Actively Recruiting
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Led by University of Colorado, Denver · Updated on 2026-05-04
24
Participants Needed
4
Research Sites
188 weeks
Total Duration
On this page
Sponsors
U
University of Colorado, Denver
Lead Sponsor
C
Children's Healthcare of Atlanta
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 12 to 45 years with any genotypic form of sickle cell disease
- History of stroke or cerebrovascular disease (including asymptomatic) for pediatric patients
- Recurrent moderate to severe acute chest syndrome
- Recurrent vaso-occlusive pain episodes needing parenteral analgesia despite supportive care
- Need for chronic transfusion therapy to prevent vaso-occlusive complications
- For adults, echocardiographic tricuspid valve regurgitant jet velocity of 2.7 m/sec or greater
- Presence of an HLA haploidentical first-degree relative willing and able to donate bone marrow
- Must meet institutional eligibility criteria for hematopoietic cell transplant
You will not qualify if you...
- Presence of an HLA-matched sibling donor willing and able to donate bone marrow
- Uncontrolled infection, active tuberculosis, Hepatitis B or C, or HIV infection
- Previous hematopoietic cell transplant or solid organ transplant
- CNS revascularization, myocardial infarction, pulmonary embolus, or deep vein thrombosis within past 6 months
- Use of medications that interfere significantly with ruxolitinib metabolism
- Known severe allergy or reaction to ruxolitinib or conditioning regimen components
- Inability to swallow and retain oral medication (nasogastric or gastrostomy tube allowed)
- History of malignancy except treated basal cell carcinoma or carcinoma in situ
- Participation in another investigational drug or device trial within past 3 months
- Currently pregnant or breastfeeding
- Clinically significant uncontrolled autoimmune disease
- High-titer anti-donor specific HLA antibodies without study chair approval
- Inability or unwillingness to comply with dosing schedule, study evaluations, or contraception requirements
- Any condition posing significant risk or interfering with participation or data interpretation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Not Yet Recruiting
2
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
3
Manning Family Children's
New Orleans, Louisiana, United States, 70118
Not Yet Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
Research Team
L
Laura McLaughlin, MD
CONTACT
K
Kayla Ortiz
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here