Actively Recruiting
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Led by Paul Szabolcs · Updated on 2025-08-12
5
Participants Needed
1
Research Sites
469 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
CONDITIONS
Official Title
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent and/or assent provided by patient, parent, or legal guardian
- Ages 5 to 40 years at time of consent
- Diagnosis of sickle cell disease with complications such as recurrent painful episodes, acute chest syndrome, stroke or neurologic events, chronic transfusion dependence, elevated transcranial Doppler velocities, elevated TRV, renal insufficiency, hepatopathy, or irreversible organ damage
- OR diagnosis of beta-thalassemia major or Diamond-Blackfan anemia with transfusion dependence and iron overload
- Minimum donor match of 4/8 related or 6/8 unrelated with completed donor search and no eligible matched donor available
- Adequate organ function including kidney, liver, heart, and lungs
- Cardiology and pulmonology clearance for transplant conditioning
- HIV negative by PCR
- Negative pregnancy test if female aged 10 years or older or post-menarche unless surgically sterilized
- Agreement to use approved birth control for up to 24 months post-transplant for females of childbearing potential and sexually active males
- Counseling on infertility risks and discussion of sperm banking or oocyte harvesting
- Hydroxyurea trialed and failed in sickle cell disease patients
You will not qualify if you...
- Availability of superior donor options such as matched sibling or matched unrelated donor
- Stem cell transplantation within 6 months prior to conditioning
- Central nervous system event within 6 months before conditioning
- Pregnancy or lactation
- Uncontrolled bacterial, viral, or fungal infection
- Other medical problems or findings that pose additional risks or interfere with study participation as judged by investigators
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
Research Team
P
Paul Szabolcs, MD
CONTACT
S
Shawna McIntyre, RN
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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