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| Name | Class |
|---|---|
| Seventh Framework Programme | OTHER |
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International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).
The aim of this study is to monitor the long-term safety profile of emapalumab in participants who have previously received at least one dose of emapalumab, including survival time after the administration of emapalumab.
Moreover, the elimination profile of emapalumab and the immunogenicity will also be assessed.
Furthermore, safety, tolerability, efficacy, and pharmacokinetic (PK) profile of emapalumab will be closely monitored in the event that some participants, upon request of the treating physician, will receive emapalumab treatment in the follow-up study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Enrolled-04 Cohort | Other | Participants enrolled in Study NI-0501-04 (NCT01818492) will be invited to participate for long-term follow-up for 1 year either after haematopoietic stem cell transplantation (HSCT) or after the last administration of emapalumab. In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT. Participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT will be delayed for reasons unrelated to the administration of emapalumab, can continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05, NCT02069899) at the request of the investigator, providing a favorable benefit/risk assessment of treatment is established. The dose and timing was either carried forward from the last administered emapalumab dose as part of the parent protocol or an adjusted dose was administered, if necessary. |
|
| Enrolled-06 Cohort | No Intervention | All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 (NCT03311854) will be invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants will not receive emapalumab in the current study (NI-0501-05, NCT02069899). | |
| Enrolled-CU Cohort | Other | In exceptional cases, at the spontaneous request of a treating physician, CU treatment will be granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who receive at least 1 dose of emapalumab under CU will be invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants can continue treatment in the context of the current Study (NI-0501-05, NCT02069899) while stem cell donor search is ongoing, or if the investigator assesses that continuation of treatment is beneficial. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emapalumab | Drug | Treatment with emapalumab is not planned for all enrolled participants. For participants who will continue receiving emapalumab in the context of this study (NI-0501-05), the dose and timing will be either carried forward from the last administered emapalumab dose as part of the parent study in which the participant was enrolled, or an adjusted dose will be administered, if necessary. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Event (AE) | Adverse events were defined as any undesirable experience occurring in a participant during the study, whether or not considered related to emapalumab. | From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Duration of Response (Enrolled-04 Cohort) | Cumulative duration of response: total number of days in response from 1st achievement of overall response until HSCT or last treatment date if the participant did not undergo HSCT. Overall response: achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI). CR: no fever, normal spleen size, no cytopenia (absolute neutrophil count [ANC] ≥1.0 x 10^9/L and platelet count ≥ 100 x 10^9/L), no hyperferritinemia (serum ferritin <2000 μg/L), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and cerebrospinal fluid [CSF] abnormalities attributed to HLH, no sustained worsening of soluble cluster of differentiation (CD) 25. PR: at least 3 HLH clinical and laboratory criteria (including central nervous system [CNS] abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology. HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory abnormalities (including CNS involvement). |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Spectrum Health Helen Devos Children's Hospital | Grand Rapids | Michigan | 49503 | United States | ||
| University of North Carolina at Chapel Hill |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| Result | Jordan MB, Locatelli F, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington T, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, Grom A, De Benedetti F, de Min C. Post-Transplant Outcomes of Children with Primary Hemophagocytic Lymphohistiocytosis Treated with Emapalumab.Transplant Cell Ther. 2021; 27(Supplement 3): S118. | ||
| Result | Laveille C, Jacqmin P, de Graaf K, de Min C. Population Pharmacokinetic Analysis of Emapalumab, a Fully Human, Anti-Interferon Gamma Monoclonal Antibody, in Children with Primary Hemophagocytic Lymphohistiocytosis. Blood 2020; 136 (Supplement 1): 20 | ||
| 32374962 | Result | Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326. |
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Participants with hemophagocytic lymphohistiocytosis (HLH) who had received at least 1 dose of emapalumab in the context of a previous emapalumab clinical study (Study NI-0501-04 [NCT01818492] or Study NI-0501-06 [NCT03311854]) in which no long-term follow-up was planned, and participants who received emapalumab through a compassionate use (CU) were enrolled in the current study (NI-0501-05, NCT02069899).
