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Sponsor Decision related to Business Priorities, Assessment of Development Options
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| Name | Class |
|---|---|
| PRA Health Sciences | INDUSTRY |
| Cytel Inc. | INDUSTRY |
| Q2 Solutions | INDUSTRY |
| ABF Pharmaceutical Services GmbH |
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This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF. Patients presenting CXCL9 levels above a defined threshold and other clinical criteria will be eligible to receive emapalumab.
Both children and adults, with malignant and non-malignant underlying diseases, receiving allo-HSCT who are at high risk of GF as defined in the inclusion criteria will be included in the study. The main objective of the study is to determine the appropriate emapalumab dose regimen neutralizing interferon gamma (IFNγ) activity to pre-empt graft failure post allo-HSCT in a population with various underlying diseases and at high risk of graft failure (GF).
Maximum 3 cohorts are foreseen to determine the appropriate dose regimen to pre-emptively treat patients at risk of primary GF.
Emapalumab will be administered by IV infusion and treatment will last up to 56 days (15 infusions) or until evidence of engraftment.
The study is expected to last approximately 3 years from screening to the last follow-up phone call for each patient.
This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF. Patients presenting CXCL9 levels above a defined threshold and other clinical criteria will be eligible to receive emapalumab.
Both children and adults, with malignant and non-malignant underlying diseases, receiving allo-HSCT who are at high risk of GF as defined in the inclusion criteria will be included in the study. The main objective of the study will be to determine the appropriate emapalumab dose regimen neutralizing interferon gamma (IFNγ) activity to pre-empt graft failure post allo-HSCT in a population with various underlying diseases and at high risk of graft failure (GF).
Maximum 3 cohorts are foreseen to determine the appropriate dose regimen to pre-emptively treat patients at risk of primary GF.
Emapalumab will be administered by IV infusion over 1 to 2 hours depending on the volume of the infusion. Treatment will last up to 56 days (15 infusions) or until evidence of engraftment.
The study is comprised of the following study periods: screening (Day -21 to Day -8), allogeneic HSCT Day 0, monitoring period for primary GF (Day 1 up to Day 42), extended monitoring for secondary GF (up to Day 98), treatment period (up to 56 days) and follow-up period of 3 years after HSCT.
The main objective of this proof of concept study is:
• To determine the appropriate emapalumab dose regimen neutralizing interferon gamma (IFNγ) activity to pre-empt graft failure post allogeneic hematopoietic stem cell transplantation (HSCT) in a population with various underlying diseases and at high risk of graft failure (GF)
The following objectives will support the dose selection:
Exploratory objectives will be:
• To evaluate further data on the correlation between relevant biomarkers including C-X-C motif chemokine ligand 9 (CXCL9) levels and the risk of GF post allo-HSCT in a population with various underlying diseases and at high risk of GF also in the context of development of a diagnostic test.
The study is expected to last approximately 3 years from screening to the last follow-up phone call for each patient.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Emapalumab | Experimental | The first cohort of patients will receive a first infusion of 6 mg/kg at TD0, followed by a second infusion at 3 mg/kg after 3 days (treatment day 3 - TD3). Subsequent infusions of 3 mg/kg will be every 3 or 4 days from previous dose until dose 15 or until engraftment. A maximum of 2 additional cohorts may be added to allow dosing regimen adaptation based on the PK/PD data observed from the previous cohort(s). Efficacy and safety data will also be considered before adding additional cohorts. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emapalumab | Drug | Emapalumab is a fully human immunoglobulin G1 (IgG1) anti-IFNγ monoclonal antibody that binds to and neutralizes IFNγ. Emapalumab binds to both soluble and receptor (IFNγR1)-bound forms of IFNγ. Emapalumab is in development for treatment of primary and secondary HLH. The benefit expected from the targeted neutralization of IFNγ by emapalumab has been validated by the recent FDA approval of emapalumab for treatment of patients with pHLH who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. The safety profile has been assessed as acceptable. Emapalumab will be administered by intravenous infusion over 1 to 2 hours, depending on the volume of the infusion. The first infusion must be performed within 12 hours after CXCL9 levels have been measured above defined threshold. Treatment will last until maximum dose 15 (up to 56 days) or until evidence of engraftment, whichever comes first. |
| Measure | Description | Time Frame |
|---|---|---|
| CXCL9 in Serum | Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9) | From start of treatment to EoS Visit, up to 34 weeks |
| Primary Graft Failure (GF) | Number of participants with primary graft failure (GF) | From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks |
| Secondary GF | Number of participants with secondary GF | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Free & Total Interferon Gamma (IFNγ) in Serum | Serum concentration of free and total Interferon gamma (IFNγ) | From start of treatment to EoS Visit, up to 34 weeks |
| Emapalumab in Serum - Peak | Peak emapalumab serum concentration |
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Inclusion Criteria:
Informed consent form signed by the patient (as required by law) or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable
Recipients of allogeneic Hematopoietic Stem Cell Transplantation (HSCT) and at high risk of graft failure (GF) based on at least one of the following criteria:
Patients requiring allo-HSCT with the following underlying diseases:
Male and female patients
Children aged at least 1 year and adults. Once the appropriate dose has been determined in one of the three cohorts and safety has been assessed by the Independent Data Monitoring Committee (IDMC), children less than 1 year old may be included in the study.
