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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-002844-66 | EudraCT Number |
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A business decision was made to discontinue further enrollment. There were no safety concerns that contributed to this decision.
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The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
Prospective participants must have primary wAIHA as well as other protocol-defined criteria. After participants have been determined to be eligible for the study, they will be randomized to 2:1, with stratification factor of corticosteroid dose and hemoglobin (Hgb <9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments in the double-blind period, the participant will have the opportunity to receive parsaclisib in the open-label treatment which will last up to another 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group A: Parsaclisib | Experimental | Participants will receive parsaclisib for 24 weeks (double-blind period). Participant who completed the double-blind period and tolerating the study treatment upon investigator's opinion will continue into open-label period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period. |
|
| Group B: Placebo followed by Parsaclisib | Placebo Comparator | Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive parsaclisib in the 24 week open-label period. Participants may then continue to receive parsaclisib in a long-term extension period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| parsaclisinib | Drug | parsaclisib will be administered QD orally |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Attaining a Durable Hemoglobin Response | A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period. | up to Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24 | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kathleen Butler, MD | Incyte Corporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site US005 | Los Angeles | California | 90089 | United States | ||
| Investigative Site US004 |
Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency
Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement
This study was designed to evaluate parsaclisib 2.5 mg QD compared with placebo over a 24-week double-blind treatment period followed by a 24-week open-label treatment period with parsaclisib. Participants could then continue to receive parsaclisib in a long-term extension period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| 24-week Double-blind Period |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 10, 2023 | Oct 9, 2024 |
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Study will be a 24 week double-blind period followed by a 24 week open-label period, followed by a long term extension period.
| placebo | Drug | placebo will be administered QD orally follwed by Parsaclisinib in the open label period |
|
| Baseline; Week 24 |
| Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT) | The 6MWT is used to evaluate submaximal exercise capacity. It is a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes. | Baseline; Week 24 |
| Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 |
| Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 |
| Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 |
| Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 |
| Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Transfusion was permitted as a rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of warm autoimmune hemolytic anemia (wAIHA). | Week 6 to Week 24; Week 24 to Week 48 |
| Change From Baseline in Daily Corticosteroid Dose at Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; Week 24 |
| Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. | Baseline; Week 24 |
| Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. | Week 6 to Week 24; Week 24 to Week 48 |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. | up to 446 days |
| Number of Participants With Any ≥Grade 3 TEAE | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. The severity of AEs were assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal. | up to 446 days |
| Whittier |
| California |
| 90603 |
| United States |
| Investigative Site US006 | Miami | Florida | 33165 | United States |
| Investigative Site US012 | Indianapolis | Indiana | 46260 | United States |
| Investigative Site US007 | The Bronx | New York | 10461 | United States |
| Investigative Site US002 | The Bronx | New York | 10467 | United States |
| Investigative Site US003 | Greenville | North Carolina | 27858 | United States |
| Investigative Site US009 | Canton | Ohio | 44718 | United States |
| Investigative Site US010 | Easton | Pennsylvania | 18045 | United States |
| Investigative Site US001 | Knoxville | Tennessee | 37920 | United States |
| Investigative Site AT002 | Salzburg CET | A-5020 | Austria |
| Investigative Site AT001 | Vienna | 01090 | Austria |
| Investigative Site BE001 | La Louvière | 07100 | Belgium |
| Investigative Site BE002 | Liège | 04000 | Belgium |
| Investigative Site CA001 | Edmonton | Alberta | T6G 2P4 | Canada |
| Investigative Site FR002 | Lille | 59037 | France |
| Investigative Site FR003 | Marseille | 13285 | France |
| Investigative Site FR001 | Paris | 75015 | France |
| Investigative Site DE001 | Essen | 45147 | Germany |
| Investigative Site DE002 | Ulm | 89081 | Germany |
| Investigative Site IL002 | Haifa | 31096 | Israel |
| Investigative Site IL001 | Nahariya | 2210001 | Israel |
| Investigative Site IT003 | Florence | 50134 | Italy |
| Investigative Site IT002 | Milan | 20122 | Italy |
| Investigative Site IT001 | Novara | 28100 | Italy |
| Investigative Site IT004 | Pavia | 27100 | Italy |
| Investigative Site IT006 | Rome | 00168 | Italy |
| Investigative Site IT005 | Trieste | 34125 | Italy |
| Investigative Site JP008 | Fukuoka | 807-8556 | Japan |
| Investigative Site JP004 | Isehara | 259-1193 | Japan |
| Investigative Site JP006 | Nagoya | 467-8602 | Japan |
| Investigative Site JP009 | Okayama | 700-8557 | Japan |
| Investigative Site JP002 | Okayama | 701-0192 | Japan |
| Investigative Site JP010 | Osakasayama-shi | 589-8511 | Japan |
| Investigative Site JP005 | Saitama | 350-0495 | Japan |
| Investigative Site JP007 | Sendai | 980-8574 | Japan |
| Investigative Site JP001 | Suita-shi | 565-0871 | Japan |
| Investigative Site JP003 | Tokyo | 141-8625 | Japan |
| Investigative Site NL001 | Rotterdam | 3015CA | Netherlands |
| Investigative Site PL001 | Legnica | 59220 | Poland |
| Investigative Site PL006 | Lodz | 93-510 | Poland |
| Investigative Site PL003 | Nowy Sącz | 33-300 | Poland |
| Investigative Site PL005 | Opole | 45-372 | Poland |
| Investigative Site PL004 | Wałbrzych | 58-309 | Poland |
| Investigative Site PL002 | Wroclaw | 53-439 | Poland |
| Investigative Site ES006 | Badalona | 08916 | Spain |
| Investigative Site ES001 | Barcelona | 08036 | Spain |
| Investigative Site ES003 | Madrid | 28006 | Spain |
