Using Ruxolitinib to Treat Lung Problems After Stem Cell Transplant

Ruxolitinib for Early Lung Dysfunction After HSCT: a Phase II Study

This study is testing if Ruxolitinib can help children and young adults with lung problems after a stem cell transplant to breathe better and avoid serious lung damage.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 5 Years to 60 Years
• Sex: All
• Sponsor: Children's Hospital Medical Center, Cincinnati (Academic / other)
• Drugs / interventions: ruxolitinib
• Locations: 2 sites (Minneapolis, Minnesota and 1 other locations)
• Trial ID: NCT04908735 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of Ruxolitinib, a medication, to address early lung dysfunction in children and young adults who have undergone hematopoietic stem cell transplant (HSCT). The study focuses on patients exhibiting signs of bronchiolitis obliterans (BO), a serious lung condition that can develop post-transplant. By identifying and treating lung issues early, the trial aims to improve outcomes for patients who are at risk of irreversible lung damage. Participants will be monitored for specific lung function indicators to assess the effectiveness of the treatment.

Who should consider this trial

Good fit: Ideal candidates include children and young adults aged 5 to 60 who have undergone allogeneic HSCT and show early signs of lung dysfunction.

Not a fit: Patients who do not exhibit early lung dysfunction or are outside the age range of 5 to 60 years may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the incidence of long-term lung damage in pediatric HSCT patients.

How similar studies have performed: While there is limited data on the specific use of Ruxolitinib for this condition, similar approaches targeting lung dysfunction post-transplant have shown promise in other studies.

Eligibility criteria

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 effective method of birth control (e.g., implants, injectables, combined oral contraceptives, some intrauterine devices \[IUDs\], complete abstinence or sterilized partner) and a barrier method (e.g., condoms, cervical ring, sponge, etc.) during the period of therapy and for 90 days for both females and males after the last dose of study drug.
* Subjects previously treated with investigational agent for GVHD within the 30 days prior to first dose of study treatment. Other non-GVHD additional investigational agents may be allowed on a case by case basis with review/approval by the study Lead PI.

Where this trial is running

Minneapolis, Minnesota and 1 other locations

• University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)

Study contacts

• Principal investigator: Kasiani Myers, MD — Children's Hospital Medical Center, Cincinnati
• Study coordinator: Sara Loveless, BSN, RN

Conditions Hematopoietic Stem Cell Transplant; Bronchiolitis Obliterans