Actively Recruiting
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Led by Children's National Research Institute · Updated on 2025-09-26
12
Participants Needed
1
Research Sites
1586 weeks
Total Duration
On this page
Sponsors
C
Children's National Research Institute
Lead Sponsor
A
Alberta Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.
CONDITIONS
Official Title
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with sickle cell disease aged 2 to 24.99 years who have a healthy HLA-identical sibling donor with major ABO incompatibility or red blood cell alloantibodies against donor RBC antigens
- Absolute neutrophil count of at least 1 x 10^9/L
- Platelet count of at least 100 x 10^9/L
- Lansky or Karnofsky score of 70 or higher
- For hemoglobin SS and Sb20 thalassemia genotypes, at least one of: abnormal transcranial Doppler velocity 65200 cm/sec on two occasions, progression of CNS vasculopathy on MRA, history of cerebral infarction on MRI, two or more acute chest syndrome episodes, three or more pain events needing opioid or IV treatment, any hospitalization for SCD complications, two or more priapism episodes, regular red blood cell transfusions (8 or more in past 12 months), or two or more splenic sequestration episodes requiring transfusion or splenectomy
- For other sickle genotypes, at least one of: overt stroke, two or more acute chest syndrome episodes in past 2 years, three or more pain events needing opioid or IV treatment in past year, hospitalization for pain or acute chest syndrome while on hydroxyurea, two or more priapism episodes, regular red blood cell transfusions (8 or more in past 12 months), or two or more splenic sequestration episodes requiring transfusion or splenectomy
You will not qualify if you...
- Life expectancy less than 6 months
- Pregnant or breastfeeding
- Uncontrolled bacterial, viral, or fungal infections within 1 month before conditioning
- Febrile illness or suspected minor infection not resolved before conditioning
- Confirmed HIV infection or active/resolved Hepatitis B or C infection
- Direct bilirubin greater than 1.5 mg/dL
- Transaminases more than 5 times the upper limit of normal for age
- Left ventricular shortening fraction less than 25% or ejection fraction less than 50%
- Uncontrolled cardiac arrhythmia
- Estimated creatinine clearance less than 60 mL/min/1.73m2
- Diffusion capacity of carbon monoxide less than 35% adjusted for hemoglobin
- Baseline oxygen saturation less than 94% at rest or PaO2 less than 70
- Moderate or severe persistent asthma in past 2 years or uncontrolled asthma
- Availability of a medically suitable HLA-matched sibling donor without major ABO incompatibility or red blood cell alloantibodies against the patient
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
Research Team
R
Robert Nickel, MD
CONTACT
M
Maryanne Odinakachukwu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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