Trial Outcomes & Findings for Efficacy and Safety of Oral OPS-2071 in Participants With Crohn's Disease Showing Symptoms of Active Inflammation (NCT NCT03850509)
NCT ID: NCT03850509
Last Updated: 2021-06-15
Results Overview
Clinical remission was defined as CDAI score \<150 at Week 12. The CDAI evaluated severity of signs and symptoms of Chron's Disease. Some components of the CDAI were reported by the investigator (physical examination for the presence of an abdominal mass and extraintestinal complications, laboratory results for hematocrit levels, and weight) while other components were determined with data collected in a participant diary (number of liquid or soft stools, number of antidiarrheal medications, abdominal pain score, and general well-being). The index values of 150 and below were associated with quiescent disease; values above that indicated active disease, values \>=220 indicated moderate to severe disease, and values above 450 were seen with extremely severe disease.
TERMINATED
PHASE2
3 participants
Week 12
2021-06-15
Participant Flow
Participants were enrolled at 2 investigative sites in the United States and Poland from 25 February 2020 to 22 May 2020.
Participants with Crohn's disease showing symptoms of active inflammation despite ongoing treatment were enrolled and randomized in this study to receive OPS-2071 300 mg BID and OPS-2071 matched placebo. Participants were also to be randomized in OPS-2071 150 mg and 600 mg, however, the study was terminated before the enrollment of participants in the respective arms.
Participant milestones
| Measure |
OPS-2071 300 mg BID
Participants received OPS-2071 300 mg, tablets, orally, twice daily (BID) in the morning and evening (8 to 12 hours apart) with 240 milliliters (mL) of water for up to 6 weeks.
|
Placebo
Participants received OPS-2071-matched placebo, tablets, orally, BID in morning and evening (8 to 12 hours apart) with 240 mL of water for up to 4 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
OPS-2071 300 mg BID
Participants received OPS-2071 300 mg, tablets, orally, twice daily (BID) in the morning and evening (8 to 12 hours apart) with 240 milliliters (mL) of water for up to 6 weeks.
|
Placebo
Participants received OPS-2071-matched placebo, tablets, orally, BID in morning and evening (8 to 12 hours apart) with 240 mL of water for up to 4 weeks.
|
|---|---|---|
|
Overall Study
Study Terminated by the Sponsor (Due to COVID-19 Situation)
|
2
|
1
|
Baseline Characteristics
Efficacy and Safety of Oral OPS-2071 in Participants With Crohn's Disease Showing Symptoms of Active Inflammation
Baseline characteristics by cohort
| Measure |
OPS-2071 300 mg BID
n=2 Participants
Participants received OPS-2071 300 mg, tablets, orally, twice daily (BID) in the morning and evening (8 to 12 hours apart) with 240 milliliters (mL) of water for up to 6 weeks.
|
Placebo
n=1 Participants
Participants received OPS-2071-matched placebo, tablets, orally, BID in morning and evening (8 to 12 hours apart) with 240 mL of water for up to 4 weeks.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
Clinical remission was defined as CDAI score \<150 at Week 12. The CDAI evaluated severity of signs and symptoms of Chron's Disease. Some components of the CDAI were reported by the investigator (physical examination for the presence of an abdominal mass and extraintestinal complications, laboratory results for hematocrit levels, and weight) while other components were determined with data collected in a participant diary (number of liquid or soft stools, number of antidiarrheal medications, abdominal pain score, and general well-being). The index values of 150 and below were associated with quiescent disease; values above that indicated active disease, values \>=220 indicated moderate to severe disease, and values above 450 were seen with extremely severe disease.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
Endoscopic response was defined as a reduction of the SES-CD by at least 50%, at Week 12. The SES-CD is a total score that indicates endoscopic disease activity status based on endoscopy results regarding the size of ulcers, surface ulceration, affected surface size, and presence of luminal narrowing. Each item is scored from 0-3, with total score from 0-60 . Higher score indicates more severe endoscopic activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (Day 1) and Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
The SES-CD is a total score that indicates endoscopic disease activity status based on endoscopy results regarding the size of ulcers, surface ulceration, affected surface size, and presence of luminal narrowing. Each item is scored from 0-3, with a total score from 0-60. A higher score indicates more severe endoscopic activity. A negative change from baseline indicates improvement.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
PRO-2 remission was defined as stool frequency =\< 3 times per day and abdominal pain =\< 1 at Week 12. The PRO-2 is a symptom control measure based on 2 participant-reported components (stool frequency and abdominal pain) of the CDAI (on an 11-point scale where 0=no pain to 10=worst imaginable pain). A weekly score was calculated for the liquid or soft stool frequency and a separate weekly score was calculated for abdominal pain, in each case based on daily symptom reporting. PRO2 is a composite index consisting of weighted scoring of both variables. PRO2 scores ranges from 0 to approximately 45, higher score indicates higher disease activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
Clinical response was defined as at least a 25% decrease in the CDAI score at Week 12. The CDAI evaluated the severity of signs and symptoms of Chron's Disease. Some components of the CDAI were reported by the investigator (physical examination for the presence of an abdominal mass and extraintestinal complications, laboratory results for hematocrit levels, and weight) while other components were determined with data collected in a participant diary (number of liquid or soft stools, number of antidiarrheal medications, abdominal pain score, and general well-being). The index values of 150 and below were associated with quiescent disease; values above that indicated active disease, values \>=220 indicated moderate to severe disease, and values above 450 were seen with extremely severe disease.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
Endoscopic remission was defined as an SES-CD total score of 0 to 2; or a score of 0 to 4, with no individual subscore greater than 1 at Week 12. The SES-CD is a total score that indicates endoscopic disease activity status based on endoscopy results regarding the size of ulcers, surface ulceration, affected surface size, and presence of luminal narrowing. Each item is scored from 0-3, with a total score from 0-60. A higher score indicates more severe endoscopic activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 12Population: This outcome measure was not analyzed as no participants reached Week 12 time point due to early termination of the study.
