Can a Sibling's bone marrow cure sickle cell disease in children?
NCT ID NCT04018937
First seen Sep 24, 2026 · Last updated Sep 25, 2026 · Updated 1 time
Summary
Researchers are testing whether a bone marrow transplant from a matched sibling can cure sickle cell disease in children under 13. Participants receive chemotherapy to prepare their bodies, then donor cells. The goal is to see how many children can live without the disease and without needing immune-suppressing drugs two years later.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a bone marrow transplant from a matched sibling donor, using chemotherapy drugs alemtuzumab and fludarabine to prepare the body
- What this could lead to
- If it works, children with sickle cell disease could receive a one-time transplant that removes the disease and lets them stop immune-suppressing medicines for good.
- What could go wrong
- The chemotherapy and transplant can cause serious complications, including organ damage and rejection of the donor cells. The trial is small and early, so it may not show that the approach works for most children.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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43 people
The number who actually took part.
- Started
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Mar 2019
- Expected to finish
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Nov 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 13 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients must be at least 2 years and less than 13 years old and have a sickle hemoglobinopathy. * Patient must have an HLA identical sibling donor who is less than 13 years old. Sibling donors must not have any form of SCD. It is acceptable for the donor to carry a hemoglobinopathy trait. * Patients must meet criteria for symptomatic SCD as defined below. * Severe disease: * Previous clinical stroke, defined as a neurological deficit lasting longer than 24 hours plus new finding on head CT or brain MRI/MRA. * Progressive silent cerebral infarction, as evidenced by serial MRI scans that demonstrate the development of a succession of lesions (at least two temporally discreet lesions, each measuring at least 3 mm in greatest dimension on the most recent brain MRI/MRA) or the enlargement of a single lesion, initially measuring at least 3 mm). Lesions must be visible on T2-weighted MRI sequences. * Abnormal TCD testing (confirmed elevated velocities in any single vessel of TAMMV \> 200 cm/sec for non-imaging TCD) * Significant vasculopathy on MRA (greater than 50% stenosis of \> 2 arterial segments or complete occlusion of any single arterial segment). * Frequent (at least 3 per year for preceding 2 years) painful vaso-occlusive episodes (defined as episode lasting at least 4 hours and requiring hospitalization or outpatient treatment with parenteral opioids). If patient is on hydroxyurea and its use has been associated with a decrease in the frequency of episodes, the frequency should be gauged from the 2 years prior to the start of hydroxyurea. * Recurrent (at least 3 in lifetime) acute chest syndrome events which have necessitated erythrocyte transfusion therapy. * Any combination of at least 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years. If patient is on hydroxyurea and its use has been associated with a decrease in the frequency of episodes, the frequency should be gauged from the 3 years prior to the start of hydroxyurea. * Less severe disease: to qualify as having less severe disease, patients must not meet criteria for severe disease and must have one of the following: * Asymptomatic cerebrovascular disease, as evidenced by one the following: Silent cerebral infarction with at least one lesion measuring at least 3 mm in one dimension that is visible on two planes on the most recent brain MRI, or, cerebral arteriopathy, as evidenced by conditional TCD (TAMMV\>170cm/sec but \<200cm/sec) on two separate scans \>2 weeks apart). If patient has a conditional TCD, then a brain MRI/MRA to evaluate for vasculopathy is required. * 2 or more painful vaso-occlusive episodes (in lifetime) requiring hospitalization or outpatient treatment with parenteral opioids. * 2 or more episodes of acute chest syndrome (in lifetime) irrespective of SCD modifying therapy administered. * Any combination of at least 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above, lifetime). * Patients with HbSS and HbSβ°thalassemia who have no clinical complications of their sickle cell disease and do not meet the criteria for less severe or severe disease. * Participant's parent or legal guardian must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines. * Patient must have been evaluated and parent(s)/legal guardian, and the patient as age appropriate as determined by the treating center, adequately counseled regarding treatment options for SCD by a pediatric hematologist. * Co-enrollment on STAR Project Sickle Cure (PSC) study is required for sites that are activated and participating in the study. Exclusion Criteria: * Bridging (portal to portal) fibrosis or cirrhosis of the liver. * Parenchymal lung disease stemming from SCD or other process defined as a diffusing capacity of the lungs for carbon monoxide (DLCO; corrected for hemoglobin) or forced vital capacity of less than 45% of predicted. Children unable to perform pulmonary function testing will be excluded if they require daytime oxygen supplementation. * Renal dysfunction with an estimated glomerular filtration rate (GFR) \< 50% of predicted normal for age. * Cardiac dysfunction with shortening fraction \< 25%. * Neurologic impairment other than hemiplegia, defined as full-scale intelligence quotient (IQ) of less than or equal to 70, quadriplegia or paraplegia, or inability to ambulate. * Lansky functional performance score \< 70%. * Patient is HIV infected. * Donor is HIV infected. * Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate HSCT. * Patient's parent(s) or legal guardian is unable to understand the nature and the risks inherent in the HSCT process. * History of lack of adherence with medical care that would jeopardize transplant course. * Donor who for psychological, physiologic, or medical reasons is unable to tolerate a bone marrow harvest or receive general anesthesia. * Active viral, bacterial, fungal or protozoal infection. * Patients with viral upper respiratory tract infections should be asymptomatic for at least 7 days prior to enrollment. PCR testing for respiratory viruses (nasopharyngeal sample) should be negative at the start of the conditioning regimen. Exceptions may be made in patients with prolonged carriage (repeatedly positive over many weeks) of rhinovirus. These exceptions should be discussed with and approved by both study co-chairs and STAR Medical Director.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Atrium Health Levine Cancer Institute
Charlotte, North Carolina, 28204, United States
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Cancercare Manitoba/Winnipeg Children's Hospital
Winnipeg, Manitoba, Canada
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Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
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Children's Healthcare of Altanta
Atlanta, Georgia, 30322, United States
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Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Dana-Farber Cancer Institute/Boston Children's Hospital
Boston, Massachusetts, 02215, United States
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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Phoenix Children's
Phoenix, Arizona, 85016, United States
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Riley Children's Health/Indiana University
Indianapolis, Indiana, 46202, United States
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University of Chicago
Chicago, Illinois, 60637, United States
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University of North Carolina Medical Center
Chapel Hill, North Carolina, 27514, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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Yale University, Yale Cancer Center
New Haven, Connecticut, 06520, United States
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Other studies related to the condition(s) this trial covers.
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