Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04731298
Registration number
NCT04731298
Ethics application status
Date submitted
13/11/2020
Date registered
29/01/2021
Date last updated
28/12/2023
Titles & IDs
Public title
Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.
Query!
Scientific title
An Open Label, Single Arm, Multicentre, Proof of Concept, Phase 2 Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After Allogeneic Hematopoietic Stem Cell Transplantation
Query!
Secondary ID [1]
0
0
NI-0501-12
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Graft Failure
0
0
Query!
Condition category
Condition code
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Emapalumab
Experimental: Emapalumab - 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.
Treatment: Drugs: Emapalumab
Emapalumab is a fully human immunoglobulin G1 (IgG1) anti-IFN? monoclonal antibody that binds to and neutralizes IFN?. Emapalumab binds to both soluble and receptor (IFN?R1)-bound forms of IFN?.
Emapalumab is in development for treatment of primary and secondary HLH. The benefit expected from the targeted neutralization of IFN? 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.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
CXCL9 in Serum
Query!
Assessment method [1]
0
0
Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9)
Query!
Timepoint [1]
0
0
From start of treatment to EoS Visit, up to 34 weeks
Query!
Primary outcome [2]
0
0
Primary Graft Failure (GF)
Query!
Assessment method [2]
0
0
Number of participants with primary graft failure (GF)
Query!
Timepoint [2]
0
0
From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks
Query!
Primary outcome [3]
0
0
Secondary GF
Query!
Assessment method [3]
0
0
Number of participants with secondary GF
Query!
Timepoint [3]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [1]
0
0
Free & Total Interferon Gamma (IFN?) in Serum
Query!
Assessment method [1]
0
0
Serum concentration of free and total Interferon gamma (IFN?)
Query!
Timepoint [1]
0
0
From start of treatment to EoS Visit, up to 34 weeks
Query!
Secondary outcome [2]
0
0
Emapalumab in Serum - Peak
Query!
Assessment method [2]
0
0
Peak emapalumab serum concentration
Query!
Timepoint [2]
0
0
From start of treatment to EoS, up to 34 weeks
Query!
Secondary outcome [3]
0
0
Ctrough (Emapalumab)
Query!
Assessment method [3]
0
0
Concentration just before administration
Query!
Timepoint [3]
0
0
From start of treatment to EoS, up to 34 weeks
Query!
Secondary outcome [4]
0
0
Exploratory Biomarkers: Ferritin
Query!
Assessment method [4]
0
0
Ferritin - serum concentration
Query!
Timepoint [4]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [5]
0
0
ADA and nAbs
Query!
Assessment method [5]
0
0
Number of participants developing antibodies against emapalumab (antidrug antibodies \[ADA\]) and Neutralizing antibodies (nAb)
Query!
Timepoint [5]
0
0
From Start of treatment until EoS, up to 34 weeks
Query!
Secondary outcome [6]
0
0
Number of Participants With Mixed Donor Chimerism <10% and <20%
Query!
Assessment method [6]
0
0
Based on unselected leukocytes and based on sorted T cells
Query!
Timepoint [6]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [7]
0
0
Number of Participants Receiving Thrombopoietic Agents, Stem Cell Boost, Donor Lymphocyte Infusion (DLI)
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [8]
0
0
Number of Participants Receiving a Second Allogeneic HSCT
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [9]
0
0
Number of Participants With Poor Graft Function
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [10]
0
0
Number of Participants With Event Free Engraftment
Query!
Assessment method [10]
0
0
defined as absence of GF or graft support
Query!
Timepoint [10]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [11]
0
0
Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD)
Query!
Assessment method [11]
0
0
(grade I to IV)
Query!
Timepoint [11]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [12]
0
0
Biomarker Levels, in Particular IFNy and CXCL9, as Predictors of Primary and/or Secondary Graft Failure or Acute and/or Chronic GvHD
Query!
Assessment method [12]
0
0
Query!
Timepoint [12]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [13]
0
0
Engraftment Syndrome
Query!
Assessment method [13]
0
0
Number of participants with engraftment syndrome
Query!
Timepoint [13]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [14]
0
0
Number of Participants With Endothelial Complications
Query!
Assessment method [14]
0
0
Query!
Timepoint [14]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [15]
0
0
Number of Participants With Relapse, Defined as Cumulative Incidence of Reoccurring Underlying Disease
Query!
Assessment method [15]
0
0
Query!
Timepoint [15]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [16]
0
0
Survival Rate
Query!
Assessment method [16]
0
0
Number of patients alive at the end of study.
Query!
Timepoint [16]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [17]
0
0
Change in Body Temperature
Query!
Assessment method [17]
0
0
Change from baseline in body temperature
Query!
