Actively Recruiting
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Led by Washington University School of Medicine · Updated on 2026-01-07
29
Participants Needed
4
Research Sites
836 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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: overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy; recurrent acute chest syndrome with significant respiratory compromise each time; sickle nephropathy; recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity; red cell alloimmunization with the need for chronic transfusions; recurrent osteonecrosis or multiple joint involvement from avascular necrosis
- Patients with sickle cell disease must have hemoglobin S < 30% within 30 days prior to beginning alemtuzumab
- Age ≤ 20.99 years at the time of enrollment
- Performance score ≥ 50
- Left ventricular ejection fraction > 40% or left ventricular shortening fraction > 26% by echocardiogram
- DLCO > 40% (corrected for hemoglobin) or pulse oximetry with a baseline O2 saturation of ≥ 90% on room air if too young to perform PFTs
- Serum creatinine ≤ 1.5x upper limit of normal for age and/or GFR > 70 mL/min/1.73m2
- Direct bilirubin < 2x upper limit of normal for age
- ALT and AST < 5x upper limit of normal for age
- Participants who have or are receiving ≥ 8 packed red blood cell transfusions for ≥ 1 year or ≥ 20 packed red blood cell transfusions (lifetime cumulative) will undergo liver MRI for estimation of hepatic iron content; liver biopsy is indicated for hepatic iron content ≥ 7mg Fe/mg liver dry weight by liver MRI; histologic examination of the liver must document absence of cirrhosis, bridging fibrosis, and active hepatitis
- Female subjects of childbearing potential must agree to practice 2 methods of contraception at the same time from signing informed consent through 12 months post transplant
- Male subjects must agree to practice effective barrier contraception or true abstinence from signing informed consent through 12 months post transplant
- Written informed consent must be obtained from all recipients according to institutional guidelines
You will not qualify if you...
- Patients who have an HLA-identical sibling 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 investigational drug/device or off-label use within 3 months of enrollment
- Females who are pregnant or breastfeeding
- Patients with active autoimmune disease such as sarcoidosis, lupus, or scleroderma
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Yale School of Medicine
New Haven, Connecticut, United States, 06510
Actively Recruiting
2
Nemours Children's Health
Wilmington, Delaware, United States, 19803
Actively Recruiting
3
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
4
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
S
Shalini Shenoy, MD
CONTACT
I
Ian Snyder, BS, CCRP
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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