Trial Outcomes & Findings for A Study of CNSA-001 in Women With Diabetic Gastroparesis (NCT NCT03712124)
NCT ID: NCT03712124
Last Updated: 2022-01-05
Results Overview
Participants consumed 150 milliliters (mL) of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0 = no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.
COMPLETED
PHASE2
21 participants
Baseline (Day 1), Day 14
2022-01-05
Participant Flow
Participant milestones
| Measure |
CNSA-001
Participants received CNSA-001 (sepiapterin) 20 milligrams (mg)/kilogram (kg)/day (10 mg/kg twice daily \[BID\]) as an oral suspension for 14 days.
|
Placebo
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
11
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
10
|
11
|
|
Overall Study
COMPLETED
|
9
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
CNSA-001
Participants received CNSA-001 (sepiapterin) 20 milligrams (mg)/kilogram (kg)/day (10 mg/kg twice daily \[BID\]) as an oral suspension for 14 days.
|
Placebo
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Overall Study
Protocol Violation
|
1
|
0
|
Baseline Characteristics
A Study of CNSA-001 in Women With Diabetic Gastroparesis
Baseline characteristics by cohort
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.7 years
STANDARD_DEVIATION 14.29 • n=5 Participants
|
60.5 years
STANDARD_DEVIATION 15.53 • n=7 Participants
|
54.4 years
STANDARD_DEVIATION 15.97 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 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
|
5 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 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
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 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: Baseline (Day 1), Day 14Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo). Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.
Participants consumed 150 milliliters (mL) of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0 = no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 14
Baseline
|
293.5 mL
Standard Deviation 124.52
|
345.6 mL
Standard Deviation 99.06
|
|
Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 14
Change at Day 14
|
1.3 mL
Standard Deviation 145.51
|
2.0 mL
Standard Deviation 72.61
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Day 28Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo). Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.
Participants consumed 150 mL of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0: no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 28
Baseline
|
293.5 mL
Standard Deviation 124.52
|
345.6 mL
Standard Deviation 99.06
|
|
Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 28
Change at Day 28
|
75.3 mL
Standard Deviation 61.40
|
-30.9 mL
Standard Deviation 67.00
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Days 14/15 and 28Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo). Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified category.
The GCSI consists of 9 symptom severity items covering the following domains (subscales): nausea/vomiting (Questions 1 to 3); post-prandial fullness/early satiety (Questions 4 to 7), and bloating (Questions 8 and 9). Each question was rated on a 6-point Likert scale (0 = None, 1 = Very Mild, 2 = Mild, 3 = Moderate, 4 = Severe and 5 = Very Severe), with lower scores representing lesser symptom severity. To obtain a participant-specific score on each subscale, the scores on each question within the subscale were summed for each participant and divided by the number of questions in the subscale. Total GCSI score was mean of 3 subscales. Total aggregate GCSI score ranges from 0 to 45, with lower scores indicating lesser symptom severity.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Change From Baseline (Day 1) in the Gastroparesis Cardinal Symptom Index (GCSI) Total Score at Day 14/15 and Day 28
Baseline
|
3.1778 units on a scale
Standard Deviation 0.78951
|
3.3030 units on a scale
Standard Deviation 0.68329
|
|
Change From Baseline (Day 1) in the Gastroparesis Cardinal Symptom Index (GCSI) Total Score at Day 14/15 and Day 28
Change at Day 14/15
|
-1.0864 units on a scale
Standard Deviation 0.82921
|
-1.0101 units on a scale
Standard Deviation 0.94602
|
|
Change From Baseline (Day 1) in the Gastroparesis Cardinal Symptom Index (GCSI) Total Score at Day 14/15 and Day 28
Change at Day 28
|
-0.9583 units on a scale
Standard Deviation 1.12677
|
-0.8081 units on a scale
Standard Deviation 0.98519
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Days 14 and 28Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo). Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified category.
