Trial Outcomes & Findings for Metabolic Balance Study of Apraglutide in Patients With Short Bowel Syndrome, Intestinal Failure (SBS-IF) and Colon-in-Continuity (CIC) (NCT NCT04964986)
NCT ID: NCT04964986
Last Updated: 2025-06-18
Results Overview
A TEAE was any unfavorable and unintended sign, symptom, or disease temporally associated with apraglutide, whether or not related, that occurred or worsened after the dose of apraglutide. A serious TEAE was defined as any TEAE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. Clinically significant changes from baseline in clinical chemistry, hematology, hemostasis, anti-drug antibodies (ADAs), and urine analysis were reported as adverse events. Adverse events of special interest (AESI) included injection site reaction, gastrointestinal obstruction, gallbladder, biliary, and pancreatic disease, fluid overload, colorectal polyps, malignancies.
COMPLETED
PHASE2
10 participants
Day 1 up to approximately 55 weeks
2025-06-18
Participant Flow
Of the 10 participants with SBS-IF and CIC enrolled, 9 participants were treated and analyzed. The participant who enrolled but was not treated was not included in any study analysis.
Participant milestones
| Measure |
Apraglutide SC Injections, Once Weekly
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Metabolic Balance Study of Apraglutide in Patients With Short Bowel Syndrome, Intestinal Failure (SBS-IF) and Colon-in-Continuity (CIC)
Baseline characteristics by cohort
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Age, Continuous
|
46.8 years
STANDARD_DEVIATION 17.46 • n=5 Participants
|
|
Age, Customized
18-64 years
|
8 Participants
n=5 Participants
|
|
Age, Customized
65-84 years
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 up to approximately 55 weeksA TEAE was any unfavorable and unintended sign, symptom, or disease temporally associated with apraglutide, whether or not related, that occurred or worsened after the dose of apraglutide. A serious TEAE was defined as any TEAE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. Clinically significant changes from baseline in clinical chemistry, hematology, hemostasis, anti-drug antibodies (ADAs), and urine analysis were reported as adverse events. Adverse events of special interest (AESI) included injection site reaction, gastrointestinal obstruction, gallbladder, biliary, and pancreatic disease, fluid overload, colorectal polyps, malignancies.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one treatment-related TEAE
|
5 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one TESAE
|
3 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one treatment-related TESAE
|
1 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one treatment-emergent AESI
|
3 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one TEAE leading to dose interruption
|
1 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one TEAE leading to permanent dose discontinuation
|
0 Participants
|
|
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
At least one TEAE
|
9 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 48Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Applicable to Protocol V3.0 implemented in France (classed as secondary endpoint in Protocol V4.0 \[implemented in Belgium\]). The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Absorption of Energy Over Metabolic Balance (MB) Periods From Baseline at Week 48
|
1133.963 kJ/day
Standard Deviation 1399.8793
|
SECONDARY outcome
Timeframe: Baseline, Week 4, Week 24, and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52
Week 4
|
-0.73 percentage change in PS volume
Standard Deviation 2.709
|
|
Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52
Week 24
|
-39.99 percentage change in PS volume
Standard Deviation 22.588
|
|
Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52
Week 52
|
-52.44 percentage change in PS volume
Standard Deviation 29.192
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change From Baseline in Actual Weekly PS Volume at Weeks 24 and 52
Week 24
|
-3510.000 mL
Standard Deviation 1893.7859
|
|
Absolute Change From Baseline in Actual Weekly PS Volume at Weeks 24 and 52
Week 52
|
-4701.778 mL
Standard Deviation 2389.9652
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Participants were considered to have a reduction of at least one day per week of PS from Baseline (incl. extra fluids) if the number of days with PS from weekly PS diary data recorded for the corresponding analysis timepoint was smaller compared to the number of days with PS from weekly PS diary data for Baseline.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Number of Participants Who Achieved a Reduction of at Least 1 Day Per Week of PS From Baseline at Weeks 24 and 52
Week 24
|
5 Participants
|
|
Number of Participants Who Achieved a Reduction of at Least 1 Day Per Week of PS From Baseline at Weeks 24 and 52
Week 52
|
7 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Clinical response was defined as a 20% reduction of PS volume from Baseline. The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Number of Participants Considered Clinical Responders at Weeks 24 and 52
Week 24
|
7 Participants
|
|
Number of Participants Considered Clinical Responders at Weeks 24 and 52
Week 52
|
9 Participants
|
SECONDARY outcome
Timeframe: Weeks 24 and 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Enteral autonomy was defined as a participant not receiving PS for hydration or parenteral nutrition (PN) for calories. Participants may have still receive minimal fluid to maintain patency of the central line or for specific elemental/micro-nutrient needs (e.g., \<100 mL fluid for administration of magnesium).
