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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-003632-23 | EudraCT Number |
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| Name | Class |
|---|---|
| Seventh Framework Programme | OTHER |
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The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.
The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.
All participants in the NI-0501-04 study (NCT01818492) were invited to participate in the long-term follow-up study NI-0501-05 (NCT02069899). For the primary completion date, mentioned here, we refer to the NI-0501-04 study, even though in accordance with the NI-0501-04 study objectives, namely the assessment of long-term efficacy and safety endpoints, the study analyses also included data collected in the long-term follow-up study NI-0501-05. Hence these data are reported together. Study NI-0501-05 accepts patients from NI-0501-04 and NI-0501-06. Data collection for the patients from NI-0501-04 is completed.
The primary efficacy and safety analyses are based on the regulatory cut-off date of 20 July 2017. Refer to the publication in N Engl J Med 2020 May 7; 382 (19):1811-1822. Follow-on analyses have been conducted on all patients enrolled in the study, i.e. including the patients enrolled after the cut-off date of 20 July 2017. The results reported here refer to the totality of the 45 patients enrolled.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NI-0501 | Experimental | NI-0501 administered by IV infusion at a starting dose of 1 mg/kg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NI-0501 | Biological |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) Second Line | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
| Overall Response Rate (ORR) All Treated | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
| Overall Response Rate (ORR) Follow-on Analysis Set: | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Response | Time from the date of the first dose of emapalumab to first achievement of response (at least HLH improvement) | Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
| Durability of First Response |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Radmila Kanceva, MD | Swedish Orphan Biovitrum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045-7106 | United States | ||
| Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 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. | |
| 32374962 | Derived | 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. |
| Label | URL |
|---|---|
| NI-0501-05, long-term follow-up study description on clinicaltrials.gov | View source |
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Patients were screened within 1 week prior to the first administration of emapalumab (NI-0501). 66 patients were screened, and 45 were enrolled and treated.
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| ID | Title | Description |
|---|---|---|
| FG000 | NI-0501 | All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks. |
| 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: US protocol | Mar 24, 2016 | Jun 17, 2020 |
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| End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
| Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
Maintenance of response achieved any time during the study |
| Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
| Overall Survival | Number of patients being alive at end of treatment or at week 8, pending on which comes first. | Time from the date of first dose to last dose, or 8 weeks after first dose. |
| Number of Patients Able to Reduce Glucocorticoids | Number of patients able to reduce glucocorticoids by 50% or more and between ≥30%-<50%, of baseline dose at EOT 04. | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks. |
| Cumulative Duration of Response | Percent of treatment time in response from the first achievement of an Overall Response until HSCT conditioning, or End of Treatment 04/05 (if the patient did not have HSCT performed) Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05. | up to start of HSCT conditioning, whenever HSCT conditioning is scheduled (at least 4 weeks after treatment start), or End of Treatment 04/05 (if the patient did not have HSCT performed) |
| Survival Pre-HSCT | Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients who receive HSCT will be censored at that date; patients who did not receive HSCT will be censored at last date of contact. Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05. | Assessed up to HSCT, whenever HSCT occurred (up to approximately 6 months after treatment initiation) |
| Survival Post-HSCT | Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients without an event will be censored at last assessment date in either the NI-0501-04 or NI-0501-05 study. Patients who do not proceed to HSCT will be excluded from this analysis. | Assessed up to Last Observation (up to 1 year after transplant, or up to approximately 18 months after treatment initiation) |
| Wilmington |
| Delaware |
| 19803 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | United States |
| Dana-Farber Cancer Institute (DFCI) | Boston | Massachusetts | 02115 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Cincinnati Children's Hospital - Division of Immunobiology - Department of Pediatrics | Cincinnati | Ohio | 45229-3039 | United States |
| Texas Children's Cancer Center | Houston | Texas | 77030 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| University Children's Hospital | Münster | 48149 | Germany |
| Azienda Ospedaliero Universitaria Meyer | Florence | 50139 | Italy |
| Istituto Giannina Gaslini | Genoa | 16147 | Italy |
| Azienda Ospedaliera San Gerardo | Monza | 20900 | Italy |
| Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica | Padua | 35128 | Italy |
| Ospedale Pediatrico Bambino Gesu' | Roma | 00165 | Italy |
| Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica | Verona | 37126 | Italy |
| Hospital Sant Joan de Déu | Barcelona | 08950 | Spain |
| Hospital Universitario Vall d'Hebron | Barcelona | 119-129 | Spain |
| Hospital Universitario Niño Jesús | Madrid | 28009 | Spain |
| Karolinska University Hospital | Stockholm | 17176 | Sweden |
| Great Ormond Street Hospital - Department of Haematology | London | WC1N 3JH | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | NI-0501 | All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| ||||||||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| |||||||||||||||||||||||
| HLH disease chracteristics at baseline | 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 | Overall Response Rate (ORR) Second Line | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | Primary analysis set: Second Line (27 patients who had previously received conventional HLH therapy before enrollment, data collected by regulatory cut-off: 20 July 2017) | Posted | Number | 95% Confidence Interval | percentage of participants | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Primary | Overall Response Rate (ORR) All Treated | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | Primary analysis set: All Treated (all patients who received any part of an emapalumab infusion, data collected by regulatory cut-off: 20 July 2017) | Posted | Number | 95% Confidence Interval | percentage of participants | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Primary | Overall Response Rate (ORR) Follow-on Analysis Set: | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | Follow-on analysis set: Second Line (34 patients who had previously received conventional HLH therapy before enrollment, totality of the data collected in the NI-0501-04 study) | Posted | Number | 95% Confidence Interval | percentage of participants | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Primary | Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated | Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including 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 criteria (including CNS involvement). | Follow-on analysis set: All Treated (45 patients who received any part of an emapalumab infusion, totality of the data collected in the NI-0501-04) | Posted | Number | 95% Confidence Interval | percentage of participants | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Secondary | Time to Response | Time from the date of the first dose of emapalumab to first achievement of response (at least HLH improvement) | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Median | 95% Confidence Interval | Count of days | Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Secondary | Durability of First Response | Maintenance of response achieved any time during the study | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Median | 95% Confidence Interval | Count of days | Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks) |
