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Reduced Intensity Bone Marrow Transplant for Non-Malignant Disorders

This study is looking at a type of bone marrow transplant for children and young adults (up to 21 years old) with serious non-cancerous conditions like severe sickle cell disease, bone marrow failure, or certain metabolic or immune disorders. The transplant uses a donor who is a family member but not a perfect match (familial HLA-mismatched). Before the transplant, participants receive a reduced intensity conditioning (RIC) regimen, which includes medicines like hydroxyurea, alemtuzumab, fludarabine, thiotepa, and melphalan. After the transplant, they receive medicines like cyclophosphamide, tacrolimus, mycophenolate mofetil (MMF), abatacept, and rituximab to help prevent graft-versus-host disease (GVHD), a common transplant complication. The main goal is to see how safe this treatment is and if the donor cells successfully grow in the patient at 100 days and 1 year after the transplant. The study plans to enroll 29 participants.

Study design
This is an interventional study, meaning participants receive a specific treatment. It plans to enroll 29 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed to assess donor cell engraftment at 100 days and 1 year post-transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03128996

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Recruiting
PHASE1Ages 1–21InterventionalTreatment
Washington University School of Medicine
~29 participants
Updated 2026-05-29 on ClinicalTrials.gov
What's tested:RIC regimenGVHD prophylaxis regimen

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Donor engraftment
Measured over 100 days and 1 year post-transplant

Conditions

Severe Sickle Cell Disease
Bone Marrow Failure Syndromes
Metabolic Disorders
Immunologic Disorders
Hemoglobinopathies
Non-malignant Disorders

Explore related trials

Bone Marrow Failure SyndromeHemoglobinopathyImmunologic DisordersMetabolic DisorderNon-malignant DisordersSickle Cell Disease

NCT03128996

Where you'd take part

This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Nemours Children's Health

    Wilmington, Delawarestudy coordinator listed

    Recruiting

  • Washington University School of Medicine

    St Louis, Missouristudy coordinator listed

    Recruiting

  • Helen DeVos Children's Hospital

    Grand Rapids, Michiganno site contact published

    Recruiting

  • Yale School of Medicine

    New Haven, Connecticutno site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Shalini Shenoy, MD · PRINCIPAL_INVESTIGATOR · Washington University School of Medicine

Who to contact

Shalini Shenoy, MD
[email protected]
314-454-6018
Email the study team

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Eligibility criteria

Inclusion

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:
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.
Female subjects of childbearing potential, must agree to practice 2 methods of contraception at the same time from the time of signing of informed consent through 12 months post transplant. Male subjects must agree to practice effective barrier contraception or practice true abstinence from the time of signing informed consent through 12 months post transplant.
Written informed consent must be obtained from all recipients in accordance with the guidelines of the institution's Human Studies Committee.

Exclusion

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)

What this trial measures

  • Donor engraftment100 days and 1 year post-transplant

    as measured by chimerism