CR was the time (in days) to achieve CR while on CS dose of <=10 mg/day. Complete remission was defined as the absence of new and established lesions to the "no disease activity". Kaplan-Meier method was used for the analysis. | Analysis was performed on PV mITT population. | Posted | | Median | 95% Confidence Interval | days | | Baseline to Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Time to First Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Week 37: mITT Population | Time to first CR was the time (in days) to achieve CR while on CS dose of <=10 mg/day. Complete remission was defined as the absence of new and established lesions to the "no disease activity". Kaplan-Meier method was used for the analysis. | Analysis was performed on mITT population. | Posted | | Median | 95% Confidence Interval | days | | Baseline to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Percentage of Participants Who Achieved Complete Remission With a Corticosteroids Dose of <=5 mg/Day From Week 29 to Week 37: PV mITT Population | Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". In this OM, percentage of participants who achieved CR while on a daily corticosteroids dose of <=5 mg/day were reported. | Analysis was performed on PV mITT population. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Percentage of Participants Who Achieved Complete Remission With a Corticosteroids Dose of <=5 mg/Day From Week 29 to Week 37: mITT Population | Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". In this OM, percentage of participants who achieved CR while on a daily corticosteroids dose of <=5 mg/day were reported. | Analysis was performed on mITT population. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Percentage of Participants Who Had Pemphigus Disease Area Index (PDAI) Score <3 With a Corticosteroids Dose <=10 mg/Day From Week 29 to Week 37: PV mITT Population | PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Thus, PDAI total activity score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. In this OM, participants with PDAI score <3 while on a CS dose of <=10 mg/day were reported. | Analysis was performed on PV mITT population. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
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| Secondary | Percentage of Participants Who Had Pemphigus Disease Area Index Score <3 With a Corticosteroids Dose <=10 mg/Day From Week 29 to Week 37: mITT Population | PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Thus, PDAI total activity score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. In this OM, participants with PDAI score <3 while on a CS dose of <=10 mg/day were reported. | Analysis was performed on mITT population. | Posted | | Number | | percentage of participants | | From Week 29 to Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Weeks 61 and 109: PV mITT Population | Cumulative duration (in days) of CR post all doses of CS <=10 mg/day during Baseline to Week 61 and Baseline to Week 109 were reported in this OM. Complete remission was defined as the absence of new and established lesions to the "no disease activity". | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. | Posted | | Mean | Standard Deviation | days | | Baseline to Week 61 and Baseline to Week 109 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Weeks 61 and 109: mITT Population | Cumulative duration (in days) of CR post all doses of CS <=10 mg/day during Baseline to Week 61 and Baseline to Week 109 were reported in this OM. Complete remission was defined as the absence of new and established lesions to the "no disease activity". | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. | Posted | | Mean | Standard Deviation | days | | Baseline to Weeks 61 and Baseline to Week 109 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose = 0 mg/Day From Baseline to Weeks 61 and 109: PV mITT Population | Cumulative duration (in days) of CR post all doses of CS =0 mg/day during Baseline to Week 61 and Baseline to Week 109 were reported in this OM. Complete remission was defined as the absence of new and established lesions to the "no disease activity". | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. | Posted | | Mean | Standard Deviation | days | | Baseline to Week 61 and Baseline to Week 109 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose = 0 mg/Day From Baseline to Weeks 61 and 109: mITT Population | Cumulative duration (in days) of CR post all doses of CS dose = 0 mg/day during Baseline to Week 61 and Baseline to Week 109 were reported in this OM. Complete remission was defined as the absence of new and established lesions to the "no disease activity". | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. | Posted | | Mean | Standard Deviation | days | | Baseline to Week 61 and Baseline to Week 109 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Glucocorticoid Toxicity Index (GTI) - Cumulative Worsening Score (CWS) and Aggregate Improvement Score (AIS) at Week 37: PV mITT Population | GTI assessed glucocorticoid (GC) related morbidity and GC-sparing ability of other therapies; composed of 2 components: Composite GTI and specific list. Composite GTI had 9 domains and specific list had 23 items (11 domains), used as complementary tool to C-GTI. Composite GTI score:sum of 9 domain-specific scores at each visit and cumulative GTI score:sum of composite GTI scores across each visit. 2 cumulative GTI scores: CWS and AIS at Week 37 reported in this OM. CWS assessed cumulative GC toxicity regardless of whether toxicity had lasting effects/was transient. AIS assessed new therapy effectiveness in decreasing any Baseline GC toxicity over time. CWS composite score range: 0 to 439 and AIS composite score range: -346 to 439. In CWS and AIS, minimum score=least toxicity and maximum score=most toxicity. Least square (LS) mean and standard error (SE) from analysis of covariance model using treatment, disease history as fixed effects and Baseline GTI score and CS dose as covariates. | Analysis was performed on PV mITT population. | Posted | | Least Squares Mean | Standard Error | units on a scale | | At Week 37 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
