Sickle Cell Disease study: Ruxolitinib
Official title Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
ClinicalTrials.gov ID: NCT07252050
What this study is testing
What is Ruxolitinib?
An investigational oral medicine being studied as a potential way to lower graft failure after haploidentical stem cell transplant for sickle cell disease. It is designed to inhibit JAK1 and JAK2 enzymes, which reduces inflammatory signaling that may contribute to graft rejection.
Also referred to as Jakafi.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether adding ruxolitinib to a reduced intensity conditioning regimen reduces graft failure after a haploidentical stem cell transplant in children and young adults with sickle cell disease.
- Who it's for
- You may be able to join if you are a child or young adult with sickle cell disease who is planned for a haploidentical stem cell transplant.
- What's involved
- You would receive ruxolitinib (a medicine that blocks certain signals) along with the transplant preparation. You will have the transplant and then be checked regularly.
- Time commitment
- The study lasts about 4 years total, with about 2 years to join and 2 years of follow‑up visits after the transplant.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 2 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 45
You may be able to join if
- History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
- Recurrent moderate-severe acute chest syndrome (ACS)
- Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
- Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
- For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.
You likely can't join if
- Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
- Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
- Previous HCT or solid organ transplant.
- CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.
- Use of medications which significantly interfere with ruxolitinib metabolism.
- Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients.
See the full eligibility criteria
- History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
- Recurrent moderate-severe acute chest syndrome (ACS)
- Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
- Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
- For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.
- Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow.
- Participants must meet institutional eligibility criteria for HCT.
- Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
- Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
- Previous HCT or solid organ transplant.
- CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.
- Use of medications which significantly interfere with ruxolitinib metabolism.
- Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients.
- Inability to swallow and retain oral medication (use of nasogastric or gastrostomy tube permitted).
- History of malignancy except resected basal cell carcinoma or treated carcinoma in-situ.
- Participation in another clinical trial involving an investigational or off-label use of a drug or device in the past 3 months.
- Currently pregnant or breast feeding.
- Clinically significant, uncontrolled autoimmune disease.
- High-titer anti-donor specific HLA antibodies (without review and approval by Study Chair).
- 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...
- 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.
The study team makes the final eligibility decision.
Where it is enrolling
- Atlanta, Georgia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 45 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Atlanta, Georgia, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.