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RecruitingPhase 1 / Phase 2

Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years to 45 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 2 years

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Recruitment is expected to last for about 2 years and participants will be followed for an additional 2 years post-HCT."
  • "The study will enroll up to 24 participants over approximately 2 years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether adding ruxolitinib to a less intense treatment before a blood stem cell transplant helps keep the transplant from failing. It is for people ages 12–45 with sickle cell disease and certain health problems who have a partly matched family member willing to donate bone marrow.

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. Recruitment is expected to last for about 2 years and participants will be followed for an additional 2 years post-HCT.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You must be ages 12–45 and have any type of sickle cell disease, plus at least one of the health problems listed below.
  • A past stroke or disease of the blood vessels in the brain. For children, this includes brain blood vessel disease without symptoms.
  • Repeated moderate to severe acute chest syndrome, a serious lung problem.
  • Repeated pain attacks that need pain medicine by injection or through a vein, despite supportive care.
  • A need for regular blood transfusions to prevent pain attacks, stroke, or acute chest syndrome.
  • For adults, a heart ultrasound showing blood leaking through the tricuspid valve at a speed of at least 2.7 meters per second.
  • A partly tissue-matched parent, sibling, or half sibling who is willing and able to donate bone marrow.
  • You must meet the transplant center’s other rules for getting a blood stem cell transplant.

Who can't join

  • A fully tissue-matched sibling who can and will donate bone marrow; or a past blood stem cell or solid organ transplant.
  • An uncontrolled infection, active tuberculosis (TB), hepatitis B or C, or a positive HIV test or HIV infection.
  • Within the past 6 months: surgery to improve blood flow in the brain, a heart attack, a blood clot in the lungs, or a clot in a deep vein.
  • Medicines that strongly affect how the body breaks down ruxolitinib; a known allergy or severe reaction to ruxolitinib or any part of the pre-transplant treatment; or trouble swallowing and keeping down medicine. A feeding tube is allowed.
  • A history of cancer, except removed basal cell skin cancer or treated cancer that stayed where it started; or a serious, uncontrolled disease in which the immune system attacks the body.
  • Pregnancy or breastfeeding; or participation in another trial testing an experimental drug or device, or an unapproved use of one, in the past 3 months.
  • High levels of immune proteins that attack the donor’s tissue type, unless the study leader approves.
  • You or your guardian cannot or will not follow study visits and dosing, understand or sign the consent form, or use highly effective birth control if you can become pregnant. Other conditions may also rule you out if the study doctor thinks they would make participation unsafe, limit participation, or affect the results.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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.

Study sites (4)

CO

  • Children's Hospital of Colorado · Aurora, CO 80045Chris McKinney, MD · Principal investigatorMore trials in Aurora

GA

LA

PA