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Number of Participants Who Achieved Enteral Autonomy at Weeks 24 and 52
Week 24
|
0 Participants
|
|
Number of Participants Who Achieved Enteral Autonomy at Weeks 24 and 52
Week 52
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
PN was defined as PS that includes protein, carbohydrate, fat, vitamins, and/or trace elements.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Total Energy in PN From Baseline at Weeks 24 and 52
Week 24
|
-2763.889 kcal
Standard Deviation 1830.8065
|
|
Absolute Change in Total Energy in PN From Baseline at Weeks 24 and 52
Week 52
|
-3510.111 kcal
Standard Deviation 1927.2066
|
SECONDARY outcome
Timeframe: Baseline and Week 48Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Relative Change in Absorption of Energy Over MB Periods From Baseline at Week 48
|
29.23 percentage change
Standard Deviation 36.898
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 48Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Fat - Week 4
|
247.281 kJ/day
Standard Deviation 661.1866
|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Fat - Week 48
|
406.741 kJ/day
Standard Deviation 776.4802
|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Carbohydrate - Week 4
|
272.881 kJ/day
Standard Deviation 675.1125
|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Carbohydrate - Week 48
|
877.011 kJ/day
Standard Deviation 945.2279
|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Protein - Week 4
|
176.178 kJ/day
Standard Deviation 280.0788
|
|
Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
Protein - Week 48
|
194.400 kJ/day
Standard Deviation 285.2597
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 48Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Based on average daily urine volume data derived as per balance period calculations.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Urine Volume Over MB Periods From Baseline at Week 4 and Week 48
Week 48
|
-178.556 mL/day
Standard Deviation 299.5613
|
|
Absolute Change in Urine Volume Over MB Periods From Baseline at Week 4 and Week 48
Week 4
|
246.889 mL/day
Standard Deviation 343.3609
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Absorption of Energy Over MB Periods From Baseline at Week 4
|
494.259 kJ/day
Standard Deviation 1355.5857
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 48Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
Since urinary electrolytes data were measured over the 72-hour MB period, the average of all available results were used for analyses for each MB parameter at a given analysis time point.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Calcium - Week 4
|
0.1444 cmol/L
Standard Deviation 1.09818
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Potassium - Week 48
|
-8.012 cmol/L
Standard Deviation 13.5798
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Calcium - Week 48
|
-0.5532 cmol/L
Standard Deviation 2.28264
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Magnesium - Week 4
|
-0.258 cmol/L
Standard Deviation 0.8370
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Magnesium - Week 48
|
-2.306 cmol/L
Standard Deviation 3.1400
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Sodium - Week 4
|
31.668 cmol/L
Standard Deviation 24.1946
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Sodium - Week 48
|
17.097 cmol/L
Standard Deviation 50.1676
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Potassium - Week 4
|
2.047 cmol/L
Standard Deviation 16.8154
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Urea - Week 4
|
7.693 cmol/L
Standard Deviation 59.2590
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Urea - Week 48
|
-89.649 cmol/L
Standard Deviation 77.9786
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Creatinine - Week 4
|
0.198 cmol/L
Standard Deviation 1.0071
|
|
Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48
Creatinine - Week 48
|
-0.011 cmol/L
Standard Deviation 1.3283
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
The PSQI is a patient-reported questionnaire used to measure the quality and patterns of sleep, over the past month. The PSQI has seven components: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleep medication, and daytime dysfunction over the last month. Minimum Score = 0 (better); Maximum Score = 21 (worse). A negative change from baseline represents an reduction in symptoms.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Change From Baseline in Pittsburgh Sleep Quality Inventory (PSQI) Total Score at Week 24 and Week 52
Week 24
|
-1.89 score on a scale
Standard Deviation 2.667
|
|
Change From Baseline in Pittsburgh Sleep Quality Inventory (PSQI) Total Score at Week 24 and Week 52
Week 52
|
-1.56 score on a scale
Standard Deviation 1.509
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. Participants with an assessment at given time point.