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| ID | Title | Description |
|---|---|---|
| FG000 | Enrolled-04 Cohort | Participants enrolled in Study NI-0501-04 were invited to participate for long-term follow-up for 1 year either after haematopoietic stem cell transplantation (HSCT) or after the last administration of emapalumab. In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT. For participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT was delayed for reasons unrelated to the administration of emapalumab, they could continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05) at the request of the investigator, providing a favorable benefit/risk assessment of treatment was established. Treatment with emapalumab was not planned for all enrolled participants. For participants who continued receiving emapalumab in the context of this study (NI-0501-05), the dose and timing was either carried forward from the last administered emapalumab dose as part of the parent study in which the participant was enrolled, or an adjusted dose was administered, if necessary. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Oct 31, 2019 | May 17, 2022 |
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|
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| From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days) |
| Duration of First Response (Enrolled-06 Cohort) | Duration of first response was defined as the number of days between first date of response and first date of loss of response or death. Response was defined as macrophage activation syndrome (MAS) remission, which was resolution of clinical signs and symptoms according to the Investigator (MAS clinical signs and symptoms score ≤ 1) and normalization of laboratory parameters relevant to MAS as follows: white blood cells (WBC) and platelet count above the upper limit of normal (LLN), Lactate dehydrogenase < 1.5 × lower limit of normal (ULN), aspartate aminotransferase/alanine aminotransferase <1.5 × ULN, fibrinogen > 100 mg/dL, ferritin level decreased by at least 80% from values at screening or baseline (whichever was higher) or < 2000 ng/mL, whichever was lower. | From first date of response and first date of loss of response or death (maximum 416 days) |
| Overall Survival (Enrolled-04 Cohort) | Overall survival was defined as time from the date of the last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As some participants had their last emapalumab dose in the parent study (NI-0501-04), data from both NI-0501-05 and NI-0501-04 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) |
| Overall Survival (Enrolled-06 Cohort) | Overall survival was defined as time from the date of last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As participants in the Enrolled-06 Cohort did not receive emapalumab in the current study, data from both NI-0501-05 and NI-0501-06 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) |
| Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) | For participants who underwent HSCT either in parent study (NI-0501-04) or current study (NI-0501-05), engraftment rate was based on the number of participants experiencing primary or secondary graft failure (blood stem cell transplant failure, engraft failure, or transplant dysfunction), as reported as an adverse event. | From HSCT up to 12 months |
| Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) | For participants who underwent HSCT, achievement of donor chimerism was considered based on donor chimerism in peripheral blood completed, that is, donor cells >95%. | From HSCT to 12 months |
| Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) | Occurrence of graft-versus-host-disease, reported in Study NI-0501-05 as an AE. | From HSCT to 12 months |
| MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort) | MAS activity was monitored using a visual analogue scale ranging from 0 to 10 with a higher score indicted higher disease activity. | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study |
| Circulating Emapalumab Level (Enrolled-04 Cohort) | Circulating Emapalumab level in Enrolled-04 Cohort who continued to receive treatment with emapalumab in the current study (NI-0501-05). | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant |
| Circulating Emapalumab Level (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 6 |
| Total Human Interferon Gamma Levels (Enrolled-04 Cohort) | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, Day 100 post-transplant, 12 months post-transplant |
| Total Human Interferon Gamma Levels (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study |
| Number of Participants With Anti-drug Antibody | From enrolment up to 12 months post-transplant or last emapalumab infusion (maximum 639 days) |
| Chapel Hill |
| North Carolina |
| 27599 |
| United States |
| Cincinnati Children's Hospital - Division of Immunobiology | Cincinnati | Ohio | 45229-3039 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| Texas Children's Cancer Center | Houston | Texas | 77030 | United States |
| Hôpital Necker-Enfants Malades | Paris | 75743 | France |
| Fondazione MBBM c/o Ospedale San Gerardo Clinica Pediatrica | Monza | 20900 | Italy |
| Azienda Ospedaliera Padova | Padova | 35128 | Italy |
| Ospedale Pediatrico Bambino Gesu - UO Reumatologia | Rome | 00165 | Italy |
| Ospedale Pediatrico Bambino Gesu | Rome | 00165 | Italy |
| Ospedale della Donna e del Bambino - U.O.C. Oncoematologia Pediatrica | Verona | 37126 | Italy |
| Hospital Universitario Vall d'Hebron Servei de Hematologia i Oncologia | Barcelona | 08035 | Spain |
| Sant Joan de Déu Hospital - Pediatric Rheumatology Department | Barcelona | 08950 | Spain |
| Hospital Universitario Niño Jesús Servicio de Hemato-Oncología Pediátrica | Madrid | 28009 | Spain |
| UCL Institute of Child Health Great Ormond Street Hospital | London | WC1N1EH | United Kingdom |
| Great Ormond Street Hospital - Department of Haematology | London | WC1N3JH | United Kingdom |
| 38662147 | Derived | Brossard P, Laveille C. Population Pharmacokinetics of the Anti-Interferon-Gamma Monoclonal Antibody Emapalumab: An Updated Analysis. Rheumatol Ther. 2024 Jun;11(3):869-880. doi: 10.1007/s40744-024-00669-y. Epub 2024 Apr 25. |
| 37001971 | Derived | De Benedetti F, Grom AA, Brogan PA, Bracaglia C, Pardeo M, Marucci G, Eleftheriou D, Papadopoulou C, Schulert GS, Quartier P, Anton J, Laveille C, Frederiksen R, Asnaghi V, Ballabio M, Jacqmin P, de Min C. Efficacy and safety of emapalumab in macrophage activation syndrome. Ann Rheum Dis. 2023 Jun;82(6):857-865. doi: 10.1136/ard-2022-223739. Epub 2023 Mar 31. |
| FG001 | Enrolled-06 Cohort | All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 were invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants did not receive emapalumab in the current study (NI-0501-05). |
| FG002 | Enrolled-CU Cohort | In exceptional cases, at the spontaneous request of a treating physician, CU treatment was granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who received at least 1 dose of emapalumab were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants could have continued treatment in the context of the current Study (NI-0501-05) while stem cell donor search was ongoing, or if the investigator assessed that continuation of treatment was beneficial. |
| Treated in Current Study |
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| COMPLETED |
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| NOT COMPLETED |
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|
Participants who previously received treatment in a parent study (NI-0501-04 or NI-0501-06) or under compassionate use and who provided informed consent for participation in Study NI-0501-05.
