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New treatment option for Sickle Cell Disease
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?
Ruxolitinib is an investigational medicine, being studied as a potential treatment for sickle cell disease.
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 trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.
- 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
- Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:
- 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).
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
- Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:
- 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's taking place
- Aurora, Colorado, United States
- Atlanta, Georgia, United States
- New Orleans, Louisiana, United States
- Philadelphia, Pennsylvania, 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 - 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?
Study sites include Aurora, Colorado, United States; Atlanta, Georgia, United States; New Orleans, Louisiana, United States; Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.