Females of child-bearing potential, defined as all women physiologically capable of becoming pregnant, require use of highly effective contraceptive measures from screening until 6 months after the last study drug administration
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Tsila Zuckerman, Dr | The Rambam Academic Hospital | Principal Investigator |
| Henrique Bittencourt, Dr | St. Justine's Hospital | Principal Investigator |
| Polina Stepensky, Dr | Hadassah Hebrew University | Principal Investigator |
| Ashvind Prabahran, Dr | Peter MacCallum Cancer Centre, Australia | Principal Investigator |
| Richard Mitchell, Dr | Kids Cancer Centre Sydney Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peter MacCallum Cancer Centre | Melbourne | 3000 | Australia | |||
| Kids Cancer Centre Sydney Children's Hospital |
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One site was activated which screened a total of 3 subjects (2 enrolled; 1 screen failure). First and last subjects were enrolled 25May2021 and 07Oct2021 respectively.
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| ID | Title | Description |
|---|---|---|
| FG000 | Emapalumab Treated | Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment) |
| FG001 | Non-emapalumab Treated | Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Allogeneic HSCT (Day 0) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | May 28, 2021 |
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| INDUSTRY |
| Cromsource | INDUSTRY |
| BioMérieux | INDUSTRY |
Sequential dose cohorts. The first cohort of patients will receive a first emapalumab infusion of 6 mg/kg at TD0, followed by a second infusion at 3 mg/kg after 3 days. Subsequent infusions of 3 mg/kg will be every 3 or 4 days from previous dose until dose 15 or until engraftment. A maximum of 2 additional cohorts may be added to allow dosing regimen adaptation based on the PK/PD data observed from the previous cohort(s).
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|
|
| From start of treatment to EoS, up to 34 weeks |
| Ctrough (Emapalumab) | Concentration just before administration | From start of treatment to EoS, up to 34 weeks |
| Exploratory Biomarkers: Ferritin | Ferritin - serum concentration | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| ADA and nAbs | Number of participants developing antibodies against emapalumab (antidrug antibodies [ADA]) and Neutralizing antibodies (nAb) | From Start of treatment until EoS, up to 34 weeks |
| Number of Participants With Mixed Donor Chimerism <10% and <20% | Based on unselected leukocytes and based on sorted T cells | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants Receiving Thrombopoietic Agents, Stem Cell Boost, Donor Lymphocyte Infusion (DLI) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants Receiving a Second Allogeneic HSCT | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants With Poor Graft Function | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants With Event Free Engraftment | defined as absence of GF or graft support | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD) | (grade I to IV) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Biomarker Levels, in Particular IFNy and CXCL9, as Predictors of Primary and/or Secondary Graft Failure or Acute and/or Chronic GvHD | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Engraftment Syndrome | Number of participants with engraftment syndrome | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants With Endothelial Complications | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Participants With Relapse, Defined as Cumulative Incidence of Reoccurring Underlying Disease | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Survival Rate | Number of patients alive at the end of study. | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Body Temperature | Change from baseline in body temperature | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Heart Rate | Change from baseline in heart rate | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Blood Pressure | Change from baseline systolic and diastolic blood pressure | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Body Weight | Change from baseline in body weight | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology: Red Blood Cells (RBC) | Change from baseline in Hematology: red blood cells (RBC) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology: Hematocrit | Change from baseline in Hematology: hematocrit | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology: Hemoglobin | Change from baseline in Hematology: hemoglobin | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology: Platelets | Change from baseline in Hematology: platelets | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology: White Blood Cells (WBC) | Change from baseline in Hematology: white blood cells (WBC) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology Differential: Lymphocytes | Change from baseline in Hematology differential: lymphocytes | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology Differential: Monocytes | Change from baseline in Hematology differential: monocytes | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Hematology Differential: Neutrophils | Change from baseline in Hematology differential: neutrophils | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Ferritin | Change from baseline in Biochemistry: Ferritin | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Glucose | Change from baseline in Biochemistry: Glucose | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: C-reactive Protein | Change from baseline in Biochemistry: C-reactive protein | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Sodium | Change from baseline in Biochemistry: Sodium | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Potassium | Change from baseline in Biochemistry: Potassium | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Chloride | Change from baseline in Biochemistry: Chloride | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Calcium | Change from baseline in Biochemistry: Calcium | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Magnesium | Change from baseline in Biochemistry: Magnesium | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Phosphate | Change from baseline in Biochemistry: Phosphate | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Aspartate Aminotransferase (AST) | Change from baseline in Biochemistry: Aspartate aminotransferase (AST) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Alanine Aminotransferase (ALT) | Change from baseline in Biochemistry: alanine aminotransferase (ALT) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Gamma-glutamyl Transpeptidase (γGT) | Change from baseline in Biochemistry: gamma-glutamyl transpeptidase (γGT) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Alkaline Phosphatase (ALP) | Change from baseline in Biochemistry: alkaline phosphatase (ALP) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Lactate Dehydrogenase (LDH) | Change from baseline in Biochemistry: lactate dehydrogenase (LDH) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Bilirubin | Change from baseline in Biochemistry: bilirubin (total, direct and indirect) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Triglycerides | Change from baseline in Biochemistry: triglycerides | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Cholesterol | Change from baseline in Biochemistry: cholesterol (total and high-density lipoprotein [HDL]) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Albumin | Change from baseline in Biochemistry: Albumin | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Creatinine | Change from baseline in Biochemistry: Creatinine | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Biochemistry: Urea | Change from baseline in Biochemistry: Urea | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Activated Partial Thromboplastin (aPTT) | Change from baseline in Coagulation: activated partial thromboplastin (aPTT) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Prothrombin Time (PT) | Change from baseline in Coagulation: prothrombin time (PT) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Glucose | Change from baseline in Urinalysis: Glucose | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Blood | Change from baseline in Urinalysis: Blood | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Protein | Change from baseline in Urinalysis: Protein | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Leucocytes | Change from baseline in Urinalysis: Leucocytes | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Ketones | Change from baseline in Urinalysis: Ketones | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: pH | Change from baseline in Urinalysis: pH | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in Urinalysis: Specific Gravity | Change from baseline in Urinalysis: specific gravity | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Number of Subjects With Change in Donor Chimerism | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change in HLA Antibodies | Change from baseline in HLA antibodies against donor cells | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Change From Baseline in Minimal Residual Disease (MRD) | Only in patients presenting malignant disease | From HSCT (Day 0) up to study termination, approximately 46 weeks |
| Randwick |
| 2031 |
| Australia |
| CHU Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| The Rambam Academic Hospital | Haifa | 31096 | Israel |
| Hadassah Hebrew University | Jerusalem | 91120 | Israel |
| COMPLETED |
|
| NOT COMPLETED |
|
| Monitoring - Primary GF (Day 1 to 42) |
|
| Monitoring - Secondary GF (Up to Day 98) |
|
| Follow-up (3 Years From HSCT) |
|
|
No patients were enrolled in the emapalumab arm.