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| ID | Title | Description |
|---|---|---|
| BG000 | Enrolled-04 Cohort | Participants enrolled in Study NI-0501-04 were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT. For participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT was delayed for reasons unrelated to the administration of emapalumab, they could continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05) at the request of the investigator, providing a favorable benefit/risk assessment of treatment was established. Treatment with emapalumab was not planned for all enrolled participants. For participants who continued receiving emapalumab in the context of this study (NI-0501-05), the dose and timing was either carried forward from the last administered emapalumab dose as part of the parent study in which the participant was enrolled, or an adjusted dose was administered, if necessary. |
| BG001 | Enrolled-06 Cohort | All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 were invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants did not receive emapalumab in the current study (NI-0501-05). |
| BG002 | Enrolled-CU Cohort | In exceptional cases, at the spontaneous request of a treating physician, CU treatment was granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who received at least 1 dose of emapalumab were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants could have continued treatment in the context of the current Study (NI-0501-05) while stem cell donor search was ongoing, or if the investigator assessed that continuation of treatment was beneficial. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants | No |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
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| Ethnicity | Ethnicity not collected | Count of Participants | Participants | No |
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| 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 | Number of Participants With Adverse Event (AE) | Adverse events were defined as any undesirable experience occurring in a participant during the study, whether or not considered related to emapalumab. | Participants who previously received treatment in a parent study (NI-0501-04 or NI-0501-06) or under compassionate use and who provided informed consent for participation in Study NI-0501-05. | Posted | Count of Participants | Participants | No | From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days) |
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| Secondary | Cumulative Duration of Response (Enrolled-04 Cohort) | Cumulative duration of response: total number of days in response from 1st achievement of overall response until HSCT or last treatment date if the participant did not undergo HSCT. Overall response: achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI). CR: no fever, normal spleen size, no cytopenia (absolute neutrophil count [ANC] ≥1.0 x 10^9/L and platelet count ≥ 100 x 10^9/L), no hyperferritinemia (serum ferritin <2000 μg/L), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and cerebrospinal fluid [CSF] abnormalities attributed to HLH, no sustained worsening of soluble cluster of differentiation (CD) 25. PR: at least 3 HLH clinical and laboratory criteria (including central nervous system [CNS] abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology. HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory abnormalities (including CNS involvement). | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who had at least 1 response. | Posted | Mean | Standard Deviation | days | From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days) |
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| Secondary | Duration of First Response (Enrolled-06 Cohort) | Duration of first response was defined as the number of days between first date of response and first date of loss of response or death. Response was defined as macrophage activation syndrome (MAS) remission, which was resolution of clinical signs and symptoms according to the Investigator (MAS clinical signs and symptoms score ≤ 1) and normalization of laboratory parameters relevant to MAS as follows: white blood cells (WBC) and platelet count above the upper limit of normal (LLN), Lactate dehydrogenase < 1.5 × lower limit of normal (ULN), aspartate aminotransferase/alanine aminotransferase <1.5 × ULN, fibrinogen > 100 mg/dL, ferritin level decreased by at least 80% from values at screening or baseline (whichever was higher) or < 2000 ng/mL, whichever was lower. | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and who had at least 1 response. | Posted | Median | Inter-Quartile Range | days | From first date of response and first date of loss of response or death (maximum 416 days) |
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| Secondary | Overall Survival (Enrolled-04 Cohort) | Overall survival was defined as time from the date of the last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As some participants had their last emapalumab dose in the parent study (NI-0501-04), data from both NI-0501-05 and NI-0501-04 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05. | Posted | Median | Inter-Quartile Range | days | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) |