PGIC v2.0 is a single-item questionnaire using a 5-point verbal rating scale, to assess overall change in the participants status after taking the study drug. Response options range from 2= very much better to -2= very much worse.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Change From Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52
Week 24 · 2 (Much Better)
|
6 Participants
|
|
Change From Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52
Week 24 · 1 (A Little Better)
|
2 Participants
|
|
Change From Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52
Week 52 · 2 (Much Better)
|
8 Participants
|
|
Change From Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52
Week 52 · 1 (A Little Better)
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. Participants with an assessment at given time point.
This form is a single-item questionnaire assessing the participant's satisfaction with the trial medication over the preceding 7 days. Response options range from -2 to 2, very dissatisfied to very satisfied.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52
Week 24 · 2 (Very Satisfied)
|
5 Participants
|
|
Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52
Week 24 · 1 (Satisfied)
|
3 Participants
|
|
Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52
Week 52 · 2 (Very Satisfied)
|
3 Participants
|
|
Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52
Week 52 · 1 (Satisfied)
|
6 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. Partcipants with an assessment at given time point.
This is a single-item questionnaire assessing the participant's satisfaction with PS over the preceding 7 days. Response options range from -2 to 2, very dissatisfied to very satisfied. A reduction from baseline represents a decrease in satisfaction.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=5 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Change From Baseline in Patient Global Impression of Satisfaction With Parenteral Support (PGI-SPS) at Week 24 and Week 52
Week 24
|
-0.20 score on a scale
Standard Deviation 0.447
|
|
Change From Baseline in Patient Global Impression of Satisfaction With Parenteral Support (PGI-SPS) at Week 24 and Week 52
Week 52
|
0.00 score on a scale
Standard Deviation 0.000
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 52Population: The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. Participants with an assessment at given time point.
This is a three-item questionnaire assessing the impact of PS on the participant's sleep, daily activities, and quality of life (QoL) over the past 7 days. All questions have response options ranging from 0 to 4, not at all to extremely. A reduction from baseline represents a decrease in symptoms.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=5 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
Sleep Impact: Week 24
|
-0.40 score on a scale
Standard Deviation 1.517
|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
Sleep Impact: Week 52
|
-0.50 score on a scale
Standard Deviation 1.291
|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
Daily Activities Impact: Week 24
|
-0.80 score on a scale
Standard Deviation 1.643
|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
Daily Activities Impact: Week 52
|
-1.00 score on a scale
Standard Deviation 1.414
|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
QoL Impact: Week 24
|
-1.40 score on a scale
Standard Deviation 1.140
|
|
Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
QoL Impact: Week 52
|
-1.25 score on a scale
Standard Deviation 0.957
|
SECONDARY outcome
Timeframe: Pre-dose on Weeks 2, 4, 12, 24, 32, 40, 48, and 52Population: The safety analysis set included all participants exposed to trial medication. Participants with an assessment at given time point.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 2
|
1.928 ng/mL
Standard Deviation 0.7058
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 4
|
1.217 ng/mL
Standard Deviation 0.2902
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 12
|
1.934 ng/mL
Standard Deviation 0.6665
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 24
|
2.863 ng/mL
Standard Deviation 1.0668
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 32
|
1.739 ng/mL
Standard Deviation 0.6805
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 40
|
2.386 ng/mL
Standard Deviation 0.9587
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 48
|
3.810 ng/mL
Standard Deviation 1.1031
|
|
Trough Apraglutide Plasma Concentration (Ctrough)
Week 52
|
2.678 ng/mL
Standard Deviation 1.4039
|
SECONDARY outcome
Timeframe: Baseline and Weeks 2, 4, 12, 24, 32, 40, 48, and 52Population: The safety analysis set included all participants exposed to trial medication. Participants with an assessment at given time point.
