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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
No Placebo-Only Group

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:
Select...
I have leukemia or myelodysplastic syndromes (MDS).
Select...
I currently have an infection that is not under control.
Select...
I have been treated for severe aplastic anemia before.
Select...
I have a genetic condition that affects my bone marrow.
Select...
I've completed all tests for severe aplastic anemia and have a donor ready for a stem cell transplant.

Timeline

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
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
7%
Condition aggravated
7%
Nausea
7%
Epstein-Barr virus infection
7%
Rhinovirus infection
7%
Rash erythematous
7%
Enterovirus infection
7%
Viral infection
7%
Adverse event following immunisation
7%
Arthritis
7%
Osteonecrosis
7%
Dysphonia
7%
Oedema peripheral
7%
Pyrexia
7%
Arthralgia
7%
Oropharyngeal pain
7%
Urticaria
7%
Lymphopenia
7%
Skin laceration
7%
Rash papular
7%
Rectal prolapse
7%
Cestode infection
7%
Blood alkaline phosphatase increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Enrolled-06 Cohort
Enrolled-CU Cohort
Enrolled-04 Cohort

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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

Find a Location

Who is running the clinical trial?

Memorial Sloan Kettering Cancer CenterLead Sponsor
1,965 Previous Clinical Trials
596,955 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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