Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (NCT NCT04837040)

NCT ID: NCT04837040

Last Updated: 2026-01-13

Results Overview

A value \>1.0 indicates IGF-1 levels above the age- and sex-adjusted ULN. Response is defined as an IGF-1 level ≤1.0×ULN based on the average of last 2 measurements (weeks 34 and 36).

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE3
• Target enrollment: 58 participants
• Primary outcome timeframe: 36 Weeks
• Results posted on: 2026-01-13

Participant Flow

This is a Phase 3, multicenter, randomized, placebo-controlled study where a total of 115 participants were screened, of which 58 participants were randomized (30 participants in the total paltusotine group and 28 participants in the placebo group) to the randomized control (RC) Phase.

Participants were randomized to treatment or placebo in a 1:1 ratio. Participants who completed the RC phase or who met rescue criteria could enter the open-label extension (OLE) phase. The RC Phase is completed. The OLE Phase is ongoing.

Participant milestones

Measure	Placebo Participants were randomized to receive matching placebo tablets in a 1:1 ratio.	Paltusotine Participants were randomized in a 1:1 ratio and received a daily dose paltusotine orally.
RC Phase (Up to Week 36) STARTED	28	30
RC Phase (Up to Week 36) COMPLETED	27	30
RC Phase (Up to Week 36) NOT COMPLETED	1	0
OLE Phase (Up to Week 240) STARTED	26	27
OLE Phase (Up to Week 240) Ongoing	26	27
OLE Phase (Up to Week 240) COMPLETED	0	0
OLE Phase (Up to Week 240) NOT COMPLETED	26	27

Reasons for withdrawal

Measure	Placebo Participants were randomized to receive matching placebo tablets in a 1:1 ratio.	Paltusotine Participants were randomized in a 1:1 ratio and received a daily dose paltusotine orally.
RC Phase (Up to Week 36) Withdrawal by Subject	1	0

Baseline Characteristics

A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly

Baseline characteristics by cohort

Measure	Paltusotine n=30 Participants Participants were randomized in a 1:1 ratio and received a daily dose paltusotine orally.	Placebo n=28 Participants Participants were randomized to receive matching placebo tablets in a 1:1 ratio.	Total n=58 Participants Total of all reporting groups
Age, Continuous	55.9 years STANDARD_DEVIATION 14.57 • n=210 Participants	53.9 years STANDARD_DEVIATION 12.89 • n=19 Participants	54.9 years STANDARD_DEVIATION 13.70 • n=123 Participants
Sex: Female, Male Female	15 Participants n=210 Participants	17 Participants n=19 Participants	32 Participants n=123 Participants
Sex: Female, Male Male	15 Participants n=210 Participants	11 Participants n=19 Participants	26 Participants n=123 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	11 Participants n=210 Participants	8 Participants n=19 Participants	19 Participants n=123 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	17 Participants n=210 Participants	18 Participants n=19 Participants	35 Participants n=123 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=210 Participants	2 Participants n=19 Participants	4 Participants n=123 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants
Race (NIH/OMB) Asian	0 Participants n=210 Participants	2 Participants n=19 Participants	2 Participants n=123 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants
Race (NIH/OMB) Black or African American	2 Participants n=210 Participants	1 Participants n=19 Participants	3 Participants n=123 Participants
Race (NIH/OMB) White	23 Participants n=210 Participants	19 Participants n=19 Participants	42 Participants n=123 Participants
Race (NIH/OMB) More than one race	3 Participants n=210 Participants	4 Participants n=19 Participants	7 Participants n=123 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=210 Participants	2 Participants n=19 Participants	4 Participants n=123 Participants

PRIMARY outcome

Timeframe: 36 Weeks

Population: Full Analysis Set

A value >1.0 indicates IGF-1 levels above the age- and sex-adjusted ULN. Response is defined as an IGF-1 level ≤1.0×ULN based on the average of last 2 measurements (weeks 34 and 36).

Outcome measures

Measure	Paltusotine n=30 Participants Participants were randomized in a 1:1 ratio (paltusotine vs placebo) and received a daily dose paltusotine orally.	Placebo n=28 Participants Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Percentage of Participants Who Maintain Biochemical Response in IGF-1 (≤1.0× the Upper Limit of Normal [ULN]) at the End of the Randomized Control Phase (EOR)	83.3 percentage of participants	3.6 percentage of participants

SECONDARY outcome

Timeframe: Baseline to 36 Weeks

Population: Full Analysis Set

A value >1.0 indicates IGF-1 levels above the age- and sex-adjusted ULN. Baseline was defined as the last non-missing assessment prior to first dose of study drug for all assessments except IGF-1, growth hormone (GH), and acromegaly symptoms diary (ASD). Change from Baseline was determined by calculating (post-Baseline value - Baseline value).