| Investigative Site ES005 | Murcia | 30008 | Spain |
| Investigative Site ES004 | Tarragona | 43005 | Spain |
| Investigative Site ES002 | Valencia | 46026 | Spain |
| Investigative Site GB002 | Glasgow | G4 0SF | United Kingdom |
| Investigative Site GB006 | London | W12 0HS | United Kingdom |
| Investigative Site GB003 | Norwich | NR4 7UY | United Kingdom |
| Investigative Site GB004 | Plymouth | PL6 8DH | United Kingdom |
| Investigative Site GB005 | Reading | RG1 5AN | United Kingdom |
| FG001 | Placebo Followed by Parsaclisib | Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| 24-week Open-label Period |
|
|
| Long-term Extension Period (~2 Years) |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
| BG001 | Placebo Followed by Parsaclisib | Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Attaining a Durable Hemoglobin Response | A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period. | Safety Analysis Set: all randomized participants who received at least 1 dose of study drug. Treatment groups were determined according to the actual treatment the participant received on Day 1 regardless of assigned study treatment. Only participants with available data were analyzed. | Posted | Number | percentage of participants | up to Week 24 |
|
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| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24 | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | Baseline; Week 24 |
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| Secondary | Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT) | The 6MWT is used to evaluate submaximal exercise capacity. It is a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes. | Safety Analysis Set. Only participants with available data were analyzed, | Posted | Number | percentage of participants | Baseline; Week 24 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | scores on a scale | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 ("not at all") to 4 ("very much"), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. | Safety Analysis Set. Only participants with available data were analyzed, | Posted | Mean | Standard Deviation | percentage change | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | grams per liter | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | percentage change | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 |
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| Secondary | Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Transfusion was permitted as a rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of warm autoimmune hemolytic anemia (wAIHA). | Safety Analysis Set. Only participants with available data were analyzed. The number of participants analyzed at Weeks 6 to 24 reflects the number of participants who received study drug for at least 43 days. The number of participants analyzed at Weeks 24 to 48 reflects the number of participants who received study drug during the open-label period. | Posted | Number | percentage of participants | Week 6 to Week 24; Week 24 to Week 48 |
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| Secondary | Change From Baseline in Daily Corticosteroid Dose at Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | milligrams | Baseline; Week 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Percentage change from Baseline was calculated as ([the post-Baseline value minus the Baseline value]/Baseline value) x 100. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | percentage change | Baseline; Week 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a > 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. | Safety Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | Week 6 to Week 24; Week 24 to Week 48 |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. | Safety Analysis Set | Posted | Count of Participants | Participants | up to 446 days |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Any ≥Grade 3 TEAE | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. The severity of AEs were assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal. | Safety Analysis Set | Posted | Count of Participants | Participants | up to 446 days |
|
up to 446 days
For safety analysis, participants who transitioned from treatment with placebo to treatment with parsaclisib 2.5 milligrams (mg) once daily (QD) in the the open-label treatment period and the long-term extension period have been counted both in the placebo arm and the parsaclisib arm.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24. | 0 | 6 | 0 | 6 | 4 | 6 |
| EG001 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. | 0 | 11 | 6 | 11 | 10 | 11 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cholecystitis | Hepatobiliary disorders | MedDRA 27 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| Immune-mediated enterocolitis | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA 27 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 27 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27 | Systematic Assessment |
| |
| Catarrh | Respiratory, thoracic and mediastinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Choluria | Renal and urinary disorders | MedDRA 27 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 27 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 27 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 27 | Systematic Assessment |
| |
| Embolism | Vascular disorders | MedDRA 27 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Faeces soft | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA 27 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27 | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 27 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 27 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 27 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 27 | Systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA 27 | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 27 | Systematic Assessment |
| |
| Micturition disorder | Renal and urinary disorders | MedDRA 27 | Systematic Assessment |
| |
| Mood altered | Psychiatric disorders | MedDRA 27 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 27 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 27 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 27 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 27 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 27 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 27 | Systematic Assessment |
|
Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Incyte Corporation | 1-855-463-3463 | medinfo@incyte.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 17, 2024 | Oct 9, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C000656179 | parsaclisib |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Missing |
|
|
|
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years.
|
|
| Parsaclisib |
Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|
| OG002 | Parsaclisib | Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. |
|
|