Percentage of participants who had a decrease of at least =\> 100 points in CDAI scores were to be reported. The CDAI evaluated the severity of signs and symptoms of Chron's Disease. Some components of the CDAI were reported by the investigator (physical examination for the presence of an abdominal mass and extraintestinal complications, laboratory results for hematocrit levels, and weight) while other components were determined with data collected in a participant diary (number of liquid or soft stools, number of antidiarrheal medications, abdominal pain score, and general well-being). The index values of 150 and below were associated with quiescent disease; values above that indicated active disease, values \>=220 indicated moderate to severe disease, and values above 450 were seen with extremely severe disease.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the signing of the informed consent form up to early termination (up to approximately 9 weeks)Population: Safety Population included all participants who were administered at least one dose of IMP.
An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant and that does not necessarily have a causal relationship with the treatment. An SAE is defined as any fatal; life-threatening; persistently or significantly disabling or incapacitating; required in-patient hospitalization or prolonged hospitalization; a congenital anomaly/birth defect; or other medically significant event that, based upon appropriate medical judgment, may have jeopardized the subject and may have required medical or surgical intervention. A TEAE is defined as an AE that occurred after the administration of investigational medicinal product (IMP).
Outcome measures
| Measure |
OPS-2071 300 mg BID
n=2 Participants
Participants received OPS-2071 300 mg, tablets, orally, twice daily (BID) in the morning and evening (8 to 12 hours apart) with 240 milliliters (mL) of water for up to 6 weeks.
|
Placebo
n=1 Participants
Participants received OPS-2071-matched placebo, tablets, orally, BID in morning and evening (8 to 12 hours apart) with 240 mL of water for up to 4 weeks.
|
|---|---|---|
|
Percentage of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) and Serious Adverse Event (SAE)
|
50.0 percentage of participants
|
0 percentage of participants
|
Adverse Events
OPS-2071 300 mg BID
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
OPS-2071 300 mg BID
n=2 participants at risk
Participants received OPS-2071 300 mg, tablets, orally, twice daily (BID) in the morning and evening (8 to 12 hours apart) with 240 milliliters (mL) of water for up to 6 weeks.
|
Placebo
n=1 participants at risk
Participants received OPS-2071-matched placebo, tablets, orally, BID in morning and evening (8 to 12 hours apart) with 240 mL of water for up to 4 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Flatulence
|
50.0%
1/2 • Number of events 1 • From the signing of the informed consent form up to early termination (up to approximately 9 weeks)
Safety Analysis Set (SAS) population included all participants who were administered at least one dose of the investigational medicinal product (IMP).
|
0.00%
0/1 • From the signing of the informed consent form up to early termination (up to approximately 9 weeks)
Safety Analysis Set (SAS) population included all participants who were administered at least one dose of the investigational medicinal product (IMP).
|
Additional Information
Global Clinical Development
Otsuka Pharmaceutical Development & Commercialization, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor reserves the right to review results publications prior to public release and can delay such publications for a period greater than 60 days but no more than 120 days from the date that the publication is submitted to the Sponsor for review. Sponsor can require changes to the publication to protect Sponsor's intellectual property rights and/or confidential information and reserves the right to limit publication timing and scope of data published based on the number of study locations.
- Publication restrictions are in place
Restriction type: OTHER