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| ID | Title | Description |
|---|---|---|
| BG000 | Emapalumab Treated | Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment) |
| BG001 | Non-emapalumab Treated | Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Underlying disease: Aplastic anemia | Count of Participants | Participants |
| ||||||||||||||||||
| Graft - Type of conditioning | Engraftment was defined as absolute neutrophils count (ANC) >= 500 cells/microliter (mcL) for 3 consecutive measures and donor chimerism above 60% or ANC >= 500 cells/mcL for 3 consecutive measures and 2 consecutive measures of donor chimerism above 60% or 7 days of platelets above 20000 cells/mcL without transfusion. | Count of Participants | Participants |
| |||||||||||||||||
| Graft - Source of stem cells | Count of Participants | Participants |
| ||||||||||||||||||
| Graft - Donor type | Count of Participants | Participants |
| ||||||||||||||||||
| Number of transplanted cells | Median | Full Range | million cells |
| |||||||||||||||||
| ABO incompatibility | Count of Participants | Participants |
| ||||||||||||||||||
| Mean CXCL9 concentration (prior to HSCT) | Median | Full Range | pg/mL |
|
| 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 | CXCL9 in Serum | Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9) | No efficacy data was collected since no subject received study treatment. | Posted | From start of treatment to EoS Visit, up to 34 weeks |
|
| ||||||||||||||||||||||
| Primary | Primary Graft Failure (GF) | Number of participants with primary graft failure (GF) | No efficacy data was collected since no subject received study treatment. | Posted | From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Primary | Secondary GF | Number of participants with secondary GF | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Free & Total Interferon Gamma (IFNγ) in Serum | Serum concentration of free and total Interferon gamma (IFNγ) | No efficacy data was collected since no subject received study treatment. | Posted | From start of treatment to EoS Visit, up to 34 weeks |
|
| ||||||||||||||||||||||
| Secondary | Emapalumab in Serum - Peak | Peak emapalumab serum concentration | No patients enrolled in the emapalumab arm. | Posted | From start of treatment to EoS, up to 34 weeks |
|
| ||||||||||||||||||||||
| Secondary | Ctrough (Emapalumab) | Concentration just before administration | No patients enrolled in the emapalumab arm. | Posted | From start of treatment to EoS, up to 34 weeks |
|
| ||||||||||||||||||||||
| Secondary | Exploratory Biomarkers: Ferritin | Ferritin - serum concentration | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | ADA and nAbs | Number of participants developing antibodies against emapalumab (antidrug antibodies [ADA]) and Neutralizing antibodies (nAb) | No patients enrolled in the emapalumab arm. | Posted | From Start of treatment until EoS, up to 34 weeks |
|
| ||||||||||||||||||||||
| Secondary | Number of Participants With Mixed Donor Chimerism <10% and <20% | Based on unselected leukocytes and based on sorted T cells | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||
| Secondary | Number of Participants Receiving Thrombopoietic Agents, Stem Cell Boost, Donor Lymphocyte Infusion (DLI) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Number of Participants Receiving a Second Allogeneic HSCT | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Number of Participants With Poor Graft Function | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Number of Participants With Event Free Engraftment | defined as absence of GF or graft support | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||
| Secondary | Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD) | (grade I to IV) | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||
| Secondary | Biomarker Levels, in Particular IFNy and CXCL9, as Predictors of Primary and/or Secondary Graft Failure or Acute and/or Chronic GvHD | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Engraftment Syndrome | Number of participants with engraftment syndrome | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||
| Secondary | Number of Participants With Endothelial Complications | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Number of Participants With Relapse, Defined as Cumulative Incidence of Reoccurring Underlying Disease | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| |||||||||||||||||||||||
| Secondary | Survival Rate | Number of patients alive at the end of study. | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||
| Secondary | Change in Body Temperature | Change from baseline in body temperature | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Heart Rate | Change from baseline in heart rate | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Blood Pressure | Change from baseline systolic and diastolic blood pressure | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Body Weight | Change from baseline in body weight | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology: Red Blood Cells (RBC) | Change from baseline in Hematology: red blood cells (RBC) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology: Hematocrit | Change from baseline in Hematology: hematocrit | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology: Hemoglobin | Change from baseline in Hematology: hemoglobin | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology: Platelets | Change from baseline in Hematology: platelets | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology: White Blood Cells (WBC) | Change from baseline in Hematology: white blood cells (WBC) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology Differential: Lymphocytes | Change from baseline in Hematology differential: lymphocytes | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology Differential: Monocytes | Change from baseline in Hematology differential: monocytes | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Hematology Differential: Neutrophils | Change from baseline in Hematology differential: neutrophils | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: Ferritin | Change from baseline in Biochemistry: Ferritin | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: Glucose | Change from baseline in Biochemistry: Glucose | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: C-reactive Protein | Change from baseline in Biochemistry: C-reactive protein | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: Sodium | Change from baseline in Biochemistry: Sodium | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: Potassium | Change from baseline in Biochemistry: Potassium | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
