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

NCT07252050 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 24-0735
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 4

Brief Summary

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

Sickle Cell Disease; Hematopoetic Stem Cell Transplant; Haploidentical Hematopoietic Stem Cell Transplant; Haploidentical Stem Cell Transplantation; Graft Failure

Keywords

Sickle cell disease; Hematopoietic cell transplant; Ruxolitinib; Pediatric; Young Adult; Haplo

Outcome Measures

Primary Outcomes (1)

• Event Free Survival

  Event Free Survival (EFS) is defined as survival without a qualifying event (primary or secondary GF, second HCT or death).

  1 year post-HCT

Secondary Outcomes (15)

• Overall Survival

  1 and 2 years post-HCT

• Event Free Survival

  2 years post-HCT

• Neutrophil Recovery

  Up to Day 60 post-HCT

• Platelet Recovery

  Up to Day 180 post-HCT

• Acute GVHD

  Up to Day +100 post-HCT

Study Arms (1)

Ruxolitinib-Enhanced RIC

EXPERIMENTAL

Pediatric and young adult participants who are undergoing haplo HCT for SCD will receive RIC with fludarabine, cyclophosphamide, thiotepa, ATG and low-dose TBI along with ruxolitinib. Ruxolitinib will continue post-HCT in addition to post-transplant cyclophosphamide and sirolimus or a calcineurin inhibitor for GVHD prophylaxis.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

All participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.

Also known as: Jakafi

Ruxolitinib-Enhanced RIC

Eligibility Criteria

• Age: 12 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify 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).
• 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.

You may not qualify 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.
• 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 of child-bearing potential).
• 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.

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Children's Healthcare of Atlanta: collaborator
• Emory University: collaborator
• Incyte Corporation: collaborator

Study Sites (4)

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

RECRUITING

Manning Family Children's

New Orleans, Louisiana, 70118, United States

NOT YET RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Laura McLaughlin, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura McLaughlin, MD

CONTACT

720-777-7008; Laura.McLaughlin@childrenscolorado.org

Kayla Ortiz

CONTACT

720-777-4151; Kayla.Ortiz@childrenscolorado.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Lead Principal Investigator/Study Chair

Study Record Dates

• First Submitted: November 14, 2025
• First Posted: November 26, 2025
• Study Start: April 10, 2026
• Primary Completion (Estimated): November 19, 2028
• Study Completion (Estimated): November 19, 2029
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)