Hematopoietic Stem Cell Transplant (HSCT)Phase 2HSCTNCT04908735
Ruxolitinib for Early Lung Dysfunction After Hematopoietic Stem Cell Transplant
A Phase 2 study testing Ruxolitinib in people with Hematopoietic Stem Cell Transplant (HSCT) and Bronchiolitis Obliterans (BO), run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 60 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplant (HSCT), Bronchiolitis Obliterans (BO)
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited States
- Study dates
- 12 Nov 2021to 16 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 5 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
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- 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)
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Ruxolitinib for Early Lung Dysfunction After HSCT: a Phase II Study
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.
- Being tested
- Ruxolitinib
- Medical subject headings
- Bronchiolitis Obliterans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Participants will receive ruxolitinib orally twice daily for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib Treatment | Experimental | Not described Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with ruxolitinib treatment response
Treatment response is defined by stable and/or improved lung function as defined by the National Institutes of Health Chronic GVHD Response Criteria Working Group.
Measured: 6 months from early lung dysfunction diagnosis
Secondary outcomes (4)
Percentage of participants with JAK inhibition
The presence of JAK inhibition will be measured by phospho stat5
Measured: 24 weeks after ruxolitinib initiation
Number of participants with lung function response measured by a Xenon MRI scan
The presence of a lung function response will be measured by a Xenon MRI scan
Measured: 24 weeks after ruxolitinib initiation
Number of participants with lung function response measured by oscillometry
The presence of a lung function response will be measured by oscillometry
Measured: 24 weeks after ruxolitinib initiation
Number of participants with lung function response measured by home spirometry
The presence of a lung function response will be measured by home spirometry
Measured: 24 weeks after ruxolitinib initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2021
- Study start12 Nov 2021
- Primary completion17 Sep 2025
- Study completion16 Nov 2025
Why it stopped: Study closed due to slow enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic Stem Cell Transplant (HSCT)Studies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kasiani Myers, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04908735
- Sponsor's protocol ID
- 2020-0719
- Acronym
- HSCT
- Status last verified
- Apr 2026
- First submitted
- 26 May 2021
- Last updated
- 7 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ruxolitinib for Early Lung Dysfunction After HSCT: a Phase II Study
- ClinicalTrials.gov ID
- NCT04908735
- Acronym / sponsor ID
- HSCT / 2020-0719
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study closed due to slow enrollment.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 12 Nov 2021 (actual)
- Primary completion
- 17 Sep 2025 (actual)
- Study completion
- 16 Nov 2025 (actual)
- First posted
- 1 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib Treatment | Experimental | Drug: Ruxolitinib |
Who may qualify
- Ages
- 5 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibParticipants will receive ruxolitinib orally twice daily for 24 weeks. | Drug | Ruxolitinib Treatment |
Primary outcome
Number of participants with ruxolitinib treatment response
Time frame: 6 months from early lung dysfunction diagnosis
Treatment response is defined by stable and/or improved lung function as defined by the National Institutes of Health Chronic GVHD Response Criteria Working Group.
4 secondary outcomes
Percentage of participants with JAK inhibition
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
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 home spirometry
Time frame: 24 weeks after ruxolitinib initiation
Results and safety
The study was stopped early (reason given: Study closed due to slow enrollment.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2026.
- Derived from this study: PubMed 38485149Bos 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kasiani Myers, MD principal investigator
Children's Hospital Medical Center, Cincinnati
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib in people with Hematopoietic Stem Cell Transplant (HSCT) and Bronchiolitis Obliterans (BO), run by Children's Hospital Medical Center, Cincinnati at 2 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 14 inclusion and 4 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: stopped early, as of the sponsor's last update on 7 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
The study was stopped early (reason given: Study closed due to slow enrollment.). Results are not available in the source record.
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?
Number of participants with ruxolitinib treatment response (time frame: 6 months from early lung dysfunction diagnosis). 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 NCT04908735, which also lists 1 linked publication.
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 7 Apr 2026.