Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
Children's Hospital Medical Center, Cincinnati
Study ID
NCT04908735
Phase
PHASE2
Status
Terminated

Conditions

  • Bronchiolitis Obliterans (BO)
  • Hematopoietic Stem Cell Transplant (HSCT)

Eligibility Criteria

Sex
ALL
Age
5 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    Participants will receive ruxolitinib orally twice daily for 24 weeks.

Study Details

Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy, and lung injury affects as many as 25% of children receiving HSCT. Improved transplant techniques and major improvements in survival mean that HSCT is being more widely used, and more mismatched grafts are being used. Bronchiolitis obliterans (BO) is a major limitation of pediatric HSCT success as BO is commonly diagnosed late in children, when lung injury is irreversible, leading to long term morbidity or even death. Currently, there are major gaps in our knowledge regarding incidence, etiology and optimal treatment of BO following HSCT, and important diagnostic limitations specific to children. Diagnosis of BO is usually based on performance of pulmonary function tests, which is usually impossible in ill children under 10. Even older children who feel unwell or un-cooperative may be unable to produce interpretable data. These deficiencies in diagnosis mean that BO is commonly diagnosed late, meaning fibrosis has occurred and lesions are irreversible. The hypothesis for this interventional trial is that early treatment with standard Flovent/montelukast and steroids plus ruxolitinib will reverse lung injury and reduce the frequency of chronic pulmonary impairment or florid BO.

Key Dates

Start date
Nov 12, 2021
Status verified
Apr 2026
Primary completion
Sep 17, 2025
Completion
Nov 16, 2025

Study Design

Enrollment
7 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Ruxolitinib Treatment

Primary Outcome Measure

Number of participants with ruxolitinib treatment response [ Time Frame: 6 months from early lung dysfunction diagnosis ]

Locations (2)

FacilityCityStateZIPSite coordinators
University of MinnesotaMinneapolisMinnesota55455-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-

Find similar trials in Minneapolis, MN

By research site
University of Minnesota· Minneapolis, MNCincinnati Children's Hospital Medical Center· Cincinnati, OH

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