Outcome measures
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 Participants
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Mean Plasma Citrulline Level
Week 32
|
14.994 μmol/L
Standard Deviation 12.6880
|
|
Mean Plasma Citrulline Level
Baseline
|
11.571 μmol/L
Standard Deviation 9.7091
|
|
Mean Plasma Citrulline Level
Week 2
|
13.516 μmol/L
Standard Deviation 9.8136
|
|
Mean Plasma Citrulline Level
Week 4
|
15.531 μmol/L
Standard Deviation 12.4055
|
|
Mean Plasma Citrulline Level
Week 12
|
14.860 μmol/L
Standard Deviation 12.2165
|
|
Mean Plasma Citrulline Level
Week 24
|
14.810 μmol/L
Standard Deviation 11.8877
|
|
Mean Plasma Citrulline Level
Week 40
|
17.860 μmol/L
Standard Deviation 15.8020
|
|
Mean Plasma Citrulline Level
Week 48
|
16.638 μmol/L
Standard Deviation 14.9346
|
|
Mean Plasma Citrulline Level
Week 52
|
14.714 μmol/L
Standard Deviation 11.7896
|
Adverse Events
Apraglutide SC Injections, Once Weekly
Serious adverse events
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 participants at risk
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Hepatobiliary disorders
Cholangitis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Campylobacter gastroenteritis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Septic shock
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Product Issues
Device dislocation
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
Other adverse events
| Measure |
Apraglutide SC Injections, Once Weekly
n=9 participants at risk
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
|
|---|---|
|
Injury, poisoning and procedural complications
Vascular access site pain
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Vascular disorders
Haematoma
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Asthenia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Complication associated with device
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Catheter site irritation
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Injection site erythema
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Injection site pruritus
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Fatigue
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Injection site haematoma
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
General disorders
Injection site pain
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Investigations
Weight decreased
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Procedural vomiting
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Bone contusion
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Post procedural complication
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Post procedural haematoma
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Procedural nausea
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Injury, poisoning and procedural complications
Radius fracture
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Nervous system disorders
Dizziness
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Nervous system disorders
Metabolic encephalopathy
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Blood and lymphatic system disorders
Anaemia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Stomatitis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Vomiting
|
55.6%
5/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
3/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Abdominal pain
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Anal fissure
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Anorectal discomfort
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Regurgitation
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Hepatobiliary disorders
Portal vein thrombosis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Renal and urinary disorders
Dysuria
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Renal and urinary disorders
Nephrolithiasis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Musculoskeletal and connective tissue disorders
Tenosynovitis stenosans
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Influenza
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Oral herpes
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Sinusitis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
COVID-19
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Vascular device infection
|
22.2%
2/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Nasopharyngitis
|
55.6%
5/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Asymptomatic COVID-19
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Metabolism and nutrition disorders
Magnesium deficiency
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Hepatobiliary disorders
Cholangitis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
|
Infections and infestations
Campylobacter gastroenteritis
|
11.1%
1/9 • Day 1 up to approximately 55 weeks
The safety analysis set included all participants exposed to trial medication.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place