Trial Outcomes & Findings for A Study of Single Ascending Doses of IW-3300 in Healthy Volunteers (NCT NCT05188261)

Last Updated: 2023-12-08

Results Overview

An adverse event (AE) is any untoward medical occurrence in a participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE was considered a treatment-emergent AE (TEAE) if the AE started after initial study drug administration and within 1 day of the last dose of study drug.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

32 participants

Primary outcome timeframe

From first dose of study drug through 24 hours post-Day 1 dose

Results posted on

2023-12-08

Participant Flow

The study was designed to include up to 5 cohorts with 8 participants per cohort. Within each cohort, participants were randomized to receive a single dose of IW-3300 (6 participants) or placebo (2 participants). Doses to be evaluated were 100, 300, 900, and 2500 μg. An optional 5th cohort was planned to test an IW-3300 dose of ≤ 2500 μg. Based on a blinded review of safety and tolerability data from Cohort 1 through 4, the Dose Escalation Committee decided not to enroll the optional 5th Cohort.

As pre-specified by the statistical analysis plan, for the analyses reported herein, data from the 2 participants dosed with placebo in each of the 4 cohorts were pooled into a single placebo group (N=8).

Participant milestones

Participant milestones
Measure	Placebo Participants received a single dose of placebo administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	100 μg IW-3300 Participants received a single dose of 100 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	300 μg IW-3300 Participants received a single dose of 300 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	900 μg IW-3300 Participants received a single dose of 900 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	2500 μg IW-3300 Participants received a single dose of 2500 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.
Overall Study STARTED	8	6	6	6	6
Overall Study COMPLETED	8	6	6	6	6
Overall Study NOT COMPLETED	0	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Single Ascending Doses of IW-3300 in Healthy Volunteers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=8 Participants Participants received a single dose of placebo administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	100 μg IW-3300 n=6 Participants Participants received a single dose of 100 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	300 μg IW-3300 n=6 Participants Participants received a single dose of 300 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	900 μg IW-3300 n=6 Participants Participants received a single dose of 900 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	2500 μg IW-3300 n=6 Participants Participants received a single dose of 2500 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	Total n=32 Participants Total of all reporting groups
Age, Continuous	38.1 years STANDARD_DEVIATION 12.64 • n=5 Participants	29.8 years STANDARD_DEVIATION 13.04 • n=7 Participants	48.2 years STANDARD_DEVIATION 11.30 • n=5 Participants	39.7 years STANDARD_DEVIATION 13.62 • n=4 Participants	43.3 years STANDARD_DEVIATION 13.76 • n=21 Participants	39.7 years STANDARD_DEVIATION 13.44 • n=10 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	1 Participants n=4 Participants	2 Participants n=21 Participants	7 Participants n=10 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	5 Participants n=7 Participants	4 Participants n=5 Participants	5 Participants n=4 Participants	4 Participants n=21 Participants	25 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=21 Participants	13 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=21 Participants	19 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race/Ethnicity, Customized White	5 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants	4 Participants n=4 Participants	4 Participants n=21 Participants	23 Participants n=10 Participants
Race/Ethnicity, Customized Black or African American	3 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	1 Participants n=21 Participants	7 Participants n=10 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=10 Participants
Race/Ethnicity, Customized Other, Not Specified	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	1 Participants n=10 Participants

PRIMARY outcome

Timeframe: From first dose of study drug through 24 hours post-Day 1 dose

Outcome measures

Outcome measures
Measure	Placebo n=8 Participants Participants received a single dose of placebo administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	100 μg IW-3300 n=6 Participants Participants received a single dose of 100 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	300 μg IW-3300 n=6 Participants Participants received a single dose of 300 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	900 μg IW-3300 n=6 Participants Participants received a single dose of 900 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.	2500 μg IW-3300 n=6 Participants Participants received a single dose of 2500 μg IW-3300 administered rectally (as a low-volume \[20 mL\] enema) following a fast of at least 6 hours.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	2 Participants	1 Participants	0 Participants	1 Participants	2 Participants

PRIMARY outcome

Timeframe: From first dose of study drug through 24 hours post-Day 1 dose

A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability/incapacity; is a congenital anomaly/birth defect; or other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the subject or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition. An SAE was considered a treatment-emergent SAE (serious TEAE) if the SAE started after initial study drug administration and within 1 day of the last dose of study drug.