The PAGI-SYM is a 20-item upper gastrointestinal (GI) symptom severity instrument with 6 subscales: nausea/vomiting (Questions 1 to 3), post-prandial fullness/early satiety (Questions 4 to 7), bloating (Questions 8 and 9), upper abdominal pain (Questions 10 and 11), lower abdominal pain (Questions 12 and 13), and heartburn/regurgitation (Questions 14 to 20). Each question was rated on a 6-point Likert scale (0 = None, 1 = Very Mild, 2 = Mild, 3 = Moderate, 4 = Severe and 5 = Very Severe), with lower scores representing lesser symptom severity. To obtain a participant-specific score on each subscale and the total PAGI-SYM, the scores on each question within the subscale were summed for each participant and divided by the number of questions in the subscale.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Upper Abdominal Pain Subscale Score: Baseline
|
2.55 units on a scale
Standard Deviation 1.343
|
3.00 units on a scale
Standard Deviation 0.975
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Upper Abdominal Pain Score: Change at Day 14
|
-0.94 units on a scale
Standard Deviation 0.917
|
-1.00 units on a scale
Standard Deviation 1.360
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Heartburn Subscale Score: Change at Day 28
|
-0.7143 units on a scale
Standard Deviation 1.26629
|
-0.7922 units on a scale
Standard Deviation 0.55462
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Nausea/Vomiting Subscale Score: Baseline
|
1.7667 units on a scale
Standard Deviation 1.25757
|
1.7879 units on a scale
Standard Deviation 1.19511
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Nausea/Vomiting Subscale Score: Change at Day 14
|
-0.4074 units on a scale
Standard Deviation 0.64070
|
-0.6970 units on a scale
Standard Deviation 0.88763
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Nausea/Vomiting Subscale Score: Change at Day 28
|
-0.2500 units on a scale
Standard Deviation 0.72921
|
-0.7273 units on a scale
Standard Deviation 1.31503
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Fullness Subscale Score: Baseline
|
4.025 units on a scale
Standard Deviation 0.9461
|
4.068 units on a scale
Standard Deviation 0.7753
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Fullness Subscale Score: Change at Day 14
|
-1.278 units on a scale
Standard Deviation 1.1622
|
-1.023 units on a scale
Standard Deviation 1.1750
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Fullness Subscale Score: Change at Day 28
|
-1.344 units on a scale
Standard Deviation 1.6687
|
-0.818 units on a scale
Standard Deviation 1.2554
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Bloating Subscale Score: Baseline
|
4.25 units on a scale
Standard Deviation 0.979
|
4.09 units on a scale
Standard Deviation 1.020
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Bloating Subscale Score: Change at Day 14
|
-1.67 units on a scale
Standard Deviation 0.612
|
-1.23 units on a scale
Standard Deviation 1.348
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Bloating Subscale Score: Change at Day 28
|
-1.69 units on a scale
Standard Deviation 1.361
|
-1.05 units on a scale
Standard Deviation 1.036
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Upper Abdominal Pain Score: Change at Day 28
|
-0.69 units on a scale
Standard Deviation 1.413
|
-1.00 units on a scale
Standard Deviation 1.323
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Lower Abdominal Pain Subscale Score: Baseline
|
2.20 units on a scale
Standard Deviation 1.337
|
2.41 units on a scale
Standard Deviation 1.429
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Lower Abdominal Pain Score: Change at Day 14
|
-0.39 units on a scale
Standard Deviation 0.821
|
-0.68 units on a scale
Standard Deviation 1.383
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Lower Abdominal Pain Score: Change at Day 28
|
-0.19 units on a scale
Standard Deviation 1.668
|
-0.23 units on a scale
Standard Deviation 1.126
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Heartburn Subscale Score: Baseline
|
2.3571 units on a scale
Standard Deviation 0.84448
|
2.7013 units on a scale
Standard Deviation 1.39453
|
|
Change From Baseline (Day 1) in the Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity (PAGI-SYM) Subscale Scores at Day 14 and Day 28
Heartburn Subscale Score: Change at Day 14
|
-0.9048 units on a scale
Standard Deviation 1.08327
|
-1.0260 units on a scale
Standard Deviation 1.23357
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Days 14 and 28Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo). Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.