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| Secondary | Overall Survival (Enrolled-06 Cohort) | Overall survival was defined as time from the date of last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As participants in the Enrolled-06 Cohort did not receive emapalumab in the current study, data from both NI-0501-05 and NI-0501-06 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05. | Posted | Median | Inter-Quartile Range | days | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) |
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| Secondary | Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) | For participants who underwent HSCT either in parent study (NI-0501-04) or current study (NI-0501-05), engraftment rate was based on the number of participants experiencing primary or secondary graft failure (blood stem cell transplant failure, engraft failure, or transplant dysfunction), as reported as an adverse event. | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | Posted | Number | 95% Confidence Interval | percentage of participants | From HSCT up to 12 months |
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| Secondary | Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) | For participants who underwent HSCT, achievement of donor chimerism was considered based on donor chimerism in peripheral blood completed, that is, donor cells >95%. | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | Posted | Number | 95% Confidence Interval | percentage of participants | From HSCT to 12 months |
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| Secondary | Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) | Occurrence of graft-versus-host-disease, reported in Study NI-0501-05 as an AE. | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | Posted | Number | 95% Confidence Interval | percentage of participants | From HSCT to 12 months |
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| Secondary | MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort) | MAS activity was monitored using a visual analogue scale ranging from 0 to 10 with a higher score indicted higher disease activity. | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and had available data for MAS activity. | Posted | Mean | Standard Deviation | score on a scale | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study |
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| Secondary | Circulating Emapalumab Level (Enrolled-04 Cohort) | Circulating Emapalumab level in Enrolled-04 Cohort who continued to receive treatment with emapalumab in the current study (NI-0501-05). | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who had data available for specified timepoints. Circulating emapalumab level post-transplant included participants who had HSCT and data available at 12 months post-HSCT (Samples were not to be taken once it had been determined that emapalumab was below measurable level of 62.5 µg/L). | Posted | Mean | Standard Deviation | µg/L | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant |
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| Secondary | Circulating Emapalumab Level (Enrolled-06 Cohort) | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and had available data at specified timepoints (Samples were not to be taken once it had been determined that emapalumab was below measurable level of 62.5 µg/L). | Posted | Mean | Standard Deviation | µg/L | Baseline (first NI-0501-05 visit), Day 100, Month 6 |
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| Secondary | Total Human Interferon Gamma Levels (Enrolled-04 Cohort) | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who had data available for specified timepoints. Total human interferon gamma levels post-transplant included participants who underwent HSCT and data available at Day 100 and 12 months post-HSCT. | Posted | Mean | Standard Deviation | ng/L | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, Day 100 post-transplant, 12 months post-transplant |
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| Secondary | Total Human Interferon Gamma Levels (Enrolled-06 Cohort) | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and had available data at specified timepoints. | Posted | Mean | Standard Deviation | ng/L | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study |
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| Secondary | Number of Participants With Anti-drug Antibody | Participants who previously received treatment in a parent study (NI-0501-04 or NI-0501-06) or under CU and who provided informed consent for participation in Study NI-0501-05. Number of participants analysed included participants who had available data for anti-drug antibody. | Posted | Count of Participants | Participants | No | From enrolment up to 12 months post-transplant or last emapalumab infusion (maximum 639 days) |
|
From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days)
Participants who previously received treatment in a parent study (NI-0501-04 or NI-0501-06) or under CU and who provided informed consent for participation in Study NI-0501-05.
