Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
35 participants
INTERVENTIONAL
2024-05-21
2029-05-21
Brief Summary
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Funding Source- FDA OOPD
Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Emapalumab, then Standard IST
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will receive standard IST with drugs called equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab
Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Emapalumab, then HCT
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Interventions
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Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
0 Years
25 Years
ALL
Yes
Sponsors
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Memorial Sloan Kettering Cancer Center
OTHER
Responsible Party
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Principal Investigators
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Andromachi Scaradavou, MD
Role: PRINCIPAL_INVESTIGATOR
Memorial Sloan Kettering Cancer Center
Locations
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Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, United States
Cincinnati Children's Hospital Medical Center (Data collection only)
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Philadelphia, Pennsylvania, United States
Virginia Commonwealth University (Data Collection Only )
Richmond, Virginia, United States
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, Wisconsin, United States
Medical College of Wisconsin (Data Collection AND Data Analysis)
Milwaukee, Wisconsin, United States
Countries
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Central Contacts
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Facility Contacts
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Andromachi Scaradavou, MD
Role: primary
Tim Olson, MD, PhD
Role: primary
Joe Laver, MD, MHA
Role: primary
Larisa Broglie, MD
Role: primary
Larisa Broglie, MD
Role: primary
Related Links
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Memorial Sloan Kettering Cancer Center
Other Identifiers
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FD-R-008175
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
23-278
Identifier Type: -
Identifier Source: org_study_id