Could a cancer drug make stem cell transplants safer for sickle cell patients?
NCT ID NCT07252050
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether adding the drug ruxolitinib to a standard stem cell transplant can prevent graft failure in children and young adults with severe sickle cell disease. Up to 24 participants will receive the drug during conditioning and be followed for two years after transplant. The goal is to improve survival without complications like graft failure or needing a second transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Ruxolitinib (Jakafi)
- What this could lead to
- If it works, this could make stem cell transplants safer and more effective for people with sickle cell disease, reducing the chance of graft failure.
- What could go wrong
- This is an early-phase trial with only 24 participants, so results may not apply to everyone. Adding ruxolitinib could also increase side effects like infection or bleeding.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jun 2026
An estimate. Start dates often move.
- Expected to finish
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Nov 2029
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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12 to 45 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: 1. Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following: 1. History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients. 2. Recurrent moderate-severe acute chest syndrome (ACS) 3. Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care. 4. Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS). 5. For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec. 2. Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow. 3. Participants must meet institutional eligibility criteria for HCT. Exclusion Criteria: 1. Presence of an HLA-matched sibling who is willing and able to donate bone marrow. 2. Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection. 3. Previous HCT or solid organ transplant. 4. CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months. 5. Use of medications which significantly interfere with ruxolitinib metabolism. 6. Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients. 7. Inability to swallow and retain oral medication (use of nasogastric or gastrostomy tube permitted). 8. History of malignancy except resected basal cell carcinoma or treated carcinoma in-situ. 9. Participation in another clinical trial involving an investigational or off-label use of a drug or device in the past 3 months. 10. Currently pregnant or breast feeding. 11. Clinically significant, uncontrolled autoimmune disease. 12. High-titer anti-donor specific HLA antibodies (without review and approval by Study Chair). 13. Participant (or guardian) inability or unwillingness to comply with the dose schedule and study evaluations, comprehend or sign informed consent and utilize a highly effective method of contraception (for participants of child-bearing potential). 14. Any condition that would, in the investigator's judgment, interfere with full participation in the study, pose a significant risk to the subject, or interfere with interpretation of study data.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Children's Hospital of Colorado
NOT_YET_RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Manning Family Children's
NOT_YET_RECRUITINGNew Orleans, Louisiana, 70118, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding common pain drugs reduce morphine needs in sickle cell crises?
- Gene editing offers hope for a One-Time sickle cell cure
- Tiny biochip could reveal sickle cell severity
- Can a milder transplant cure sickle cell and thalassemia in adults?
- Can an antioxidant supplement calm sickle cell blood cells?
- Can a softer transplant cure sickle cell disease?