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 | Enrolled-04 Cohort | Participants enrolled in Study NI-0501-04 were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT. For participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT was delayed for reasons unrelated to the administration of emapalumab, they could continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05) at the request of the investigator, providing a favorable benefit/risk assessment of treatment was established. Treatment with emapalumab was not planned for all enrolled participants. For participants who continued receiving emapalumab in the context of this study (NI-0501-05), the dose and timing was either carried forward from the last administered emapalumab dose as part of the parent study in which the participant was enrolled, or an adjusted dose was administered, if necessary. | 9 | 37 | 28 | 37 | 37 | 37 |
| EG001 | Enrolled-06 Cohort | All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 were invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants did not receive emapalumab in the current study (NI-0501-05). | 0 | 14 | 3 | 14 | 11 | 14 |
| EG002 | Enrolled-CU Cohort | In exceptional cases, at the spontaneous request of a treating physician, CU treatment was granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who received at least 1 dose of emapalumab were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants could have continued treatment in the context of the current Study (NI-0501-05) while stem cell donor search was ongoing, or if the investigator assessed that continuation of treatment was beneficial. | 2 | 7 | 6 | 7 | 7 | 7 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Coombs positive haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Right ventricular dysfunction | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Eye movement disorder | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumatosis intestinalis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute graft versus host disease in intestine | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Engraftment syndrome | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Klebsiella sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchiolitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Enterococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gianotti-Crosti syndrome | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia cytomegaloviral | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Engraft failure | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Blood stem cell transplant failure | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Myositis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neurological decompensation | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Subdural hygroma | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary alveolar haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Circulatory collapse | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Shock | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Still's disease | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Extremity necrosis | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Device breakage | Product Issues | MedDRA 23.0 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Increased tendency to bruise | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lymphocytosis | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Macrocytosis | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cyanosis | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Left ventricular hypertrophy | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Right ventricular dysfunction | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Adrenal insufficiency | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Secondary adrenocortical insufficiency | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Eye movement disorder | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Maculopathy | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Periorbital oedema | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Retinal disorder | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Retinal ischaemia | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diverticulum | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Duodenal ulcer | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastric mucosa erythema | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lip ulceration | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oral mucosal discolouration | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Tongue ulceration | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Complication associated with device | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Complication of device insertion | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fibrosis | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Localised oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Swelling face | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Visceral pain | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Xerosis | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Drug-induced liver injury | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperplastic cholecystopathy | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Graft versus host disease in skin | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Allergy to immunoglobulin therapy | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Graft versus host disease in gastrointestinal tract | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Graft versus host disease in liver | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute graft versus host disease in skin | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bacille Calmette-Guerin scar reactivation | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Engraftment syndrome | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Graft versus host disease | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Enterococcal bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis adenovirus | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| BK virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Bacillus bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Cytomegalovirus chorioretinitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Epstein-Barr viraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Escherichia bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis norovirus | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis salmonella | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Herpes simplex gastritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Human herpesvirus 6 infection reactivation | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Klebsiella bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Meningitis enterococcal | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Oesophageal candidiasis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Serratia bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Skin bacterial infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary tract infection viral | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral haemorrhagic cystitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Allergic transfusion reaction | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Blood stem cell transplant failure | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Burns first degree | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Engraft failure | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Transplant dysfunction | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Unintentional medical device removal | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Vascular access complication | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| BK polyomavirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Roseolovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Atypical mycobacterium test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood albumin decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood electrolytes abnormal | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood immunoglobulin G decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Campylobacter test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Clostridium test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Cytomegalovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Herpes simplex test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Mycobacterium test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Norovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Respirovirus test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Urine output decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypercreatininaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Milk soy protein intolerance | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Poor feeding infant | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Growth failure | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hydrocephalus | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypotonia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nystagmus | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Opisthotonus | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Subdural effusion | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Separation anxiety disorder | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pelvi-ureteric obstruction | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Scrotal swelling | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nasal flaring | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Drug reaction with eosinophilia and systemic symptoms | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperaemia | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rectal prolapse | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cestode infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Adverse event following immunisation | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Still's disease | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sickle cell trait | Congenital, familial and genetic disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Enterocolitis haemorrhagic | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Enterobacter sepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Periorbital cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Tracheobronchitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Torus fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Acinetobacter test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Candida test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Klebsiella test positive | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Ubiquinone decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypertrophic osteoarthropathy | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pleocytosis | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Anuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oliguria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Panniculitis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
|
The sponsor can review results communication prior to public release. The PI shall consider the comments received from the sponsor and shall delete any confidential information. The sponsor may require the delay of publication submission if it contains any patentable subject matter which requires protection.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Radmila Kanceva/Senior Medical Director Immunology | Sobi AG | + 41 22 551 91 63 | Radmila.Kanceva@sobi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 26, 2021 | May 17, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D051359 | Lymphohistiocytosis, Hemophagocytic |
| ID | Term |
|---|---|
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000644327 | Emapalumab |
Not provided
Not provided
Not provided
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| Participants |
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All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 were invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants did not receive emapalumab in the current study (NI-0501-05). |
| OG002 | Enrolled-CU Cohort | In exceptional cases, at the spontaneous request of a treating physician, CU treatment was granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who received at least 1 dose of emapalumab were invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants could have continued treatment in the context of the current Study (NI-0501-05) while stem cell donor search was ongoing, or if the investigator assessed that continuation of treatment was beneficial. |
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