Timepoint [17]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [18]
0
0
Change in Heart Rate
Query!
Assessment method [18]
0
0
Change from baseline in heart rate
Query!
Timepoint [18]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [19]
0
0
Change in Blood Pressure
Query!
Assessment method [19]
0
0
Change from baseline systolic and diastolic blood pressure
Query!
Timepoint [19]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [20]
0
0
Change in Body Weight
Query!
Assessment method [20]
0
0
Change from baseline in body weight
Query!
Timepoint [20]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [21]
0
0
Change in Hematology: Red Blood Cells (RBC)
Query!
Assessment method [21]
0
0
Change from baseline in Hematology: red blood cells (RBC)
Query!
Timepoint [21]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [22]
0
0
Change in Hematology: Hematocrit
Query!
Assessment method [22]
0
0
Change from baseline in Hematology: hematocrit
Query!
Timepoint [22]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [23]
0
0
Change in Hematology: Hemoglobin
Query!
Assessment method [23]
0
0
Change from baseline in Hematology: hemoglobin
Query!
Timepoint [23]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [24]
0
0
Change in Hematology: Platelets
Query!
Assessment method [24]
0
0
Change from baseline in Hematology: platelets
Query!
Timepoint [24]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [25]
0
0
Change in Hematology: White Blood Cells (WBC)
Query!
Assessment method [25]
0
0
Change from baseline in Hematology: white blood cells (WBC)
Query!
Timepoint [25]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [26]
0
0
Change in Hematology Differential: Lymphocytes
Query!
Assessment method [26]
0
0
Change from baseline in Hematology differential: lymphocytes
Query!
Timepoint [26]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [27]
0
0
Change in Hematology Differential: Monocytes
Query!
Assessment method [27]
0
0
Change from baseline in Hematology differential: monocytes
Query!
Timepoint [27]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [28]
0
0
Change in Hematology Differential: Neutrophils
Query!
Assessment method [28]
0
0
Change from baseline in Hematology differential: neutrophils
Query!
Timepoint [28]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [29]
0
0
Change in Biochemistry: Ferritin
Query!
Assessment method [29]
0
0
Change from baseline in Biochemistry: Ferritin
Query!
Timepoint [29]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [30]
0
0
Change in Biochemistry: Glucose
Query!
Assessment method [30]
0
0
Change from baseline in Biochemistry: Glucose
Query!
Timepoint [30]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [31]
0
0
Change in Biochemistry: C-reactive Protein
Query!
Assessment method [31]
0
0
Change from baseline in Biochemistry: C-reactive protein
Query!
Timepoint [31]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [32]
0
0
Change in Biochemistry: Sodium
Query!
Assessment method [32]
0
0
Change from baseline in Biochemistry: Sodium
Query!
Timepoint [32]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [33]
0
0
Change in Biochemistry: Potassium
Query!
Assessment method [33]
0
0
Change from baseline in Biochemistry: Potassium
Query!
Timepoint [33]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [34]
0
0
Change in Biochemistry: Chloride
Query!
Assessment method [34]
0
0
Change from baseline in Biochemistry: Chloride
Query!
Timepoint [34]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [35]
0
0
Change in Biochemistry: Calcium
Query!
Assessment method [35]
0
0
Change from baseline in Biochemistry: Calcium
Query!
Timepoint [35]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [36]
0
0
Change in Biochemistry: Magnesium
Query!
Assessment method [36]
0
0
Change from baseline in Biochemistry: Magnesium
Query!
Timepoint [36]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [37]
0
0
Change in Biochemistry: Phosphate
Query!
Assessment method [37]
0
0
Change from baseline in Biochemistry: Phosphate
Query!
Timepoint [37]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [38]
0
0
Change in Biochemistry: Aspartate Aminotransferase (AST)
Query!
Assessment method [38]
0
0
Change from baseline in Biochemistry: Aspartate aminotransferase (AST)
Query!
Timepoint [38]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [39]
0
0
Change in Biochemistry: Alanine Aminotransferase (ALT)
Query!
Assessment method [39]
0
0
Change from baseline in Biochemistry: alanine aminotransferase (ALT)
Query!
Timepoint [39]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [40]
0
0
Change in Biochemistry: Gamma-glutamyl Transpeptidase (?GT)
Query!
Assessment method [40]
0
0
Change from baseline in Biochemistry: gamma-glutamyl transpeptidase (?GT)
Query!
Timepoint [40]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [41]
0
0
Change in Biochemistry: Alkaline Phosphatase (ALP)
Query!
Assessment method [41]
0
0
Change from baseline in Biochemistry: alkaline phosphatase (ALP)
Query!
Timepoint [41]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [42]
0
0
Change in Biochemistry: Lactate Dehydrogenase (LDH)
Query!
