Ruxolitinib for Newly Diagnosed Bronchiolitis Obliterans Syndrome

NCT05413356 | Unknown Phase 2 DMC

• 1 other identifier: ZJU-HSCT-BOS
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Bronchiolitis Obliterans Syndrome; Hematologic Malignancy

Keywords

Allogeneic Hematopoietic stem cell transplantation; chronic graft versus host disease; bronchiolitis obliterans syndrome

Outcome Measures

Primary Outcomes (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)

  3 Months

Secondary Outcomes (7)

• treatment failure rate

  3 Months

• absolute FEV1 increase

  6 Months, 9 Months, 12 Months and 24 Months

• Improvements in chronic GVHD organ specific manifestations

  6 Months, 9 Months, 12 Months and 24 Months

• Overall Survival

  2 years

• cGVHD progression-free survival

  2 years

Study Arms (1)

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

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Oral ruxolitinib twice daily

Also known as: Ruxolitinib twice daily

treatment group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• First Affiliated Hospital of Zhejiang University: lead
• Second Affiliated Hospital, School of Medicine, Zhejiang University: collaborator
• Zhejiang Provincial People's Hospital: collaborator
• The First Affiliated Hospital of Zhejiang Chinese Medical University: collaborator
• Sir Run Run Shaw Hospital: collaborator
• First Affiliated Hospital of Wenzhou Medical University: collaborator
• Ningbo No. 1 Hospital: collaborator
• The Affiliated People's Hospital of Ningbo University: collaborator
• Jinhua Central Hospital: collaborator
• Taizhou Hospital: collaborator
• Union hospital of Fujian Medical University: collaborator
• Xiangya Hospital of Central South University: collaborator

Study Sites (1)

The first Affiliated Hospital of Zhejiang University

Hangzhou, Zhejiang, 310000, China

RECRUITING

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome; Hematologic Neoplasms

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases; Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Yi Luo, M.D.

  First Affilaated Hospital of Medical School of Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yi Luo, M.D.

CONTACT

+86057187233801; luoyijr@163.com

Yibo Wu, M.D.

CONTACT

+8619858876273; wuyibo7@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2022
• First Posted: June 10, 2022
• Study Start: June 1, 2022
• Primary Completion: June 1, 2024
• Study Completion: January 1, 2025
• Last Updated: October 19, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)