Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant

• Register: ClinicalTrials.gov
• Last refreshed on: 6 January 2025
• Main ID: NCT04908735
• Date of registration: 26/05/2021
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant HSCT
• Scientific title: Ruxolitinib for Early Lung Dysfunction After HSCT: a Phase II Study
• Date of first enrolment: November 12, 2021
• Target sample size: 40
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04908735
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sara Loveless, BSN, RN

• Name: Kasiani Myers, MD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion 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.


Age minimum: 5 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hematopoietic Stem Cell Transplant (HSCT)

Bronchiolitis Obliterans (BO)

Intervention(s)

Drug: Ruxolitinib

Primary Outcome(s)

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

Secondary Outcome(s)

Number of participants with lung function response measured by oscillometry [Time Frame: 24 weeks after ruxolitinib initiation]

Number of participants with lung function response measured by a Xenon MRI scan [Time Frame: 24 weeks after ruxolitinib initiation]

Percentage of participants with JAK inhibition [Time Frame: 24 weeks after ruxolitinib initiation]

Number of participants with lung function response measured by home spirometry [Time Frame: 24 weeks after ruxolitinib initiation]

Secondary ID(s)

2020-0719

Source(s) of Monetary Support

Please refer to primary and secondary sponsors