RecruitingSevere Sickle Cell DiseaseBone Marrow Failure SyndromesMetabolic Disorders
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Eligible age
0–21 yrs
Accepts
All genders
Locations
4 states
Healthy volunteers
No
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About this study
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
Sponsor: Washington University School of Medicine
You may qualify if…
- ✓ Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy
- ✓ For patients with sickle cell disease, must have one of the following severe manifestations:
- ✓ 1. Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy
- ✓ 2. Recurrent acute chest syndrome with significant respiratory compromise each time
- ✓ 3. Sickle nephropathy
- ✓ 4. Recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity
- ✓ 5. Red cell alloimmunization with the need for chronic transfusions
- ✓ 6. Recurrent osteonecrosis or multiple joint involvement from avascular necrosis
You may not qualify if…
- ✕ Patients who have an HLA-identical sibling who is able and willing to donate bone marrow
- ✕ Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis
- ✕ Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment
- ✕ Evidence of HIV infection or known HIV positive serology
- ✕ Patients who have received a previous stem cell transplant
- ✕ Patients who have received an investigational drug or device or off-label use of a drug or device within 3 months of enrollment
- ✕ Females who are pregnant or breast feeding
- ✕ Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma)
Where it's recruiting
Source: ClinicalTrials.gov · NCT03128996 · last updated 2026-05-29