Study of EXE-346 Live Biotherapeutic to Reduce High Bowel Movement Frequency in Subjects with an IPAA (PROF)
- Registration Number
- NCT05938465
- Lead Sponsor
- Exegi Pharma, LLC
- Brief Summary
The aim of this study is to assess the safety and preliminary efficacy of treatment with EXE-346, a live biotherapeutic, which may reduce bowel movement frequency in patients with an ileal pouch-anal anastomosis (IPAA) and lead to a higher quality of life.
- Detailed Description
The aim of this study is to assess the safety and preliminary efficacy of treatment with EXE-346 which may reduce bowel movement frequency in patients with an IPAA and lead to a higher quality of life. EXE-346 is a live biotherapeutic product containing a fixed proportion mixture of 8 individual bacterial strains.
The Phase 1b part of the study is an open label (OL), single-arm study to assess the safety of EXE-346 administered orally for up to 4 weeks.
The Phase 2 part of the study is a randomized, double-blinded study to assess the safety and efficacy of the same dose of EXE-346 administered orally for up to 8 weeks, compared with placebo. Subjects who complete the Phase 2 double-blinded part of the study will be eligible to participate in an optional open label extension phase to receive EXE-346 for up to 8 weeks.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 50
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Phase 1b Open Label EXE-346 EXE-346 live biotherapeutic product, 1500x10\^9 colony forming units (CFU) twice daily (BID), 4 weeks Phase 2: Active Arm EXE-346 EXE-346 live biotherapeutic product, 1500x10\^9 CFU BID, 8 weeks Phase 2: Placebo Arm Placebo Powder containing same inactive ingredients as EXE-346 but none of the active ingredients, BID, 8 weeks Phase 2 Open Label Extension (optional) EXE-346 EXE-346 live biotherapeutic product, 1500x10\^9 CFU BID, 8 weeks
- Primary Outcome Measures
Name Time Method Phase 2 Open Label: Incidence, Severity, Relatedness, and Frequency of Treatment Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) 8 weeks To assess the safety of EXE-346 using incidence, severity, relationship to study treatment, and frequency of TEAEs and SAEs.
Phase 2: Number of Participants with Abnormal Physical Examinations 8 weeks To assess the safety of EXE-346 using abnormal findings in physical examinations after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 2: Number of Participants with Abnormal Safety Labs 8 weeks To assess the safety of EXE-346 using markedly abnormal findings in safety labs after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 2: Change in Total Daily Bowel Movement Frequency 8 weeks To assess the efficacy of EXE-346 to reduce the total daily bowel movement frequency using change in average daily bowel movement frequency from baseline to each post-baseline week
Phase 1b: Number of Participants with Abnormal Vital Signs 4 weeks To assess the safety of EXE-346 using abnormal findings in vital sign readings after the start of study treatment that suggest a clinically significant worsening of medical issue, including blood pressure.
Phase 1b: Number of Participants with Abnormal Safety Labs 4 weeks To assess the safety of EXE-346 using markedly abnormal findings in safety labs after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 2: Number of Participants with Abnormal Vital Signs 8 weeks To assess the safety of EXE-346 using abnormal findings in vital sign readings after the start of study treatment that suggest a clinically significant worsening of medical issue, including blood pressure.
Phase 2 Open Label: Number of Participants with Abnormal Safety Labs 8 weeks To assess the safety of EXE-346 using markedly abnormal findings in safety labs after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 1b: Study Treatment Discontinuation Due to Treatment Emergent Adverse Events (TEAEs) 4 weeks To assess the safety of EXE-346 using study treatment discontinuation due to TEAE(s).
Phase 2 Open Label: Number of Participants with Abnormal Physical Examinations 8 weeks To assess the safety of EXE-346 using abnormal findings in physical examinations after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 2 Open Label: Number of Participants with Abnormal Vital Signs 8 weeks To assess the safety of EXE-346 using abnormal findings in vital sign readings after the start of study treatment that suggest a clinically significant worsening of medical issue, including blood pressure.
Phase 2 Open Label: Study Treatment Discontinuation Due to Treatment Emergent Adverse Events (TEAEs) 8 weeks To assess the safety of EXE-346 using study treatment discontinuation due to TEAE(s).
Phase 1b: Incidence, Severity, Relatedness, and Frequency of Treatment Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) 4 weeks To assess the safety of EXE-346 using incidence, severity, relationship to study treatment, and frequency of treatment emergent adverse events (TEAE) and serious adverse events (SAE).
Phase 1b: Number of Participants with Abnormal Physical Examinations 4 weeks To assess the safety of EXE-346 using abnormal findings in physical examinations after the start of study treatment that suggest a clinically significant worsening of medical issue.
Phase 2: Incidence, Severity, Relatedness, and Frequency of Treatment Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) 8 weeks To assess the safety of EXE-346 using incidence, severity, relationship to study treatment, and frequency of TEAEs and SAEs.
Phase 2: Study Treatment Discontinuation Due to Treatment Emergent Adverse Events (TEAEs) 8 weeks To assess the safety of EXE-346 using study treatment discontinuation due to TEAE(s).
- Secondary Outcome Measures
Name Time Method Phase 2: Bowel Movement Consistency 8 weeks To assess the effect of EXE-346 on bowel movement consistency using change in average consistency of daily bowel movements from baseline to each post-baseline week
Phase 1b: Bowel Movement Consistency 4 weeks To assess the effect of EXE-346 on bowel movement consistency using change in average consistency of daily bowel movements from baseline to each post-baseline week
Phase 2: Nighttime Awakening Frequency 8 weeks To assess the effect of EXE 346 on nighttime awakening frequency using change in average nighttime awakenings for bowel movements from baseline to each post-baseline week
Phase 1b: Nighttime Awakening Frequency 4 weeks To assess the effect of EXE-346 on nighttime awakening frequency using change in average nighttime awakenings for bowel movements from baseline to each post-baseline week
Phase 1b: Bowel Movement Frequency 4 weeks To assess the effect of EXE-346 on bowel movement frequency using change in average daily bowel movement frequency from baseline to each post-baseline week
Trial Locations
- Locations (8)
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Mayo Clinic - Florida (Inflammatory Bowel Disease Center)
🇺🇸Jacksonville, Florida, United States
Corewell Health
🇺🇸Grand Rapids, Michigan, United States
Mayo Clinic Department of Gastroenterology
🇺🇸Rochester, Minnesota, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
NYU Langone Health
🇺🇸New York, New York, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Penn State Health (Milton S. Hershey Medical Center)
🇺🇸Hershey, Pennsylvania, United States