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| Secondary | Glucocorticoid Toxicity Index - Cumulative Worsening Score and Aggregate Improvement Score at Week 37: mITT Population | GTI assessed GC related morbidity and GC-sparing ability of other therapies; composed of 2 components: Composite GTI and specific list. Composite GTI contained 9 domains and specific list contained of 23 items (11 domains), used as complementary tool to C-GTI. Composite GTI score: sum of 9 domain-specific scores at each visit and cumulative GTI score: sum of composite GTI scores across each visit. Two cumulative GTI scores: CWS and AIS at Week 37 were reported in this OM. CWS assessed cumulative GC toxicity regardless of whether toxicity had lasting effects/was transient. AIS assessed new therapy effectiveness in decreasing any Baseline GC toxicity over time. CWS composite score ranged from 0 to 439 and AIS composite score ranged from -346 to 439. For CWS and AIS, minimum score = least toxicity and maximum score = most toxicity. LS mean and SE from analysis of covariance model using treatment, disease history as fixed effects and Baseline GTI score and CS dose as covariates. | Analysis was performed on mITT population. | Posted | | Least Squares Mean | Standard Error | units on a scale | | At Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Change From Baseline in Pemphigus Disease Area Index Score at Weeks 5, 13, 25, 37, 61, and 109: PV mITT Population | PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Thus, PDAI total activity score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. PDAI activity score was measured independently of the CS dose administered in the participants. | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | units on a scale | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Change From Baseline in Pemphigus Disease Area Index Score at Weeks 5, 13, 25, 37, 61, and 109: mITT Population | PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Thus, PDAI total activity score ranged from 0 to 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity). Higher score indicated more disease activity. PDAI activity score was measured independently of the CS dose administered in the participants. | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | units on a scale | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Change From Baseline in Autoimmune Bullous Disease Quality of Life (ABQOL) Score at Weeks 5, 13, 25, 37, 61, and 109: PV mITT Population | ABQOL, a 17-item questionnaire is a valid and reliable tool to assess quality of life in participants with autoimmune blistering diseases. Each question score ranged from 0 to 3 points, 0 = never, 1 = occasionally, 2 = sometimes and 3 = all the time, where higher score = poor QOL. The total ABQoL score was calculated by summing the score of each question and it ranged from 0 to 51, where higher score = more QOL impairment. | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'overall number of participants analyzed'=participants evaluable for this outcome measure and 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
|
| Secondary | Change From Baseline in Autoimmune Bullous Disease Quality of Life Score at Weeks 5, 13, 25, 37, 61, and 109: mITT Population | ABQOL, a 17-item questionnaire is a valid and reliable tool to assess quality of life in participants with autoimmune blistering diseases. Each question score ranged from 0 to 3 points, 0 = never, 1 = occasionally, 2 = sometimes and 3 = all the time, where higher score = poor QOL. The total ABQoL score was calculated by summing the score of each question and it ranged from 0 to 51, where higher score = more QOL impairment. | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'overall number of participants analyzed'=participants evaluable for this outcome measure and 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
|
| Secondary | Percentage of Participants With Autoimmune Bullous Disease Quality of Life Score of Zero at Weeks 5, 13, 25, 37, 61, and 109: PV mITT Population | ABQOL, a 17-item questionnaire is a valid and reliable tool to assess quality of life in participants with autoimmune blistering diseases. Each question score ranged from 0 to 3 points, 0 = never, 1 = occasionally, 2 = sometimes and 3 = all the time, where higher score = poor QOL. The total ABQoL score was calculated by summing the score of each question and it ranged from 0 to 51, where higher score = more QOL impairment. Percentage of participants with ABQOL score of '0' were reported in this outcome measure. | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Number | | percentage of participants | | At Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
|
| Secondary | Percentage of Participants With Autoimmune Bullous Disease Quality of Life Score of Zero at Weeks 5, 13, 25, 37, 61, and 109: mITT Population | ABQOL, a 17-item questionnaire is a valid and reliable tool to assess quality of life in participants with autoimmune blistering diseases. Each question score ranged from 0 to 3 points, 0 = never, 1 = occasionally, 2 = sometimes and 3 = all the time, where higher score = poor QOL. The total ABQoL score was calculated by summing the score of each question and it ranged from 0 to 51, where higher score = more QOL impairment. Percentage of participants with ABQOL score of '0' were reported in this outcome measure. | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Number | | percentage of participants | | At Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
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| Secondary | Change From Baseline in the European Quality of Life Working Group Health Status Measure 5 Dimensions, 5 Levels (EQ-5D-5L) Score: Visual Analogue Scale (VAS) Scores at Weeks 5, 13, 25, 37, 61, and 109 | EQ-5D-5L: standardized measure of health status that provides general assessment of health and wellbeing. EQ-5D VAS was used to record a participant's rating for his/her current health-related quality of life state and captured on a vertical VAS scale ranging from 0 to 100, where 0=worst imaginable health state and 100=best imaginable health state, where higher states indicated better outcomes. | For this OM, the planned data collection and analysis was not performed because of early termination of the study. | Posted | | | | | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | |
