Emapalumab for Aplastic Anemia

Recruiting at 5 trial locations

Overseen By Jaap Jan Boelens, MD, PhD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: Uncontrolled infection, Previous sAA treatment, Inherited bone marrow failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

55 Participants Needed

Trial Summary

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.

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.

Research Team

Joseph H. Oved, M.D.

Principal Investigator

Memorial Sloan Kettering Cancer Center

Eligibility Criteria

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.

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

Timeline

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

Treatment Details

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.

Participant Groups

Group I: Emapalumab, then Standard IST Experimental Treatment 1 Intervention

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

Group II: Emapalumab, then HCT Experimental Treatment 1 Intervention

Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).

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

Approved in United States as Gamifant for:

• Primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+