Family donor bone marrow transplants for children with non-malignant disorders
Tests family donor marrow transplants in children with non-malignant disorders.
Plain-language title generated from the registry record
Registry title: Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Key facts
- Where
- Grand Rapids, New Haven, St Louis, and Wilmington, United StatesSee the 4 sites
- Phase
- Phase 1/2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children and young people under 21 with a non-cancer blood, bone marrow, metabolic or immune disorder who need a bone marrow transplant from a partly matched family member.
- Age
- Up to 20.99 years old at enrollment (the registry lists 1 day to 21 years).
- Sex
- All sexes can join.
- Disease stage
- A non-cancer disorder that needs a bone marrow transplant: bone marrow failure, metabolic, immune or hemoglobin disorders. People with sickle cell disease must have a severe form, such as stroke, repeated acute chest syndrome, kidney damage from sickle cell disease, repeated painful crises needing long opioid use, red cell alloimmunization needing regular transfusions, or bone death in the joints.
- Earlier treatment
- People with sickle cell disease must have hemoglobin S below 30% within 30 days before starting alemtuzumab. People who had a previous stem cell transplant cannot join.
- Biomarker or test result
- Needs a performance score of 50 or more, and heart, lung, kidney and liver test results within set limits. People with sickle cell disease need hemoglobin S below 30%.
Main reasons for not taking part
- Has an HLA-identical sibling who is able and willing to donate bone marrow
- Cirrhosis, bridging fibrosis of the liver, or active hepatitis
- Uncontrolled bacterial, viral or fungal infection within 6 weeks before enrollment
- HIV infection or known HIV positive test
- Pregnant or breastfeeding
Read by machine on 12 November 2026.
Where in United States
- Helen DeVos Children's HospitalGrand Rapids, United StatesContact: Troy Quigg, DOTaking people
- Yale School of MedicineNew Haven, United StatesContact: Lakshmanan Krishnamurti, MDTaking people
- Washington University School of MedicineSt Louis, United StatesContact: Shalini Shenoy, MDTaking people
- Nemours Children's HealthWilmington, United StatesContact: Emi Caywood, MDTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Washington University School of Medicine
- Trial code
- NCT03128996
- Record updated
- 29 May 2026
- Original description
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.Read full original descriptionShow less
A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders
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.
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:; 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 for the 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 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: Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment; 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; Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis; Evidence of HIV infection or known HIV positive serology; Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma); Patients who have an HLA-identical sibling who is able and willing to donate bone marrow; Females who are pregnant or breast feeding.