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Trial Title:
A Study of Emapalumab for Pediatric Aplastic Anemia
NCT ID:
NCT06430788
Condition:
Aplastic Anemia
Cytopenia
Hypocellular Marrow
Conditions: Official terms:
Anemia
Anemia, Aplastic
Cytopenia
Conditions: Keywords:
pediatric aplastic anemia
aplastic anemia
cytopenia
hypocellular marrow
Emapalumab
Memorial Sloan Kettering Cancer Center
23-278
Study type:
Interventional
Study phase:
Phase 2
Overall status:
Recruiting
Study design:
Allocation:
Non-Randomized
Intervention model:
Sequential Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Biological
Intervention name:
Emapalumab
Description:
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Arm group label:
Emapalumab, then HCT
Arm group label:
Emapalumab, then Standard IST
Other name:
Gamifant
Summary:
The purpose of this study is to find out whether upfront emapalumab treatment can help in
sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard
treatment options.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
- Patients undergoing workup for suspected newly diagnosed sAA:
- Patients with severe cytopenias and a hypocellular marrow concerning for sAA
- Patients that meet the definition for suspected sAA (Camitta Criteria) as
follows:
Marrow Cellularity: <25%, or 25-50% with <30% residual hematopoietic cells Peripheral
cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): <500 x 10^9/L Platelets:
<20 x 10^9/L Absolute Reticulocyte Count: <60 x 10^9/L
- Patients that do not have evidence of leukemia or MDS
- Patients < 25 years of age at time of diagnosis
- Able to tolerate emapalumab and IST (with standard institutional organ function
criteria)
Exclusion Criteria:
- Uncontrolled infection at presentation.
- Patients who have undergone previous treatment for sAA.
- Patients with known inherited bone marrow failure
- Patient who has completed a full workup for sAA including having results back from
telomere testing, DEB and genetics (when applicable), as well as having an
appropriate willing and available donor and would otherwise be admitted for HSCT
within 2 weeks of enrolling on the trial
- Patients with leukemia or MDS
- Patient or parent or guardian unable to give informed consent or unable to comply
with the treatment protocol including research tests.
Gender:
All
Minimum age:
0 Years
Maximum age:
25 Years
Healthy volunteers:
Accepts Healthy Volunteers
Locations:
Facility:
Name:
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
Address:
City:
New York
Zip:
10065
Country:
United States
Status:
Recruiting
Contact:
Last name:
Joseph Oved, MD
Phone:
1-833-MSK-KIDS
Facility:
Name:
Cincinnati Children's Hospital Medical Center (Data collection only)
Address:
City:
Cincinnati
Zip:
45229
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Anthony Sabulski, MD
Phone:
513-636-3200
Email:
anthony.sabulski@cchmc.org
Facility:
Name:
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Address:
City:
Philadelphia
Zip:
19104
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Tim Olson, MD, PhD
Phone:
800-879-2467
Facility:
Name:
Virginia Commonwealth Univeristy (Data Collection Only )
Address:
City:
Richmond
Zip:
23219
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Joe Laver, MD, MHA
Phone:
804-828-9213
Facility:
Name:
Medical College of Wisconsin (Data Collection AND Data Analysis)
Address:
City:
Milwaukee
Zip:
53226
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Larisa Broglie, MD
Phone:
414-266-2420
Start date:
May 21, 2024
Completion date:
May 21, 2029
Lead sponsor:
Agency:
Memorial Sloan Kettering Cancer Center
Agency class:
Other
Source:
Memorial Sloan Kettering Cancer Center
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06430788
http://www.mskcc.org
