Bronchiolitis Obliterans SyndromePhase 2NCT05413356
Ruxolitinib for Newly Diagnosed Bronchiolitis Obliterans Syndrome
A Phase 2 study testing Ruxolitinib in people with Bronchiolitis Obliterans Syndrome and Hematologic Malignancy, run by First Affiliated Hospital of Zhejiang University at 1 site in China.
Trial summary
Lung is one of the target organs in chronic graft versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Bronchiolitis obliterans syndrome (BOS) after allo-HSCT was a clinical syndrome characterized by persistent airflow restriction which is the result of lung cGVHD. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Bronchiolitis Obliterans Syndrome, Hematologic Malignancy
- 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
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2022to 1 Jan 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female; 18-65 years old
- Diagnosis of BOS after allo-HCT defined as the 2014 NIH criteria
- Life expectancy > 6 months at the time of enrollment
- At least 4 weeks since initiation of the most recent systemic therapy for cGVHD or BOS
- 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
- Hepatic dysfunction: transaminases (ALT, AST) > 5X ULN and/or total bilirubin > 3X ULN
- Hematologic dysfunction: absolute neutrophil count <1000/μL, platelet cout <30*10E9/L, and/or Hgb < 8 g/dL
- Renal dysfunction: calculated creatinine clearance < 30 mL/min (Cockcroft-Gault formula)
- previously received second-line treatment or any drugs in clinical trials for cGVHD
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The first Affiliated Hospital of Zhejiang University
Hangzhou, Zhejiang, 310000
Yi Luo, M.D., +86057187233801, luoyijr@163.com
About this study
Official title: Ruxolitinib for Newly Diagnosed Bronchiolitis Obliterans Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation
Lung is one of the target organs in chronic graft versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Bronchiolitis obliterans syndrome (BOS) after allo-HSCT was a clinical syndrome characterized by persistent airflow restriction which is the result of lung cGVHD. BOS is one of the main causes of late mortality after allo-HSCT, severely restricting the daily activities and respiratory function of patients. It limits the quality of life and increased the non-relapse mortality (NRM) after allo-HSCT. Currently, the first-line treatment for BOS is FAM ( oral fluticasone, azithromycin and montelukast). However, more than 50% of patients develop as steroids resistant (SR)-BOS, and SR-BOS has a poor prognosis and irreversible impaired lung function. Ruxolitinib is an effective drug in the treatment of SR-cGVHD. This is a phase Ⅱ prospective clinical study to explore the efficacy and safety of ruxolitinib as a first-line treatment for newly diagnosed BOS after allo-HSCT.
Read the detailed description
The incidence of chronic graft versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) was 30%-70%, Which extremely limited the quality of life and the survival of patients after allo-HSCT. Lung is one of the target organs in cGVHD after allo-HSCT. Bronchiolitis obliterans syndrome (BOS) after allo-HSCT was a clinical syndrome characterized by persistent airflow restriction which is the result of lung cGVHD. BOS is one of the main causes of late mortality after allo-HSCT, severely restricting the daily activities and respiratory function of patients. It limits the quality of life and increased the non-relapse mortality (NRM) after allo-HSCT. Currently, the first-line treatment for BOS is FAM ( oral fluticasone, azithromycin and montelukast). However, more than 50% of patients develop as steroids resistant (SR)-BOS, and SR-BOS has a poor prognosis and irreversible impaired lung function. Ruxolitinib is an effective drug in the treatment of SR-cGVHD. This is a phase Ⅱ prospective clinical study to explore the efficacy and safety of ruxolitinib as a first-line treatment for newly diagnosed BOS after allo-HSCT.
- Being tested
- Ruxolitinib
- Keywords
- Allogeneic Hematopoietic stem cell transplantation, chronic graft versus host disease, bronchiolitis obliterans syndrome
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called Ruxolitinib twice daily
Oral ruxolitinib twice daily
| Study group | Role | What they receive |
|---|---|---|
| treatment group | Experimental | Ruxolitinib twice daily treatment, combined with steroids 1mg/kg/day for two weeks, and tampering 0.25 mg/kg/day every week Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 3 Months
Secondary outcomes (7)
treatment failure rate
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)
Measured: 3 Months
absolute FEV1 increase
The proportion of participants with a sustained, absolute FEV1 increase by ≥ 10% after treatment with ruxolitinib (compared to baseline measure prior to study enrollment)
Measured: 6 Months, 9 Months, 12 Months and 24 Months
Improvements in chronic GVHD organ specific manifestations
mprovements in chronic GVHD organ specific manifestations will be categorized according to the NIH chronic GVHD consensus criteria.
Measured: 6 Months, 9 Months, 12 Months and 24 Months
Overall Survival
The proportion of patients survival at two years after enrollment of ruxolitinib treatment
Measured: 2 years
cGVHD progression-free survival
Participants alive without cGVHD progression are censored at the date of last disease evaluation
Measured: 2 years
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 2 years
The change of systemic corticosteroid dose over time
The change of systemic corticosteroid dose over time during the treatment of BOS
Measured: From the start of treatment until the end of treatment, up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2022
- Study start1 Jun 2022
- Primary completion1 Jun 2024 (estimated)
- Study completion1 Jan 2025 (estimated)
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 Bronchiolitis Obliterans SyndromeStudies of the same condition, open and under way.