Assessment method [42]
0
0
Change from baseline in Biochemistry: lactate dehydrogenase (LDH)
Query!
Timepoint [42]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [43]
0
0
Change in Biochemistry: Bilirubin
Query!
Assessment method [43]
0
0
Change from baseline in Biochemistry: bilirubin (total, direct and indirect)
Query!
Timepoint [43]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [44]
0
0
Change in Biochemistry: Triglycerides
Query!
Assessment method [44]
0
0
Change from baseline in Biochemistry: triglycerides
Query!
Timepoint [44]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [45]
0
0
Change in Biochemistry: Cholesterol
Query!
Assessment method [45]
0
0
Change from baseline in Biochemistry: cholesterol (total and high-density lipoprotein \[HDL\])
Query!
Timepoint [45]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [46]
0
0
Change in Biochemistry: Albumin
Query!
Assessment method [46]
0
0
Change from baseline in Biochemistry: Albumin
Query!
Timepoint [46]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [47]
0
0
Change in Biochemistry: Creatinine
Query!
Assessment method [47]
0
0
Change from baseline in Biochemistry: Creatinine
Query!
Timepoint [47]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [48]
0
0
Change in Biochemistry: Urea
Query!
Assessment method [48]
0
0
Change from baseline in Biochemistry: Urea
Query!
Timepoint [48]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [49]
0
0
Activated Partial Thromboplastin (aPTT)
Query!
Assessment method [49]
0
0
Change from baseline in Coagulation: activated partial thromboplastin (aPTT)
Query!
Timepoint [49]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [50]
0
0
Prothrombin Time (PT)
Query!
Assessment method [50]
0
0
Change from baseline in Coagulation: prothrombin time (PT)
Query!
Timepoint [50]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [51]
0
0
Change in Urinalysis: Glucose
Query!
Assessment method [51]
0
0
Change from baseline in Urinalysis: Glucose
Query!
Timepoint [51]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [52]
0
0
Change in Urinalysis: Blood
Query!
Assessment method [52]
0
0
Change from baseline in Urinalysis: Blood
Query!
Timepoint [52]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [53]
0
0
Change in Urinalysis: Protein
Query!
Assessment method [53]
0
0
Change from baseline in Urinalysis: Protein
Query!
Timepoint [53]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [54]
0
0
Change in Urinalysis: Leucocytes
Query!
Assessment method [54]
0
0
Change from baseline in Urinalysis: Leucocytes
Query!
Timepoint [54]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [55]
0
0
Change in Urinalysis: Ketones
Query!
Assessment method [55]
0
0
Change from baseline in Urinalysis: Ketones
Query!
Timepoint [55]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [56]
0
0
Change in Urinalysis: pH
Query!
Assessment method [56]
0
0
Change from baseline in Urinalysis: pH
Query!
Timepoint [56]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [57]
0
0
Change in Urinalysis: Specific Gravity
Query!
Assessment method [57]
0
0
Change from baseline in Urinalysis: specific gravity
Query!
Timepoint [57]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [58]
0
0
Number of Subjects With Change in Donor Chimerism
Query!
Assessment method [58]
0
0
Query!
Timepoint [58]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [59]
0
0
Change in HLA Antibodies
Query!
Assessment method [59]
0
0
Change from baseline in HLA antibodies against donor cells
Query!
Timepoint [59]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Secondary outcome [60]
0
0
Change From Baseline in Minimal Residual Disease (MRD)
Query!
Assessment method [60]
0
0
Only in patients presenting malignant disease
Query!
Timepoint [60]
0
0
From HSCT (Day 0) up to study termination, approximately 46 weeks
Query!
Eligibility
Key inclusion criteria
1. 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
2. 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:
* Receiving reduced intensity conditioning (RIC) or non-myeloablative conditioning (NMA) combined with a non-malignant disease or with a graft from Bone Marrow (BM)
* Ex vivo T cell depleted graft
* Graft from mismatched unrelated or haploidentical donor
* Graft from Umbilical Cord Blood (UCB)
3. Patients requiring allo-HSCT with the following underlying diseases:
* Malignant disease with high risk of GF, i.e. Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL) with primary induction failure, second partial remission or relapse; Chronic Myeloid Leukemia (CML) in blastic phase (circulating blast or blast above 5% in biopsy); Non Hodgkin and Hodgkin Lymphoma and multiple myeloma with primary induction failure, second partial remission or relapse, myelodysplastic syndromes (MDS) and myeloproliferative disorders (MPD) with splenomegaly, myelofibrosis with portal hypertension pre-transplant, MDS/MPD overlap syndromes
* Non-malignant hematological diseases (e.g. autoimmune and metabolic disorders, aplastic anemia, Sickle cell anemia, Fanconi anemia, Diamond-Blackfan anemia, thalassemia, osteopetrosis, Wiskott-Aldrich syndrome, severe combined immunodeficiency, Hemophagocytic lymphohistiocytosis and other immunoregulatory disorders)
4. Male and female patients
5. 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.
