Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant (NCT04908735) | Clinical Trial Compass
TerminatedPhase 2
Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant
Stopped: Study closed due to slow enrollment.
United States7 participantsStarted 2021-11-12
Plain-language summary
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.
Who can participate
Age range
5 Years – 60 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Subjects ≥ 5 years and ≤ 60 years of age who have undergone allogeneic HCT AND exhibit early lung dysfunction as defined by any one of the following:
* \>10% decrease in FEV1 from baseline or decrease of 25% of FEF 25-75 from baseline
* active GVHD in another organ system + pulmonary symptoms (Tachypnea without wheezing, new oxygen requirement, cough)
* Increased R5 by 50% by clinical oscillometry
* Air trapping on CT, small airway thickening, or bronchiectasis
AND - All age groups, including adults:
Adequate renal function defined as estimated Creatinine Clearance (CrCl) ≥ 30 mL/min as calculated by the cystatin c GFR or nuclear GFR
Adequate hepatic function as defined by:
* ALT and AST ≤ 5 x ULN, unless the ALT / AST increase is due to cGVHD
* Total bilirubin of ≤ 5 x ULN (unless of non-hepatic origin or due to Gilbert's Syndrome) or Total bilirubin of \< 10 x ULN if due to GVHD
Adequate hematological function defined as:
* Absolute neutrophil count ≥1.0 x 10\^9/L
* Platelets ≥30 x 10\^9/L
PT/INR \<2 x ULN and PTT (aPTT) \< 2 x ULN (unless abnormalities are unrelated to coagulopathy or bleeding disorder)
Exclusion Criteria:
* Known hypersensitivity to any constituent of the study medication.
* Active uncontrolled pulmonary infection (preceding infectious evaluation including bronchoscopy as clinically indicated)
* Subjects who are pregnant or breastfeeding or are at risk of pregnancy or fathering a baby and are unable to use acceptable highly …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with ruxolitinib treatment response
Timeframe: 6 months from early lung dysfunction diagnosis
Trial details
NCT IDNCT04908735
SponsorChildren's Hospital Medical Center, Cincinnati