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| Secondary | Overall Survival | Number of patients being alive at end of treatment or at week 8, pending on which comes first. | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Number | participants | Time from the date of first dose to last dose, or 8 weeks after first dose. |
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| Secondary | Number of Patients Able to Reduce Glucocorticoids | Number of patients able to reduce glucocorticoids by 50% or more and between ≥30%-<50%, of baseline dose at EOT 04. | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Number | Percentage of patients | End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks. |
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| Secondary | Cumulative Duration of Response | Percent of treatment time in response from the first achievement of an Overall Response until HSCT conditioning, or End of Treatment 04/05 (if the patient did not have HSCT performed) Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05. | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Median | Inter-Quartile Range | Percentage of treatment time | up to start of HSCT conditioning, whenever HSCT conditioning is scheduled (at least 4 weeks after treatment start), or End of Treatment 04/05 (if the patient did not have HSCT performed) |
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| Secondary | Survival Pre-HSCT | Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients who receive HSCT will be censored at that date; patients who did not receive HSCT will be censored at last date of contact. Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05. | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Number | 95% Confidence Interval | Kaplan-Meier survival probability | Assessed up to HSCT, whenever HSCT occurred (up to approximately 6 months after treatment initiation) |
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| Secondary | Survival Post-HSCT | Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients without an event will be censored at last assessment date in either the NI-0501-04 or NI-0501-05 study. Patients who do not proceed to HSCT will be excluded from this analysis. | All Treated (all patients who received any part of an emapalumab infusion) | Posted | Number | 95% Confidence Interval | Kaplan-Meier survival probability | Assessed up to Last Observation (up to 1 year after transplant, or up to approximately 18 months after treatment initiation) |
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Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation).
Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
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 | Pre-conditioning | All patients before start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation. | 10 | 45 | 28 | 45 | 41 | 45 |
| EG001 | Post-conditioning | All patients after start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation. | 5 | 30 | 20 | 30 | 28 | 30 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
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| Lymphocytosis | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
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| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
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| Cardiac arrest | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
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| Cardiomyopathy | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
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| Cardiopulmonary failure | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
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| Right ventricular dysfunction | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
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| Eye movement disorders | Eye disorders | MedDRA 20.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Inguinal hernia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Pneumatosis intestinalis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Condition aggravated | General disorders | MedDRA 20.0 | Systematic Assessment |
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| Multiple organ dysfunction | General disorders | MedDRA 20.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
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| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA 20.0 | Systematic Assessment |
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| Acute GVHD in intestine | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
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| Anaphylactic reaction | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
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| Engraftment syndrome | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Enterococcal sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Gastroenteritis norovirus | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Urosepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Appendicitis perforated | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Bacterial sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Gianotti-Crosti syndrome | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Histoplasmosis disseminated | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Necrotizing fasciitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Pneumocystis jirovecii pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Respiratory syncytial virus infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Staphylococcal bacteraemia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Clostridium difficile colitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Clostridium difficile infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Klebsiella sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Bronchiolitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Enterococcal infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Epstein-Barr virus infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Gastroenteritis rotavirus | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Pneumonia cytomegaloviral | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Pneumonia pseudomonal | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Rhinovirus infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| BSCT failure | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Engraft failure | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cerebral disorders | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Neurological decompensation | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Subdural hygroma | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ARDS | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary alveolar haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary artery thrombosis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Circulatory collapse | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lymphocytosis | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Graft versus host disease | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Graft versus host disease in gastrointestinal tract | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Graft versus host disease in liver | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Graft versus host disease in skin | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| BK virus infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood immunoglobulin G decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Polyomavirus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Carl Johan Treutiger | Sobi AB | 46 76 00 11 815 | CarlJohan.Treutiger@sobi.com |
| Prot_000.pdf |
| Prot | Yes | No | No | Study Protocol: EU protocol | Feb 26, 2016 | Jun 17, 2020 | Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 20, 2019 | Jun 19, 2020 | SAP_002.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
| ≥ 12 - <18 years |
|
| African Descent |
|
| Mixed/multi-racial |
|
| LOther |
|
| Platelet counts < 100 x10^9/L |
|
| Neutrophil counts < 1 x10^9/L |
|
| Ferritin > 500 μg/L |
|
| Ferritin > 2000 μg/L |
|
| Fibrinogen < 1.5 g/L |
|
| Triglycerides > 3 mmol/L |
|
| D-Dimers > 500 μg/L |
|
| ALT > 125 IU/L |
|
| Total bilirubin > 25 μmol/L |
|
| LDH > 1000 IU/L |
|
| CNS involvement |
|
|
|
|
|
|
|
| Participants |
|
|
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
|
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|