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Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

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

A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders

NCT03128996•Washington University School of Medicine

View on ClinicalTrials.gov

Summary

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.

Detailed Description

Patients \< 21 years of age with a non-malignant disorder benefited by hematopoietic stem cell transplant will receive a reduced intensity conditioning regimen consisting of hydroxyurea, alemtuzumab, fludarabine, thiotepa, and melphalan. This will be followed by a familial HLA-mismatched bone marrow transplant. The primary objective is to establish safety and donor cell engraftment at 100 days and 1 year post-transplant.

Eligibility

Age

0 - 21 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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
•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
+10 more criteria

Exclusion Criteria

•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)

Locations (4)

Yale School of Medicine

New Haven, Connecticut, United States

Nemours Children's Health

Wilmington, Delaware, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Washington University School of Medicine

St Louis, Missouri, United States

Key Dates

Start Date

March 20, 2017

Primary Completion Date

April 1, 2028

Completion Date

April 1, 2033

Last Updated

January 7, 2026

Enrollment

ESTIMATED participants

Conditions

Severe Sickle Cell DiseaseBone Marrow Failure SyndromesMetabolic DisordersImmunologic DisordersHemoglobinopathiesNon-malignant Disorders

Interventions

RIC regimen

DRUG

GVHD prophylaxis regimen

DRUG

Contacts

Shalini Shenoy, MD

CONTACT

314-454-6018 shalinishenoy@wustl.edu

Ian Snyder, BS, CCRP

CONTACT

314-273-5953 ian.s@wustl.edu

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 7, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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