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| Secondary | Change From Baseline in the European Quality of Life Working Group Health Status Measure 5 Dimensions, 5 Levels Score: Individual Dimension at Weeks 5, 13, 25, 37, 61, and 109 | EQ-5D-5L: standardized measure of health status that provides general assessment of health and wellbeing. The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has a 5-level response: no problems, slight problems, moderate problems, severe problems, and extreme problems. Response options are measured with a 5-point Likert scale (for the 5L version). The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. | For this OM, the planned data collection and analysis was not performed because of early termination of the study. | Posted | | | | | | Baseline, Weeks 5, 13, 25, 37, 61, and 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | |
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| Secondary | Time to First Complete Remission With a Corticosteroids Dose <=10 mg/Day From Baseline to Weeks 61 and 109 | Time to first CR was the time (in days) to achieve CR while on a CS dose of <=10 mg/day from Baseline to Week 61 and from Baseline to Week 109. Complete remission was defined as absence of new and established lesions to the "no disease activity". | For this OM, the planned data collection and analysis was not performed because of early termination of the study. | Posted | | | | | | Baseline to Week 61 and Baseline to Week 109 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Total Number of Disease Relapses/Flares From Initial Control of Disease Activity (CDA) to Week 37: PV mITT Population | Total Number of Disease Relapses/Flares which occurred from initial CDA to Week 37 were reported. CDA was defined as disappearance of new lesions cease to form and established lesions begin to heal. Relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. | Analysis was performed on PV mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | relapses | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib |
|
| Secondary | Total Number of Disease Relapses/Flares From Initial Control of Disease Activity to Week 37: mITT Population | Total Number of Disease Relapses/Flares which occurred from initial CDA to Week 37 were reported. CDA was defined as disappearance of new lesions cease to form and established lesions begin to heal. Relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | relapses | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib |
|
| Secondary | Time to Initial Relapse/Flare From Initial Control of Disease Activity to Week 37: PV mITT Population | Time duration (in days) from the time of initial relapse/flare which occurred from initial CDA up to Week 37 were reported in this OM. CDA was defined as the visit at which new lesions cease to form and established lesions begin to heal. Relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. Kaplan-Meier method was used for the analysis. | Analysis was performed on PV mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Median | 95% Confidence Interval | days | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 |
|
| Secondary | Time to Initial Relapse/Flare From Initial Control of Disease Activity to Week 37: mITT Population | Time duration (in days) from the time of initial relapse/flare which occurred from initial CDA up to Week 37 were reported in this OM. CDA was defined as the visit at which new lesions cease to form and established lesions begin to heal. Relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. Kaplan-Meier method was used for the analysis. | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Median | 95% Confidence Interval | days | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 |
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| Secondary | Percentage of Participants With 3 or More New Lesions Within 1 Month That do Not Heal Spontaneously Within 1 Week, or With Extension of Established Lesions at Week 37: PV mITT Population | Percentage of Participants With 3 or More New Lesions Within past 1 month that do not heal spontaneously within past 1 week, or with extension of established lesions, were assessed at Week 37 and reported in this OM. | Analysis was performed on PV mITT population. | Posted | | Number | | percentage of participants | | At Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Percentage of Participants With 3 or More New Lesions Within 1 Month That do Not Heal Spontaneously Within 1 Week, or With Extension of Established Lesions at Week 37: mITT Population | Percentage of participants with 3 or more new lesions within past 1 month that do not heal spontaneously within past 1 week, or with extension of established lesions, were assessed at Week 37 and reported in this OM. | Analysis was performed on mITT population. | Posted | | Number | | percentage of participants | | At Week 37 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Percentage of Participants With at Least One Disease Relapse/Flare From Initial Control of Disease Activity to Week 37: PV mITT Population | CDA was defined as the visit at which new lesions cease to form and established lesions begin to heal. Disease relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. Percentages were calculated based on number of participants assessed (i.e. participants achieved CDA) in each treatment group. Percentage of participants with at least one disease relapse/flare from Initial CDA to Week 37 were reported in this OM. | Analysis was performed on PV mITT population. Here, 'overall number of participants analyzed' = participants evaluable for this outcome measure. | Posted | | Number | | percentage of participants | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Percentage of Participants With at Least One Disease Relapse/Flare From Initial Control of Disease Activity to Week 37: mITT Population | CDA was defined as the visit at which new lesions cease to form and established lesions begin to heal. Disease relapse/flare was defined by the appearance of 3 or more new lesions after CDA and within a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who had achieved CDA. Percentages were calculated based on number of participants assessed (i.e. participants achieved CDA) in each treatment group. Percentage of participants with at least one disease relapse/flare from Initial CDA to Week 37 were reported in this OM. | Analysis was performed on mITT population. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Number | | percentage of participants | | From initial CDA up to Week 37 (i.e., during any time from Baseline up to Week 37) | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Week 37 to Week 61: PV mITT Population | Cumulative duration (in days) of CR post first dose of CS <=10 mg/day from Week 37 to Week 61 were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | days | | From Week 37 to Week 61 | | | | ID | Title | Description |
