Actively Recruiting
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
Led by Robert Nickel · Updated on 2026-03-10
100
Participants Needed
7
Research Sites
654 weeks
Total Duration
On this page
Sponsors
R
Robert Nickel
Lead Sponsor
T
The Hospital for Sick Children
Collaborating Sponsor
AI-Summary
What this Trial Is About
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
CONDITIONS
Official Title
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 2 to 25 years with sickle cell disease.
- For hemoglobin SS and Sβ0 thalassemia genotypes, must have at least one: abnormal transcranial Doppler measurement on two occasions, cerebral infarction on brain MRI, two or more acute chest syndrome episodes in lifetime, three or more pain events requiring opioids or IV treatment in lifetime, hospitalization for SCD pain or acute chest syndrome while on hydroxyurea, two or more priapism episodes, regular red blood cell transfusions (≥8 in last 12 months), or at least two splenic sequestration episodes requiring transfusion or splenectomy.
- For other sickle genotypes (hemoglobin SC, Sβ+ thalassemia), must have at least one: overt stroke, two or more acute chest syndrome episodes in last 2 years, three or more pain events requiring opioids or IV treatment in last year, hospitalization for SCD pain or acute chest syndrome while on hydroxyurea, two or more priapism episodes, regular red blood cell transfusions (≥8 in last 12 months), or at least two 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 Hepatitis B or C.
- Liver dysfunction with direct bilirubin >1.5 mg/dL or transaminases >5 times upper limit for age.
- Cardiac dysfunction with left ventricular shortening fraction <25% or ejection fraction <50%.
- Kidney impairment with creatinine clearance less than 60 mL/min/1.73m2.
- Pulmonary impairment with diffusion capacity (DLCO) <35%, oxygen saturation <85%, or PaO2 <70.
- History of red blood cell alloantibodies against donor antigens.
- Major ABO blood group incompatibility with donor.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
Children's National Health System
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Columbia University
New York, New York, United States, 10032
Actively Recruiting
4
Levine Children's Hospital
Charlotte, North Carolina, United States, 28203
Actively Recruiting
5
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
6
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Actively Recruiting
7
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
Research Team
R
Robert Nickel, MD
CONTACT
M
Maryanne Odinakachukwu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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