AzTrials
AzTrials

Other CancerPhase 2NCT03674047

Ruxolitinib for Bronchiolitis Obliterans Syndrome (BOS) After Allogeneic Hematopoietic Cell Transplantation (HCT)

A Phase 2 study testing ruxolitinib in people with Other Cancer, run by Massachusetts General Hospital at 7 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This research study is studying a drug as a possible treatment for Bronchiolitis Obliterans Syndrome (BOS) after having an Allogeneic Hematopoietic Cell Transplantation (HCT). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Other Cancer
What is being tested?
ruxolitinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
7 sitesUnited States
Study dates
19 Apr 2019to Dec 2025
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of BOS after HCT defined when all of the following criteria are met (as defined by the 2014 NIH criteria):
  • FEV1/VC < 0.7 or the 5th percentile of predicted.
  • FEV1 = Forced Expiratory Volume in 1 second.
  • VC = Vital Capacity (Forced Vital Capacity "FVC" or Slow Vital Capacity "SVC", whichever is greater)
  • The 5th percentile of predicted is the lower limit of the 90% confidence interval.
  • For elderly patients, use the lower limits of normal defined according to NHANESIII calculations.
Show all 17Show fewer
  • FEV1 <75% of predicted with ≥ 10% absolute decline over less than 2 years. FEV1 should not correct to >75% of predicted with albuterol, and the absolute decline for the corrected values should still remain ≥ 10% over 2 years. The remote comparator would be an evaluation of PFTs done within 2 years of the PFTs assessment being evaluated to determine eligibility.
  • Absence of active infection in the respiratory tract, documented with investigations directed by clinical symptoms, such as chest radiographs or computed tomographic scans or microbiologic cultures (sinus aspiration, upper respiratory tract viral screen, sputum culture, bronchoalveolar lavage).
  • One of the two supporting features of BOS:
  • Evidence of air trapping by expiratory CT or small airway thickening or bronchiectasis by high-resolution chest CT OR
  • Evidence of air trapping by PFTs: RV (Residual Volume) > 120% of predicted or RV/TLC elevated outside the 90% confidence interval (RV/Total Lung Capacity).
  • Life expectancy > 6 months at the time of enrollment as judged by the enrolling investigator.
  • Male or female; 18-75 years old.
  • ECOG Performance Status 0-2.
  • At least 4 weeks since initiation of the most recent systemic therapy for cGVHD or BOS
  • All females of childbearing potential must have a negative serum or urine pregnancy test < 7 days before study drug administration.
  • The ability to understand and willingness to sign a written consent document

Who cannot join

  • Recurrent malignancy or disease progression requiring anticancer therapy.
  • Currently receiving or have previously received ruxolitinib for chronic GVHD therapy.
  • Known history of allergy to ruxolitinib or its excipients.
  • Pregnant females or nursing mothers.
  • Hepatic dysfunction: transaminases (ALT, AST) > 5X ULN and/or total bilirubin > 3X ULN.
  • Hematologic dysfunction: absolute neutrophil count <1000/μL, platelet cout <50K, and/or Hgb < 8 g/dL.
Show all 18Show fewer
  • Renal dysfunction: calculated creatinine clearance < 40 mL/min (Cockcroft-Gault formula)
  • Receipt of any non-FDA approved study medication within the last 4 weeks (This does not apply to use of FDA-approved drugs for an off-label indication).
  • Presence of an active uncontrolled infection. An active uncontrolled infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs, or radiographic findings attributable to infection. Persisting fever without signs or symptoms will not be interpreted as an active uncontrolled infection.
  • Known human immunodeficiency virus infection.
  • Active hepatitis B virus (HBV) or hepatitis C virus infection that requires treatment or at risk for HBV reactivation. At risk for HBV reactivation is defined as hepatitis B surface antigen positive or anti-hepatitis B core antibody positive. Subjects with previous positive serology results must have negative polymerase chain reaction results. Subjects whose immune status is unknown or uncertain must have results confirming immune status before enrollment.
  • Severe organ dysfunction unrelated to underlying GVHD, including: Cholestatic disorders or unresolved veno-occlusive disease of the liver (defined as persistent bilirubin abnormalities not attributable to GVHD and ongoing organ dysfunction).
  • Clinically significant or uncontrolled cardiac disease, including unstable angina, acute myocardial infarction within 6 months from Day 1 of study drug administration, New York Heart Association Class III or IV congestive heart failure, circulatory collapse requiring vasopressor or inotropic support, or arrhythmia that requires therapy.
  • Clinically active asthma (variable and recurring symptoms of airflow obstruction and bronchial hyper-responsiveness), chronic obstructive pulmonary disease, interstitial lung disease, or cryptogenic organizing pneumonia or other causes of restrictive lung disease such as neuromuscular weakness or diaphragmatic paralysis.
  • Any condition that, in the opinion of the investigator, would interfere with the subject's ability to comply with the study requirements.
  • Uncontrolled substance abuse or psychiatric disorder.
  • Deemed (by the local PI or the PFT lab) unable to reliably perform pulmonary function tests.
  • Active smoker of cigarettes or marijuana.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • City of Hope Cancer Center

    Duarte, California, 91010

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

  • H. Lee Moffitt Cancer Center

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase II Study of Ruxolitinib for Bronchiolitis Obliterans Syndrome (BOS) After Allogeneic Hematopoietic Cell Transplantation (HCT)

This research study is studying a drug as a possible treatment for Bronchiolitis Obliterans Syndrome (BOS) after having an Allogeneic Hematopoietic Cell Transplantation (HCT).