| ||||||||||||||||||||||
| Secondary | Change in Biochemistry: Chloride | Change from baseline in Biochemistry: Chloride | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Calcium | Change from baseline in Biochemistry: Calcium | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Magnesium | Change from baseline in Biochemistry: Magnesium | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Phosphate | Change from baseline in Biochemistry: Phosphate | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Aspartate Aminotransferase (AST) | Change from baseline in Biochemistry: Aspartate aminotransferase (AST) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Alanine Aminotransferase (ALT) | Change from baseline in Biochemistry: alanine aminotransferase (ALT) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
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| Secondary | Change in Biochemistry: Gamma-glutamyl Transpeptidase (γGT) | Change from baseline in Biochemistry: gamma-glutamyl transpeptidase (γGT) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Alkaline Phosphatase (ALP) | Change from baseline in Biochemistry: alkaline phosphatase (ALP) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Lactate Dehydrogenase (LDH) | Change from baseline in Biochemistry: lactate dehydrogenase (LDH) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Bilirubin | Change from baseline in Biochemistry: bilirubin (total, direct and indirect) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Triglycerides | Change from baseline in Biochemistry: triglycerides | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
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| Secondary | Change in Biochemistry: Cholesterol | Change from baseline in Biochemistry: cholesterol (total and high-density lipoprotein [HDL]) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Albumin | Change from baseline in Biochemistry: Albumin | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Creatinine | Change from baseline in Biochemistry: Creatinine | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Biochemistry: Urea | Change from baseline in Biochemistry: Urea | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
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| Secondary | Activated Partial Thromboplastin (aPTT) | Change from baseline in Coagulation: activated partial thromboplastin (aPTT) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Prothrombin Time (PT) | Change from baseline in Coagulation: prothrombin time (PT) | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: Glucose | Change from baseline in Urinalysis: Glucose | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
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| Secondary | Change in Urinalysis: Blood | Change from baseline in Urinalysis: Blood | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: Protein | Change from baseline in Urinalysis: Protein | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: Leucocytes | Change from baseline in Urinalysis: Leucocytes | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: Ketones | Change from baseline in Urinalysis: Ketones | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: pH | Change from baseline in Urinalysis: pH | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in Urinalysis: Specific Gravity | Change from baseline in Urinalysis: specific gravity | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Number of Subjects With Change in Donor Chimerism | No patients enrolled in the emapalumab arm. | Posted | Count of Participants | Participants | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change in HLA Antibodies | Change from baseline in HLA antibodies against donor cells | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
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| Secondary | Change From Baseline in Minimal Residual Disease (MRD) | Only in patients presenting malignant disease | No efficacy data was collected since no subject received study treatment. | Posted | From HSCT (Day 0) up to study termination, approximately 46 weeks |
|
|
From Day 1 up to study termination, approximately 46 weeks
No patients were enrolled in the emapalumab arm.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Emapalumab Treated | Patients treated with emapalumab | 0 | 0 | 0 | 0 | 0 | 0 |
| EG001 | Non-emapalumab Treated | Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment) | 0 | 2 | 0 | 2 | 2 | 2 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Nephropathy toxic | Renal and urinary disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Graft versus host disease in skin | Immune system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hypervolaemia | Metabolism and nutrition disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Perineal abscess | Infections and infestations | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Engraftment syndrome | Immune system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Pityriasis rosea | Skin and subcutaneous tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Salmonella test positive | Investigations | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Hypertrichosis | Skin and subcutaneous tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Allergy to immunoglobulin therapy | Immune system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Ingrown toe nail | Skin and subcutaneous tissue disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Chemokine increase | Investigations | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA V24.1 Sep 202 | Systematic Assessment |
|
The study was prematurely terminated with only 2 patients enrolled. Both patients were in the non-emapalumab treated arm, i.e. no patients received study treatment (emapalumab). Consequently, the majority of analyses were not performed and no conclusion on the efficacy of emapalumab on this patient population could be drawn from the study.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Emmanuel Monnet | Sobi | +41225519156 | Emmanuel.Monnet@sobi.com |
| Sep 25, 2023 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| C000644327 | Emapalumab |
Not provided
Not provided
Not provided
| >=65 years |
|
| Unknown or Not Reported |
|
| Reduced Intensity Conditioning (RIC) |
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