Outcome measures

Measure	Paltusotine n=30 Participants Participants were randomized in a 1:1 ratio (paltusotine vs placebo) and received a daily dose paltusotine orally.	Placebo n=28 Participants Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Change From Baseline in IGF-1 to EOR	0.042 nanograms per milliliter (ng/ml) Standard Error 0.0932	0.833 nanograms per milliliter (ng/ml) Standard Error 0.0962

SECONDARY outcome

Timeframe: Week 34

Population: Full Analysis Set. Only those participants with data available at specified timepoints have been presented.

GH maintenance of response was analyzed in participants with GH<1.0 ng/mL at week 34, out of those who had GH<1.0 ng/mL at Baseline, using the same methodology as the primary endpoint.

Outcome measures

Measure	Paltusotine n=23 Participants Participants were randomized in a 1:1 ratio (paltusotine vs placebo) and received a daily dose paltusotine orally.	Placebo n=18 Participants Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Percentage of Participants With GH <1.0 ng/mL at Week 34	87.0 percentage of participants	27.8 percentage of participants

SECONDARY outcome

Timeframe: Baseline to 36 Weeks

Population: Full Analysis Set

The ASD is a sponsor-developed daily diary to assess important acromegaly symptoms from the patient perspective. The weekly average ASD total score is calculated from 7 items associated with acromegaly (headache pain, joint pain, sweating, fatigue, weakness in legs, swelling, and numbness or tingling). The ASD total score ranges from 0 to 70 with each symptom contributing up to 10 points. A higher score = higher symptom severity. Change from baseline in total ASD was defined as the postbaseline total ASD score (the average of the available scores seven days on or prior to the scheduled visit date) minus the baseline total ASD score.

Outcome measures

Measure	Paltusotine n=30 Participants Participants were randomized in a 1:1 ratio (paltusotine vs placebo) and received a daily dose paltusotine orally.	Placebo n=28 Participants Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Change From Baseline in Total Acromegaly Symptoms Diary (ASD) Score to EOR	-0.606 units on a scale Standard Error 1.5044	4.558 units on a scale Standard Error 1.5926

Adverse Events

Paltusotine

Serious events: 0 serious events

Other events: 24 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Measure	Paltusotine n=30 participants at risk Participants were randomized in a 1:1 ratio and received a daily dose of paltusotine orally.	Placebo n=28 participants at risk Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Hepatobiliary disorders cholecystitis acute	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	3.6% 1/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.

Other adverse events

Measure	Paltusotine n=30 participants at risk Participants were randomized in a 1:1 ratio and received a daily dose of paltusotine orally.	Placebo n=28 participants at risk Participants were randomized to receive matching placebo tablets in a 1:1 ratio.
Musculoskeletal and connective tissue disorders Arthralgia	30.0% 9/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	57.1% 16/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Musculoskeletal and connective tissue disorders Muscular weakness	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	10.7% 3/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Musculoskeletal and connective tissue disorders Back pain	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	10.7% 3/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Diarrhoea	23.3% 7/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	14.3% 4/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Abdominal pain	16.7% 5/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	10.7% 3/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Nausea	13.3% 4/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Constipation	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	14.3% 4/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Abdominal distension	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	3.6% 1/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Flatulence	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	3.6% 1/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
General disorders Peripheral swelling	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	35.7% 10/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
General disorders Fatigue	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	17.9% 5/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
General disorders Asthenia	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	14.3% 4/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
General disorders Hyperhidrosis	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	14.3% 4/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Nervous system disorders Headache	20.0% 6/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	35.7% 10/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Nervous system disorders Paraesthesia	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	25.0% 7/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Nervous system disorders Dizziness	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Infections and infestations COVID-19	0.00% 0/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	21.4% 6/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Infections and infestations Urinary tract infection	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Infections and infestations Nasopharyngitis	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	3.6% 1/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Infections and infestations Gastroenteritis	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	0.00% 0/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Metabolism and nutrition disorders Hyperglycaemia	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Metabolism and nutrition disorders Decreased appetite	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	0.00% 0/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Respiratory, thoracic and mediastinal disorders Dyspnoea	3.3% 1/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	7.1% 2/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Blood and lymphatic system disorders Iron deficiency anaemia	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	0.00% 0/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.
Cardiac disorders Palpitations	6.7% 2/30 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.	0.00% 0/28 • Up to 36 Weeks Serious TEAEs and TEAEs were collected in a safety analysis set which was comprised of all participants who received study drug with treatment assignment based on the treatment received. The adverse events are presented as of last participant visit in the RC phase only. Final adverse events from the ongoing OLE will be reported after study completion. All participants, irrespective of their dose level in the paltusotine or placebo arms, have been presented.

Additional Information

Clinical Research Director

Crinetics Pharmaceuticals, Inc.

Phone: (833) 276-4636

Email: medinfo@crinetics.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60