|---|
| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
|
| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose <=10 mg/Day From Week 37 to Week 61: mITT Population | Cumulative duration (in days) of CR post first dose of CS <=10 mg/day from Week 37 to Week 61 were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | days | | From Week 37 to Week 61 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose = 0 mg/Day From Week 37 to Week 61: PV mITT Population | Cumulative duration (in days) of CR post first dose of CS = 0 mg/day from Week 37 to Week 61 were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on PV mITT population, based on data collected up to database lock for the blinded treatment period. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | days | | From Week 37 to Week 61 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Cumulative Duration of Complete Remission With a Corticosteroids Dose = 0 mg/Day From Week 37 to Week 61: mITT Population | Cumulative duration (in days) of CR post first dose of CS = 0 mg/day from Week 37 to Week 61 were reported in this OM. Complete remission was defined as the absence of new and established lesions and was intended to mean "no disease activity". | Analysis was performed on mITT population, based on data collected up to database lock for the blinded treatment period. Here, overall number of participants analyzed = participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | days | | From Week 37 to Week 61 | | | | ID | Title | Description |
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| OG000 | Placebo Then Rilzabrutinib | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 in OLE period and those who were eligible after OLE period completion, continued the same treatment until Week109 in LTE period according to protocol-specified criteria. | | OG001 | Rilzabrutinib Then Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious after the administration of first dose of study drug in each period. | Analysis was performed on safety population which included all participants who had received at least one dose of study medication and were analyzed according to the treatment they actually received. | Posted | | Count of Participants | | Participants | | BT Period: From Day 1 of Week 1 to Week 37, OLE Period: from Week 37 to Week 61 and LTE Period: from Week 61 up to 4 weeks after the last dose at Week 109 (i.e., up to Week 113) | | | | ID | Title | Description |
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| OG000 | BT Period: Placebo | Participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. | | OG001 |
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| Secondary | Pharmacokinetics (PK): Plasma Concentration of Rilzabrutinib | Data for this OM was not planned to be collected and analyzed for placebo arm of the study. | Analysis was performed on PK population which included all participants who had received at least one dose of study medication and had sufficient data for PK analysis. | Posted | | Mean | Standard Deviation | nanograms per milliliter | | Pre-dose and 2 hours post-dose on Day 1 | | | | ID | Title | Description |
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| OG000 | Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Pharmacodynamics (PD): Change From Baseline in Antibody Levels - Anti-desmoglein 1 (Anti-dsg 1) and Anti-desmoglein 3 (Anti-dsg 3) at Weeks 13, 25, 37, 49, 61, 73, 85, 97, and 109: PV mITT Population | Anti-dsg1 and anti-dsg3 autoantibody levels was assessed by enzyme-linked immunosorbent assay (ELISA) method. | Analysis was performed on PV mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | units/milliliter | | Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, and 109 | | | | ID | Title | Description |
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| OG000 | Placebo | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. | | OG001 | Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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| Secondary | Pharmacodynamics: Change From Baseline in Antibody Levels - Anti-desmoglein 1 (Anti-dsg 1) and Anti-desmoglein 3 (Anti-dsg 3) at Weeks 13, 25, 37, 49, 61, 73, 85, 97 and 109: mITT Population | Anti-dsg1 and anti-dsg3 autoantibody levels was assessed by ELISA method. | Analysis was performed on mITT population. Here, 'number analyzed' = participants with available data for each specified category. | Posted | | Mean | Standard Deviation | units/milliliter | | Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, and 109 | | | | ID | Title | Description |
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| OG000 | Placebo | In BT period, participants received placebo orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. | | OG001 | Rilzabrutinib | In BT period, participants received rilzabrutinib 400 mg orally BID up to 37 weeks along with sponsor-provided CS. After at least two weeks of CDA (no new lesions and established lesions begin to heal), based on protocol-specified clinical criteria, investigators could decrease the CS dose to a minimum of 5 mg prednisone/prednisolone per day from Week 29 to Week 37. Post completion of BT period, eligible participants received rilzabrutinib 400 mg BID up to Week 61 and those who were eligible after OLE period completion, continued the same treatment until Week 109 in LTE period according to protocol-specified criteria. |
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