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Monoclonal Antibodies

Emapalumab for Aplastic Anemia

Phase 2

Recruiting

Led By Joseph Oved, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients < 25 years of age at time of diagnosis

Patients that meet the definition for suspected sAA (Camitta Criteria) including marrow cellularity <25%, or 25-50% with <30% residual hematopoietic cells, peripheral cytopenias (at least 2 of 3) with ANC <500 x 10^9/L, platelets <20 x 10^9/L, and absolute reticulocyte count <60 x 10^9/L

Must not have

Patients with leukemia or MDS

Uncontrolled infection at presentation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 weeks

Awards & highlights

Summary

This trial aims to determine if giving emapalumab as an initial treatment can improve the treatment and outcomes for patients with aplastic anemia.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, emapalumab may cause reactions related to immune system activation such as fever, rash or fatigue, and could potentially affect organ function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I was diagnosed before turning 25.

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I have severe aplastic anemia based on specific blood and bone marrow tests.

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I do not have leukemia or myelodysplastic syndromes.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have leukemia or myelodysplastic syndromes (MDS).

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I currently have an infection that is not under control.

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I have been treated for severe aplastic anemia before.

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I have a genetic condition that affects my bone marrow.

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I've completed all tests for severe aplastic anemia and have a donor ready for a stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Best Response

Side effects data

From 2021 Phase 2 & 3 trial • 58 Patients • NCT02069899

29%

Viral upper respiratory tract infection

14%

Dental caries

14%

Cholelithiasis

14%

Headache

14%

Still's disease

Condition aggravated

Nausea

Epstein-Barr virus infection

Rhinovirus infection

Rash erythematous

Enterovirus infection

Viral infection

Adverse event following immunisation

Arthritis

Osteonecrosis

Dysphonia

Oedema peripheral

Pyrexia

Arthralgia

Oropharyngeal pain

Urticaria

Lymphopenia

Skin laceration

Rash papular

Rectal prolapse

Cestode infection

Blood alkaline phosphatase increased

100%

80%

60%

40%

20%

Study treatment Arm

Enrolled-06 Cohort

Enrolled-CU Cohort

Enrolled-04 Cohort

Trial Design

2Treatment groups

Experimental Treatment

Group I: Emapalumab, then Standard ISTExperimental Treatment1 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 HCTExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Emapalumab

2014

Completed Phase 3

~110

0 / 8Eligibility criteria met

Find a Location

Who is running the clinical trial?

Memorial Sloan Kettering Cancer CenterLead Sponsor

1,956 Previous Clinical Trials

595,536 Total Patients Enrolled

Joseph Oved, MDPrincipal InvestigatorMemorial Sloan Kettering Cancer Center

1 Previous Clinical Trials

39 Total Patients Enrolled

~37 spots leftby May 2029

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