Aplastic AnemiaPhase 2NCT06430788
A Study of Emapalumab for Pediatric Aplastic Anemia
A Phase 2 study testing Emapalumab in people with Aplastic Anemia and Cytopenia, run by Memorial Sloan Kettering Cancer Center at 6 sites in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 0 to 25 yearsHealthy volunteers can take part
- Conditions the study is for
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- What is being tested?
- Emapalumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 6 sitesUnited States
- Study dates
- 21 May 2024to 21 May 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 25 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Patients undergoing workup for suspected newly diagnosed sAA:
- Patients with severe cytopenias and a hypocellular marrow concerning for sAA
- Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:
- Marrow Cellularity: <25%, or 25-50% with <30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): <500 x 10^9/L Platelets: <20 x 10^9/L Absolute Reticulocyte Count: <60 x 10^9/L
- Patients that do not have evidence of leukemia or MDS
- Patients < 25 years of age at time of diagnosis
- Able to tolerate emapalumab and IST (with standard institutional organ function criteria)
Who cannot join
- Uncontrolled infection at presentation.
- Patients who have undergone previous treatment for sAA.
- Patients with known inherited bone marrow failure
- Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
- Patients with leukemia or MDS
- Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center (Data collection only)
Cincinnati, Ohio, 45229
Anthony Sabulski, MD, 513-636-3200, anthony.sabulski@cchmc.org
- Recruiting
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, Wisconsin, 53226
Larisa Broglie, MD, 414-266-2420
- Not yet recruiting
Medical College of Wisconsin (Data Collection AND Data Analysis)
Milwaukee, Wisconsin, 53226
Larisa Broglie, MD, 414-266-2420
- Recruiting
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, 10065
Andromachi Scaradavou, MD, 1-833-MSK-KIDS
- Recruiting
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Philadelphia, Pennsylvania, 19104
Tim Olson, MD, PhD, 800-879-2467
- Recruiting
Virginia Commonwealth University (Data Collection Only )
Richmond, Virginia, 23219
Joe Laver, MD, MHA, 804-828-9213
About this study
Official title: Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
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
- Conditions
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- Being tested
- Emapalumab
- Keywords
- pediatric aplastic anemia, aplastic anemia, cytopenia, hypocellular marrow, Emapalumab, Memorial Sloan Kettering Cancer Center, 23-278
- Medical subject headings
- Anemia, Aplastic, Cytopenia, Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmapalumabBiological
Also called Gamifant
Emapalumab is an interferon gamma (IFNγ) blocking antibody
| Study group | Role | What they receive |
|---|---|---|
| Emapalumab, then Standard IST | Experimental | 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 Receives: Biological: Emapalumab |
| Emapalumab, then HCT | Experimental | Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT). Receives: Biological: Emapalumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Response
The primary objective of the study is to assess the efficacy of early upfront emapalumab on hematologic recovery within 6 weeks of starting therapy after a new diagnosis of Aplastic Anemia. Response will be determined by blood count.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2024
- Study start21 May 2024
- Primary completion21 May 2029 (estimated)
- Study completion21 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andromachi Scaradavou, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06430788
- Sponsor's protocol ID
- 23-278
- Other IDs
- FD-R-008175
- Status last verified
- Dec 2025
- First submitted
- 21 May 2024
- Last updated
- 17 Dec 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06430788
- Acronym / sponsor ID
- 23-278
- Conditions
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- Keywords
- pediatric aplastic anemia, aplastic anemia, cytopenia, hypocellular marrow, Emapalumab, Memorial Sloan Kettering Cancer Center, 23-278
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- 21 May 2024 (actual)
- Primary completion
- 21 May 2029 (estimated)
- Study completion
- 21 May 2029 (estimated)
- First posted
- 28 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Emapalumab, then Standard IST | Experimental | Biological: EmapalumabParticipants 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 |
| Emapalumab, then HCT | Experimental | Biological: EmapalumabParticipants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT). |
Who may qualify
- Ages
- 0 to 25 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Patients undergoing workup for suspected newly diagnosed sAA:
- Marrow Cellularity: <25%, or 25-50% with <30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): <500 x 10^9/L Platelets: <20 x 10^9/L Absolute Reticulocyte Count: <60 x 10^9/L
- Patients that do not have evidence of leukemia or MDS
- Patients < 25 years of age at time of diagnosis
- Able to tolerate emapalumab and IST (with standard institutional organ function criteria)
Key exclusion criteria (6 listed)
- Uncontrolled infection at presentation.
- Patients who have undergone previous treatment for sAA.
- Patients with known inherited bone marrow failure
- Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
- Patients with leukemia or MDS
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmapalumabAlso: GamifantEmapalumab is an interferon gamma (IFNγ) blocking antibody | Biological | Emapalumab, then HCT, Emapalumab, then Standard IST |
Primary outcome
Best Response
Time frame: 6 weeks
The primary objective of the study is to assess the efficacy of early upfront emapalumab on hematologic recovery within 6 weeks of starting therapy after a new diagnosis of Aplastic Anemia. Response will be determined by blood count.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2025.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Related trials for Aplastic Anemia
RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andromachi Scaradavou, MD principal investigator
Memorial Sloan Kettering Cancer Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Emapalumab in people with Aplastic Anemia and Cytopenia, run by Memorial Sloan Kettering Cancer Center at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Andromachi Scaradavou, MD (1-833-MSK-KIDS, ScaradaA@mskcc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best Response (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025.