Clinical trial NCT06430788: A Study of Emapalumab for Pediatric Aplastic Anemia
Official title: Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Aplastic Anemia
Cytopenia
Hypocellular Marrow
Keywords: pediatric aplastic anemia
aplastic anemia
cytopenia
hypocellular marrow
Emapalumab
Memorial Sloan Kettering Cancer Center
23-278
Lead sponsor: Memorial Sloan Kettering Cancer Center
Sponsor class: OTHER
First posted: 2024-05-28
Enrollment: 55
Sex: ALL
Minimum age (years): 0
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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


Eligibility criteria: Inclusion Criteria:

* 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)

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Best Response
timeFrame: 6 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center (All Protocol Activities)
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center (Data collection only)
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
state: Pennsylvania
city: Richmond
country: United States
facility: Virginia Commonwealth University (Data Collection Only )
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin (Data Collection AND Data Analysis)
state: Wisconsin
Study officials:
affiliation: Memorial Sloan Kettering Cancer Center
name: Joseph Oved, MD
role: PRINCIPAL_INVESTIGATOR
References: