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Sickle Cell Disease Transplant Study with Daratumumab

This study is for children and adolescents (ages 2-25) with sickle cell disease who are receiving a stem cell transplant from an HLA-identical sibling donor. It's designed for patients who have antibodies against the donor's red blood cells, which can cause problems after transplant. Researchers are testing if a medicine called daratumumab, given before the transplant, can prevent these issues. The transplant also involves alemtuzumab, low-dose total body irradiation (a type of radiation therapy), and sirolimus. The main goal is to see if patients who receive daratumumab have a good event-free survival (meaning they don't experience certain complications) one year after transplant, similar to patients without these antibodies. The study plans to enroll 12 participants.

Study design
This is an interventional study, meaning participants will receive specific treatments. It aims to enroll 12 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary outcome of event-free survival will be measured one year after the transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06358638

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody

Recruiting
PHASE2Ages 2–25InterventionalTreatment
Children's National Research Institute
~12 participants
Updated 2025-09-26 on ClinicalTrials.gov
What's tested:DaratumumabAlemtuzumabSirolimusTotal Body Irradiation

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To determine the event-free survival of children and adolescents with SCD undergoing nonmyeloablative HCT who received 4 doses of pre-HCT daratumumab for donor-directed red blood cell antibodies.
Measured over 1-year post-transplant

Conditions

Sickle Cell Disease

Explore related trials

Sickle Cell Disease

Where it's being run

1 sites across 1 states
District of Columbia1

Study leadership

  • Robert Nickel, MD · PRINCIPAL_INVESTIGATOR · Children's National Research Institute

Who to contact

Robert Nickel, MD
[email protected]
202-476-3122
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients with SCD age 2-24.99 years who have a healthy HLA-identical sibling donor with major ABO incompatibility OR patients with RBC alloantibodies against other donor RBC antigens.
Patients must have an absolute neutrophil count of 1 x 109/L and a platelet count of 100 x 109/L.
Lansky/Karnofsky score of, at least, 70.
History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique measured at a minimum of two separate occasions.
Progression of CNS vasculopathy on MRA determined to be secondary to SCD.
History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated inversion recovery T2-weighted images).
History of two or more episodes of Acute Chest Syndrome (ACS) in lifetime.
History of three or more SCD pain events requiring treatment with an opiate or IV pain medication in lifetime.
History of any hospitalization for a complication secondary to SCD (does NOT include empiric hospitalizations for fever only).
History of two or more episodes of priapism.
Administration of regular RBC transfusions (≥8 transfusions episodes in the previous 12 months).
At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.
Clinically significant neurologic event (overt stroke).
History of two or more episodes of ACS in the 2-year period preceding enrollment.
History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in the 1-year period preceding enrollment.
History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.
History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).
Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months)
At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.

Exclusion

Life expectancy less than 6 month
Pregnant or breastfeeding patients.
Infectious Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity for HIV and patients with active or resolved Hepatitis B or C determined by serology and/or NAAT are excluded.
Liver: Direct (conjugated) bilirubin \> 1.5 mg/dL. Transaminases \>5x upper limit of normal for age.
Cardiac: Left ventricular shortening fraction \<25% or ejection fraction \<50% by ECHO. Uncontrolled cardiac arrhythmia.
Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2.
Pulmonary function: Diffusion capacity of carbon monoxide (DLCO) \<35% (adjusted for hemoglobin). Baseline oxygen saturation \<94% at rest or PaO2 \<70. Known moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification.
Heme: Available, medically suitable, and equivalent HLA-matched sibling donor, who does not have major ABO incompatibility or express RBC antigens against which the patient is alloimmunized.

What this trial measures

  • To determine the event-free survival of children and adolescents with SCD undergoing nonmyeloablative HCT who received 4 doses of pre-HCT daratumumab for donor-directed red blood cell antibodies.1-year post-transplant

    Event-free survival rate at 1 year with events including death, graft failure (donor myeloid chimerism \<10% or second HCT), grade II-IV GVHD, and serious pure red blood cell aplasia (persistent reticulocytopenia and need for red blood cell transfusion support after day +100).