ROCKaspire: A Study to Test How Effective Belumosudil Tablets Are for Treating Adult Participants With Chronic Lung Allograft Dysfunction
Study Details
Study Description
Brief Summary
This double-blind, randomized, placebo-controlled, multinational, multicenter, parallel-group, Phase 3, 2-arm, study will investigate the efficacy and safety of belumosudil compared with placebo, both administered on top of azithromycin and standard-of-care regimen of immunosuppression in male or female participants at least 1 year after bilateral lung transplant, who are at least 18 years of age and who have evidence of progressive CLAD despite azithromycin therapy.
Study details include:
The study duration will be up to 31 weeks for participants not entering the open-label extension (OLE) period and up to 57 weeks for participants entering the OLE period but not the long-term OLE.
The treatment duration will be up to 26 weeks for participants not entering the OLE period and up to 52 weeks for participants entering the OLE period but not the long-term OLE.
The number of visits will be up to 10 visits for participants not entering the OLE period and up to 16 visits for participants entering the OLE period but not the long-term OLE.
For participants who enter the long-term OLE, treatment and study participation will continue with visits every 12 weeks per protocol specifications.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Belumosudil + Azithromycin Participants will receive 200 mg belumosudil orally once daily |
Drug: Belumosudil
Tablet, Oral
Other Names:
Drug: Azithromycin
Tablet, Oral
|
| Placebo Comparator: Placebo + Azithromycin Participants will receive placebo orally once daily |
Drug: Azithromycin
Tablet, Oral
Drug: Placebo
Tablet, Oral
|
Outcome Measures
Primary Outcome Measures
- Percent change from baseline to Week 26 in forced expiratory volume in 1 second (FEV1) [Baseline to Week 26]
Secondary Outcome Measures
- Response rate at Week 26 [Baseline to Week 26]
Defined as the proportion of participants with ≤10% decline in FEV1 at Week 26 compared with baseline
- Absolute change from baseline to Week 26 in FEV1 [Baseline to Week 26]
- Absolute change from baseline to Week 26 in percent predicted FEV1 [Baseline to Week 26]
- Percent change from baseline to Week 26 in forced vital capacity (FVC) [Baseline to Week 26]
- Absolute change from baseline to Week 26 in FVC [Baseline to Week 26]
- Absolute change from baseline to Week 26 in percent predicted FVC [Baseline to Week 26]
- Percent change from baseline to Week 26 in total lung capacity (TLC) [Baseline to Week 26]
- Time to CLAD progression during the double-blind treatment period [Baseline to Week 26]
Defined as the first of the following events: >10% decline in FEV1 compared with baseline, or change in CLAD stage, or re-transplantation, or death
- Time to re-transplantation or death [Up to 7 days after the administration of last dose of study drug]
- Percent change from baseline to Week 26 in 6-minute walk distance [Baseline to Week 26]
- Absolute change from baseline to Week 26 in 6-minute walk distance [Baseline to Week 26]
- Absolute change from baseline to Week 26 in 6-minute walk nadir oxygen saturation [Baseline to Week 26]
- Change from baseline to Week 26 in Saint George Respiratory Questionnaire (SGRQ) [Baseline to Week 26]
Total score and scores by domain
- Change from baseline to Week 26 in EQ-5D-5L [Baseline to Week 26]
Visual analogue scale and individual dimensions
- Change from baseline to Week 26 in Patient-Reported Outcomes Measurement Information System-29 profile version 2.1 [Baseline to Week 26]
Patient-Reported Outcomes Measurement Information System-29 (PROMIS-29) profile V2.1 (domain scores)
- Number of participants with adverse events (AEs) and serious adverse events (SAEs) [Up to 7 days after the administration of last dose of study drug]
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), as well as laboratory abnormalities during the double-blind treatment period and the open-label extensions
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Participant ≥1 year post bilateral lung transplantation at the time of screening
-
Participants presenting with CLAD
-
Participants who have received at least 8 weeks of azithromycin
Exclusion Criteria:
-
Lung function decline that can be explained by non-CLAD causes including but not limited to acute lung allograft rejection (>A1), antibody-mediated rejection within 4 weeks of screening, airway stenosis, or tracheobronchomalacia
-
Participants who have received other treatments for CLAD
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigational Site Number :3760001 | Petah-Tikva | Israel | 49100 |
Sponsors and Collaborators
- Sanofi
Investigators
- Study Director: Clinical Sciences & Operations, Sanofi
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- EFC17801
- U1111-1280-6777
- 2023-503462-23