55 Participants Needed

Emapalumab for Aplastic Anemia

Recruiting at 6 trial locations
JO
JJ
Overseen ByJaap Jan Boelens, MD, PhD
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: Memorial Sloan Kettering Cancer Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Who Is on the Research Team?

JO

Joseph H. Oved, M.D.

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

This trial is for children and young adults under 25 with newly diagnosed severe Aplastic Anemia (sAA), showing very low blood cell counts and bone marrow activity. They must be able to handle the treatment, have not received prior sAA treatments, and can't have leukemia or inherited bone marrow failure.

Inclusion Criteria

I was diagnosed before turning 25.
I am being tested for severe aplastic anemia due to low blood counts and a less active bone marrow.
I can safely receive emapalumab and immunosuppressive therapy according to my hospital's standards.
See 2 more

Exclusion Criteria

I have a genetic condition that affects my bone marrow.
I have leukemia or myelodysplastic syndromes (MDS).
Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests
See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Emapalumab for 6 weeks, followed by either standard IST or HCT

6 weeks

Standard IST

Participants receive standard IST with equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab

Duration not specified

HCT

Participants undergo a standard hematopoietic stem cell transplant (HCT)

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Emapalumab
Trial Overview The study tests if emapalumab given early on can improve the outcomes of standard therapies for sAA in pediatric patients. It aims to see if this approach helps in planning more effective treatment strategies.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Emapalumab, then Standard ISTExperimental Treatment1 Intervention
Group II: Emapalumab, then HCTExperimental Treatment1 Intervention

Emapalumab is already approved in United States for the following indications:

🇺🇸
Approved in United States as Gamifant for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials
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Recruited
602,000+
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