The GEBT is a nonradioactive non-invasive test, conducted over a 4-hour evaluation period and is designed to show how rapidly the stomach empties solids by measuring carbon dioxide (CO2) in a participant's breath. The GEBT measures the rate of CO2 excretion after consumption of a C-enriched test meal. The participant's CO2 excretion rate at any measurement time "t" was calculated and reported using the GEBT metric "kPCD". kPCD stands for "1000 \* Percent Carbon-13 Dose (PCD) excreted per minute." Participants provided baseline (premeal) breath samples and then consumed a standardized 230 kilocalorie (kCal) meal. Single post-meal breath samples were collected in capped glass tubes at 45, 90, 120, 150, 180, and 240 minutes after the meal was consumed. The GEBT kPCD value (13CO2 excretion rate) was proportional to the rate of gastric emptying. Increasing GEBT values represent increasing rates of gastric emptying.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 150 minutes post-meal
|
28.13 kPCD
Standard Deviation 15.552
|
28.75 kPCD
Standard Deviation 8.257
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 150 minutes post-meal
|
5.60 kPCD
Standard Deviation 16.822
|
2.02 kPCD
Standard Deviation 8.163
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 90 minutes post-meal
|
0.76 kPCD
Standard Deviation 11.739
|
4.42 kPCD
Standard Deviation 8.625
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 150 minutes post-meal
|
-0.34 kPCD
Standard Deviation 14.750
|
2.39 kPCD
Standard Deviation 9.609
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 90 minutes post-meal
|
14.62 kPCD
Standard Deviation 8.697
|
16.10 kPCD
Standard Deviation 5.414
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 120 minutes post-meal
|
22.59 kPCD
Standard Deviation 12.859
|
22.96 kPCD
Standard Deviation 7.139
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 180 minutes post-meal
|
31.87 kPCD
Standard Deviation 17.553
|
32.80 kPCD
Standard Deviation 9.733
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 240 minutes post-meal
|
34.50 kPCD
Standard Deviation 17.129
|
37.32 kPCD
Standard Deviation 10.854
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 45 minutes post-meal
|
5.68 kPCD
Standard Deviation 7.984
|
1.55 kPCD
Standard Deviation 4.954
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 90 minutes post-meal
|
7.33 kPCD
Standard Deviation 12.049
|
1.67 kPCD
Standard Deviation 3.914
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 120 minutes post-meal
|
7.03 kPCD
Standard Deviation 15.646
|
2.14 kPCD
Standard Deviation 6.549
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Baseline: 45 minutes post-meal
|
5.85 kPCD
Standard Deviation 3.727
|
9.45 kPCD
Standard Deviation 3.125
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 180 minutes post-meal
|
4.71 kPCD
Standard Deviation 19.282
|
3.34 kPCD
Standard Deviation 7.294
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 14: 240 minutes post-meal
|
0.67 kPCD
Standard Deviation 19.535
|
1.18 kPCD
Standard Deviation 10.426
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 45 minutes post-meal
|
2.28 kPCD
Standard Deviation 5.109
|
2.00 kPCD
Standard Deviation 3.326
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 120 minutes post-meal
|
0.26 kPCD
Standard Deviation 13.656
|
3.59 kPCD
Standard Deviation 11.081
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 180 minutes post-meal
|
0.44 kPCD
Standard Deviation 11.714
|
1.43 kPCD
Standard Deviation 9.221
|
|
Change Form Baseline (Day 1) in the Gastric Emptying Breath Test (GEBT) Excretion Rate at Day 14 and Day 28
Change at Day 28: 240 minutes post-meal
|
0.43 kPCD
Standard Deviation 11.656
|
-1.43 kPCD
Standard Deviation 9.286
|
SECONDARY outcome
Timeframe: Baseline up to Day 44Population: Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Outcome measures
| Measure |
CNSA-001
n=10 Participants
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 Participants
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs)
|
4 Participants
|
2 Participants
|
Adverse Events
CNSA-001
Placebo
Serious adverse events
| Measure |
CNSA-001
n=10 participants at risk
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 participants at risk
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Gastrointestinal disorders
Impaired gastric emptying
|
20.0%
2/10 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
0.00%
0/11 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
Other adverse events
| Measure |
CNSA-001
n=10 participants at risk
Participants received CNSA-001 20 mg/kg/day (10 mg/kg BID) as an oral suspension for 14 days.
|
Placebo
n=11 participants at risk
Participants received placebo matched to CNSA-001 BID for 14 days.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain lower
|
10.0%
1/10 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
0.00%
0/11 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
|
Nervous system disorders
Cervical radiculopathy
|
10.0%
1/10 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
0.00%
0/11 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
|
Nervous system disorders
Headache
|
0.00%
0/10 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
9.1%
1/11 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/10 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
9.1%
1/11 • Baseline up to Day 44
Safety population included all participants who received any amount of study drug (CNSA-001 or placebo).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER