Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant

Launched by CHILDREN'S HOSPITAL MEDICAL CENTER, CINCINNATI · May 26, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a treatment for early lung problems that can happen after a hematopoietic stem cell transplant (HSCT), which is a procedure often used to treat certain blood disorders. The trial is looking at whether adding a medication called ruxolitinib to standard treatments can help improve lung function and prevent long-term lung issues, particularly a condition called bronchiolitis obliterans (BO). BO can be very serious and is often diagnosed too late to make a difference because children may have trouble undergoing the necessary lung tests.

To be eligible for the trial, participants need to be between 5 and 60 years old and must have had an HSCT with signs of early lung dysfunction, such as a drop in lung function tests or breathing difficulties. Participants can expect to receive treatment and will be monitored closely for improvements in their lung health. It's important to note that certain medical conditions, like active lung infections or significant liver problems, may prevent someone from joining the study. Overall, this trial aims to help improve the care and outcomes for children and adults facing lung issues after transplant.

Gender

ALL

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.