6. 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
Query!
Minimum age
1
Year
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
1. Pregnant (or planning to become pregnant) or lactating female patients
2. Body weight < 3 kg
3. Underlying malignant disease with Karnofsky/Lansky performance status equal or less than 40 or an Eastern Cooperative Oncology Group (ECOG) performance status equal or less than 3
4. Patients presenting CXCL9 levels 10 times above the upper limit of the 95% interval (CI) of the normal range (reported in the CXCL9 assay laboratory manual) within 24 hours prior to HSCT
5. Clinically manifested infections caused by typical and atypical Mycobacteria, Salmonella, Histoplasma capsulatum and Herpes Zoster on the day of HSCT
6. Active or clinical suspicion of latent tuberculosis
7. Concomitant diseases that in the opinion of the Investigator may interfere with the assessment of emapalumab safety or efficacy
8. Receipt of a Bacille Calmette-Guerin (BCG) vaccine within 3 months prior to HSCT
9. Receipt of a live or attenuated live (other than BCG) vaccine within 6 weeks prior to HSCT
10. Current or scheduled administration of therapies known to potentially trigger a cytokine release syndrome within 21 days from HSCT.
11. Patients having received IFN? during the last 2 weeks prior to HSCT and/or who require treatment with IFN?.
12. Patients having received emapalumab during the last 6 months prior to HSCT, unless it is known that emapalumab is no longer detectable.
13. Patients having received kinase inhibitors (Janus kinase inhibitors [JAKi] or bruton tyrosine kinase inhibitors [BTKi]) one week (or 5 half-lives whichever is greater) prior to HSCT.
14. Intolerance to antimicrobial and virus infection prophylaxis.
15. Hypersensitivity to emapalumab or any of the excipients.
16. Ongoing participation in an interventional trial or administration of any investigational drug (within 3 half-lives of the investigational drug) with the exception of interventional trials involving supportive care such as probiotics or antiemetics, graft manipulation, or use of new combinations or new dosing of conventional therapies for conditioning and prophylaxis pre-HSCT.
Query!
Study design
Purpose of the study
Prevention
Query!
Allocation to intervention
NA
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Other
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Stopped early
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
25/05/2021
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
21/04/2022
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
2
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Peter MacCallum Cancer Centre - Melbourne
Query!
Recruitment hospital [2]
0
0
Kids Cancer Centre Sydney Children's Hospital - Randwick
Query!
Recruitment postcode(s) [1]
0
0
3000 - Melbourne
Query!
Recruitment postcode(s) [2]
0
0
2031 - Randwick
Query!
Recruitment outside Australia
Country [1]
0
0
Canada
Query!
State/province [1]
0
0
Quebec
Query!
Country [2]
0
0
Israel
Query!
State/province [2]
0
0
Haifa
Query!
Country [3]
0
0
Israel
Query!
State/province [3]
0
0
Jerusalem
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Swedish Orphan Biovitrum
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Commercial sector/industry
Query!
Name [1]
0
0
PRA Health Sciences
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Other collaborator category [2]
0
0
Commercial sector/industry
Query!
Name [2]
0
0
Cytel Inc.
Query!
Address [2]
0
0
Query!
Country [2]
0
0
Query!
Other collaborator category [3]
0
0
Commercial sector/industry
Query!
Name [3]
0
0
Q2 Solutions
Query!
Address [3]
0
0
Query!
Country [3]
0
0
Query!
Other collaborator category [4]
0
0
Commercial sector/industry
Query!
Name [4]
0
0
ABF Pharmaceutical Services GmbH
Query!
Address [4]
0
0
Query!
Country [4]
0
0
Query!
Other collaborator category [5]
0
0
Commercial sector/industry
Query!
Name [5]
0
0
Cromsource
Query!
Address [5]
0
0
Query!
Country [5]
0
0
Query!
Other collaborator category [6]
0
0
Commercial sector/industry
Query!
Name [6]
0
0
BioMérieux
Query!
Address [6]
0
0
Query!
Country [6]
0
0
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFN? 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 (IFN?) 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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT04731298
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Tsila Zuckerman, Dr
Query!
Address
0
0
The Rambam Academic Hospital
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/98/NCT04731298/Prot_SAP_000.pdf
Statistical analysis plan
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/98/NCT04731298/Prot_SAP_000.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04731298
Download to PDF