Trial Outcomes & Findings for Velusetrag for the Treatment of Chronic Intestinal Pseudo-Obstruction (CIPO). (NCT NCT05724069)
NCT ID: NCT05724069
Last Updated: 2025-08-03
Results Overview
Mean change in weekly global gastrointestinal symptoms average index score (WGGSAIS) from pretreatment (PRE) to the end of each treatment period (EOT). The weekly global gastrointestinal symptoms average index score is obtained by averaging the scores for each of the 4 symptoms, abdominal pain, bloating, nausea and vomiting, assessed using a subject's e-diary. Each symptom was rated using a recall period of 7 days and a Likert scale with the following categories: 0=none, 1=mild, 2=moderate, 3=severe, 4=very severe according to its influence on usual activity. The WGGSAIS thus ranges between 0 and 4 with lower scores representing better health.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
17 participants
Primary outcome timeframe
4 weeks. The change is derived as the end of treatment period minus the pre-treatment value of the period, for each period.
Results posted on
2025-08-03
Participant Flow
Participant milestones
| Measure |
Sequence A
Eligible subjects will be treated for 4 periods with either velusetrag (2 periods) or placebo (2 periods), with a wash-out period of 2 weeks between treatment periods.
The progression order of the treatment periods varies in each sequence.
Velusetrag: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
Placebo: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
|
Sequence B
Eligible subjects will be treated for 4 periods with either velusetrag (2 periods) or placebo (2 periods), with a wash-out period of 2 weeks between treatment periods.
The progression order of the treatment periods varies in each sequence.
Velusetrag: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
Placebo: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
|
Sequence C
Eligible subjects will be treated for 4 periods with either velusetrag (2 periods) or placebo (2 periods), with a wash-out period of 2 weeks between treatment periods.
The progression order of the treatment periods varies in each sequence.
Velusetrag: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
Placebo: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
|
Sequence D
Eligible subjects will be treated for 4 periods with either velusetrag (2 periods) or placebo (2 periods), with a wash-out period of 2 weeks between treatment periods.
The progression order of the treatment periods varies in each sequence.
Velusetrag: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
Placebo: Subjects will be randomly allocated in a blinded fashion to 1 out of the 4 treatment sequences (each sequence include 4 periods, 2 periods with velusetrag and 2 periods with placebo). There will be a wash-out period between each treatment period.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
5
|
4
|
4
|
4
|
|
Overall Study
COMPLETED
|
5
|
4
|
4
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Velusetrag for the Treatment of Chronic Intestinal Pseudo-Obstruction (CIPO).
Baseline characteristics by cohort
| Measure |
Overall
n=17 Participants
demographic characteristics are summarized descriptively overall
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
57.5 years
STANDARD_DEVIATION 10.47 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
11 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 weeks. The change is derived as the end of treatment period minus the pre-treatment value of the period, for each period.Population: Modified Full Analysis Set 1 (mFAS1): the set of subjects responder/naïve to 5-HT4 agonist randomized and treated who reported data on the primary endpoint at least once during a velusetrag treatment period and at least once during a placebo treatment period. Arms were not mutually exclusive.
Mean change in weekly global gastrointestinal symptoms average index score (WGGSAIS) from pretreatment (PRE) to the end of each treatment period (EOT). The weekly global gastrointestinal symptoms average index score is obtained by averaging the scores for each of the 4 symptoms, abdominal pain, bloating, nausea and vomiting, assessed using a subject's e-diary. Each symptom was rated using a recall period of 7 days and a Likert scale with the following categories: 0=none, 1=mild, 2=moderate, 3=severe, 4=very severe according to its influence on usual activity. The WGGSAIS thus ranges between 0 and 4 with lower scores representing better health.
Outcome measures
| Measure |
Modified Full Analysis Set 1 Velusetrag
n=15 Participants
The velusetrag arm includes all subjects who took velusetrag during the 4 periods/4 sequences crossover, multiple (n=1) trial.
|
Modified Full Analysis Set 1 Placebo
n=15 Participants
The Placebo arm includes all subjects who took placebo during the 4 periods/4 sequences cross-over, multiple (n=1) trial.
|
|---|---|---|
|
Mean Change in Weekly Global Gastrointestinal Symptoms Average Index Score (WGGSAIS).
|
-0.424 score
Standard Deviation 0.6926
|
-0.185 score
Standard Deviation 0.6876
|
Adverse Events
Velusetrag 15 mg Once a Day
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo Once a Day
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Velusetrag 15 mg Once a Day
n=17 participants at risk
|
Placebo Once a Day
n=17 participants at risk
|
|---|---|---|
|
Vascular disorders
hypertension
|
5.9%
1/17 • Number of events 1 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
General disorders
Influenza like illness
|
11.8%
2/17 • Number of events 3 • 6 months
|
11.8%
2/17 • Number of events 2 • 6 months
|
|
General disorders
Pyrexia
|
5.9%
1/17 • Number of events 1 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
General disorders
Fatigue
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
General disorders
Injection site pruritus
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Reproductive system and breast disorders
Pelvic pain
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Catarrh
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Injury, poisoning and procedural complications
Sunburn
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 2 • 6 months
|
|
Cardiac disorders
Palpitations
|
5.9%
1/17 • Number of events 2 • 6 months
|
0.00%
0/17 • 6 months
|
|
Cardiac disorders
Sinus Bradycardia
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 2 • 6 months
|
|
Nervous system disorders
Headache
|
17.6%
3/17 • Number of events 4 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Blood and lymphatic system disorders
Anemia Vitamin B12 deficiency
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Ear and labyrinth disorders
Ear pain
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Eye disorders
Eye irritation
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Eye disorders
Eye pruritus
|
5.9%
1/17 • Number of events 2 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Eye disorders
Eye swelling
|
5.9%
1/17 • Number of events 2 • 6 months
|
5.9%
1/17 • Number of events 5 • 6 months
|
|
Eye disorders
Ocular hyperaemia
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 4 • 6 months
|
|
Gastrointestinal disorders
Constipation
|
5.9%
1/17 • Number of events 1 • 6 months
|
5.9%
1/17 • Number of events 2 • 6 months
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Intestinal Pseudo-obstruction
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Toothache
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Skin and subcutaneous tissue disorders
skin depigmentation
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Urinary Retention
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.9%
1/17 • Number of events 1 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Athralgia
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Musculoskeletal and connective tissue disorders
bone pain
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Muscle contracturer
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.9%
1/17 • Number of events 1 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
|
Infections and infestations
Urinary Tract Infection
|
11.8%
2/17 • Number of events 2 • 6 months
|
0.00%
0/17 • 6 months
|
|
Infections and infestations
COVID-19
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Infections and infestations
Cystitis
|
5.9%
1/17 • Number of events 1 • 6 months
|
0.00%
0/17 • 6 months
|
|
Infections and infestations
Influenza
|
0.00%
0/17 • 6 months
|
5.9%
1/17 • Number of events 1 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60