A Study of Emapalumab for Pediatric Aplastic Anemia

Part of paid clinical trials in New York, New York.

Sponsor
Memorial Sloan Kettering Cancer Center
Study ID
NCT06430788
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
0 Years - 25 Years
Healthy Volunteers
Accepted

Interventions

  • Emapalumab — BIOLOGICAL
    Emapalumab is an interferon gamma (IFNγ) blocking antibody

Study Details

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. Funding Source- FDA OOPD

Key Dates

Start date
May 21, 2024
Status verified
Dec 2025
Primary completion
May 21, 2029
Completion
May 21, 2029

Study Design

Enrollment
35 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: 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
  • Experimental: 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).

Primary Outcome Measure

Best Response [ Time Frame: 6 weeks ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Memorial Sloan Kettering Cancer Center (All Protocol Activities)New YorkNew York10065
Andromachi Scaradavou, MD
1-833-MSK-KIDS
Cincinnati Children's Hospital Medical Center (Data collection only)CincinnatiOhio45229
Anthony Sabulski, MD
[email protected]
513-636-3200
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)PhiladelphiaPennsylvania19104
Tim Olson, MD, PhD
800-879-2467
Virginia Commonwealth University (Data Collection Only )RichmondVirginia23219
Joe Laver, MD, MHA
804-828-9213
Children's Hospital of Wisconsin (Data Collection Only)MilwaukeeWisconsin53226
Larisa Broglie, MD
414-266-2420
Medical College of Wisconsin (Data Collection AND Data Analysis)MilwaukeeWisconsin53226
Larisa Broglie, MD
414-266-2420

Find similar trials in New York, NY

By research site
Memorial Sloan Kettering Cancer Center (All Protocol Activities)· New York, NYCincinnati Children's Hospital Medical Center (Data collection only)· Cincinnati, OHChildren's Hospital of Philadelphia (Data Collection AND Specimen Analysis)· Philadelphia, PAVirginia Commonwealth University (Data Collection Only )· Richmond, VAChildren's Hospital of Wisconsin (Data Collection Only)· Milwaukee, WIMedical College of Wisconsin (Data Collection AND Data Analysis)· Milwaukee, WI

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