Read the detailed description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved ruxolitinib for this specific disease but it has been approved for other uses.

In this study the investigators are assessing the safety and effectiveness of ruxolitinib when given to participants who have been diagnosed with BOS after HCT. BOS is a sign/symptom of chronic Graft-vs-Host Disease (GVHD), a condition in which cells from the donor's tissue attack the organs after HCT occurs.

Ruxolitinib blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. The investigators believe that ruxolitinib may lower the rate of GVHD through its ability to block the JAK2 pathway since this pathway can lead to inflammation in the body.

Conditions
Other Cancer
Being tested
ruxolitinib
Keywords
Hematopoietic Cell Transplantation

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ruxolitinibDrug

    Also called Jakafi

    Ruxolitinib blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. it's believe that ruxolitinib may lower the rate of GVHD through its ability to block the JAK2 pathway since this pathway can lead to inflammation in the body

Study groups in NCT03674047
Study groupRoleWhat they receive
newly-diagnosed BOSExperimental-Participants will take ruxolitinib twice every day
Receives: Drug: ruxolitinib
Established BOSExperimental-Participants will take ruxolitinib twice every day
Receives: Drug: ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. absolute FEV1 increase

    The proportion of participants with a sustained, absolute FEV1 increase by ≥ 10% after 3 months of treatment with ruxolitinib (compared to baseline measure prior to study enrollment) among participants with newly diagnosed Bronchiolitis Obliterans Syndrome BOS.

    Measured: 3 Months

  2. treatment failure, comparing 3-month FEV1 to baseline FEV1

    The proportion of participants who do not experience a sustained, absolute decrease in FEV1 by ≥ 10% after 3 months of treatment with ruxolitinib (compared to baseline measure prior to study enrollment) among participants with established Bronchiolitis Obliterans Syndrome BOS.

    Measured: 3 Months

Secondary outcomes (6)

  1. Change scores for PFT measurements

    Change in pulmonary function test (PFT) measurements.

    Measured: 1 Year

  2. Improvements in chronic GVHD organ manifestations

    Improvements in chronic GVHD organ manifestations will be categorized according to the NIH chronic GVHD consensus criteria.

    Measured: 3 and 6 Months

  3. Overall survival

    Measured: 2 Years

  4. cGVHD progression-free survival

    cGVHD progression-free survival is defined as the time from registration to the earlier of progression of chronic GVHD or death due to any cause. Participants alive without cGVHD progression are censored at the date of last disease evaluation.

    Measured: 2 Years

  5. The incidence and types of serious adverse events

    Adverse events are graded according to Common Terminology Criteria for Adverse Events (CTCAE v4).

    Measured: From the start of treatment until 30 days after the end of treatment, up to 13 months total

  6. The total systemic corticosteroid dose longitudinally over time

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Sep 2018
  2. Study start19 Apr 2019
  3. Primary completion2 Dec 2022
  4. Study completionDec 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03674047
Sponsor's protocol ID
18-265
Status last verified
Nov 2025
First submitted
14 Sep 2018
Last updated
10 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • DeFilipp Z, Kim HT, Cheng GS, Hamilton BK, Chhabra S, Hamadani M, Sandhu KS, Perez L, Lee CJ, Brennan TL, Garrelts C, Brown BM, Gallagher K, Newcomb RA, El-Jawahri A, Chen YB. A phase 2 multicenter trial of ruxolitinib to treat bronchiolitis obliterans syndrome after allogeneic HCT. Blood Adv. 2025 Jan 28;9(2):244-253. doi: 10.1182/bloodadvances.2024014000.

    PubMed 39365992

  • Bos S, Murray J, Marchetti M, Cheng GS, Bergeron A, Wolff D, Sander C, Sharma A, Badawy SM, Peric Z, Piekarska A, Pidala J, Raj K, Penack O, Kulkarni S, Beestrum M, Linke A, Rutter M, Coleman C, Tonia T, Schoemans H, Stolz D, Vos R. ERS/EBMT clinical practice guidelines on treatment of pulmonary chronic graft-versus-host disease in adults. Eur Respir J. 2024 Mar 28;63(3):2301727. doi: 10.1183/13993003.01727-2023. Print 2024 Mar.

    PubMed 38485149

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ruxolitinib in people with Other Cancer, run by Massachusetts General Hospital at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 10 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

absolute FEV1 increase (time frame: 3 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03674047, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in