- More trials run by First Affiliated Hospital of Zhejiang UniversityThe 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
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Second Affiliated Hospital, Zhejiang University, School of Medicine, Zhejiang Provincial People's Hospital, The First Affiliated Hospital of Zhejiang Chinese Medical University, Sir Run Run Shaw Hospital, First Affiliated Hospital of Wenzhou Medical University, Ningbo No. 1 Hospital, The Affiliated People's Hospital of Ningbo University, Jinhua Central Hospital, Taizhou Hospital, Union hospital of Fujian Medical University, Xiangya Hospital of Central South University
- Responsible party
- Sponsor
- Investigators
- Yi Luo, M.D., Principal investigator, First Affilaated Hospital of Medical School of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05413356
- Sponsor's protocol ID
- ZJU-HSCT-BOS
- Status last verified
- May 2022
- First submitted
- 7 Jun 2022
- Last updated
- 19 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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 19 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ruxolitinib for Newly Diagnosed Bronchiolitis Obliterans Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT05413356
- Acronym / sponsor ID
- ZJU-HSCT-BOS
- Keywords
- Allogeneic Hematopoietic stem cell transplantation, chronic graft versus host disease, bronchiolitis obliterans syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Second Affiliated Hospital, Zhejiang University, School of Medicine, Zhejiang Provincial People's Hospital, The First Affiliated Hospital of Zhejiang Chinese Medical University, Sir Run Run Shaw Hospital, First Affiliated Hospital of Wenzhou Medical University, Ningbo No. 1 Hospital, The Affiliated People's Hospital of Ningbo University, Jinhua Central Hospital, Taizhou Hospital, Union hospital of Fujian Medical University, Xiangya Hospital of Central South University
- Study start
- 1 Jun 2022 (actual)
- Primary completion
- 1 Jun 2024 (estimated)
- Study completion
- 1 Jan 2025 (estimated)
- First posted
- 10 Jun 2022 (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.
Lung is one of the target organs in chronic graft versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Bronchiolitis obliterans syndrome (BOS) after allo-HSCT was a clinical syndrome characterized by persistent airflow restriction which is the result of lung cGVHD. BOS is one of the main causes of late mortality after allo-HSCT, severely restricting the daily activities and respiratory function of patients. It limits the quality of life and increased the non-relapse mortality (NRM) after allo-HSCT. Currently, the first-line treatment for BOS is FAM ( oral fluticasone, azithromycin and montelukast). However, more than 50% of patients develop as steroids resistant (SR)-BOS, and SR-BOS has a poor prognosis and irreversible impaired lung function. Ruxolitinib is an effective drug in the treatment of SR-cGVHD. This is a phase Ⅱ prospective clinical study to explore the efficacy and safety of ruxolitinib as a first-line treatment for newly diagnosed BOS after allo-HSCT.
Detailed description
The incidence of chronic graft versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) was 30%-70%, Which extremely limited the quality of life and the survival of patients after allo-HSCT. Lung is one of the target organs in cGVHD after allo-HSCT. Bronchiolitis obliterans syndrome (BOS) after allo-HSCT was a clinical syndrome characterized by persistent airflow restriction which is the result of lung cGVHD. BOS is one of the main causes of late mortality after allo-HSCT, severely restricting the daily activities and respiratory function of patients. It limits the quality of life and increased the non-relapse mortality (NRM) after allo-HSCT. Currently, the first-line treatment for BOS is FAM ( oral fluticasone, azithromycin and montelukast). However, more than 50% of patients develop as steroids resistant (SR)-BOS, and SR-BOS has a poor prognosis and irreversible impaired lung function. Ruxolitinib is an effective drug in the treatment of SR-cGVHD. This is a phase Ⅱ prospective clinical study to explore the efficacy and safety of ruxolitinib as a first-line treatment for newly diagnosed BOS after allo-HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment group | Experimental | Drug: RuxolitinibRuxolitinib twice daily treatment, combined with steroids 1mg/kg/day for two weeks, and tampering 0.25 mg/kg/day every week |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female; 18-65 years old
- Diagnosis of BOS after allo-HCT defined as the 2014 NIH criteria
- Life expectancy > 6 months at the time of enrollment
- At least 4 weeks since initiation of the most recent systemic therapy for cGVHD or BOS
- The ability to understand and willingness to sign a written consent document
Key exclusion criteria (7 listed)
- 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
- Hepatic dysfunction: transaminases (ALT, AST) > 5X ULN and/or total bilirubin > 3X ULN
- Hematologic dysfunction: absolute neutrophil count <1000/μL, platelet cout <30*10E9/L, and/or Hgb < 8 g/dL
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 |
|---|---|---|
| RuxolitinibAlso: Ruxolitinib twice dailyOral ruxolitinib twice daily | Drug | treatment group |
Primary outcome
absolute FEV1 increase
Time frame: 3 Months
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)
7 secondary outcomes
treatment failure rate
Time frame: 3 Months
absolute FEV1 increase
Time frame: 6 Months, 9 Months, 12 Months and 24 Months
Improvements in chronic GVHD organ specific manifestations
Time frame: 6 Months, 9 Months, 12 Months and 24 Months
Overall Survival
Time frame: 2 years
cGVHD progression-free survival
Time frame: 2 years
The incidence and types of serious adverse events
Time frame: From the start of treatment until 30 days after the end of treatment, up to 2 years
The change of systemic corticosteroid dose over time
Time frame: From the start of treatment until the end of treatment, up to 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2022.
- 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.
- Individual participant dataSharing planned: No
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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.
Yi Luo, M.D. principal investigator
First Affilaated Hospital of Medical School of Zhejiang University
1 site 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 Bronchiolitis Obliterans Syndrome and Hematologic Malignancy, run by First Affiliated Hospital of Zhejiang University at 1 site in China.
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 5 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 19 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yi Luo, M.D. (+86057187233801, luoyijr@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
absolute FEV1 increase (time frame: 3 Months). 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 NCT05413356, 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 19 Oct 2022.