Trial Outcomes & Findings for An Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (ACROBAT Edge) (NCT NCT03789656)

NCT ID: NCT03789656

Last Updated: 2025-02-17

Results Overview

Change from baseline in IGF-1 level at Week 13/End of Treatment (W13/EoT) in Group 1 subjects Efficacy Analysis Set (EAS).

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 47 participants
• Primary outcome timeframe: 13 Weeks
• Results posted on: 2025-02-17

Participant Flow

A total of 45 subjects were planned to be enrolled in the study. 47 subjects participated in the study.

A total of 45 subjects were planned to be enrolled in the study. 47 subjects participated in the study.

Participant milestones

Measure	Group 1 Partial responders on a stable treatment of octreotide long-acting release (LAR) or lanreotide depot (at least 1 Screening IGF-1 value was \> upper limit of normal (ULN), and the V2 value was ≤2.5× ULN)	Group 2 Partial responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (at least 1 Screening IGF-1 value was \>ULN, and the V2 value was ≤2.5 × ULN).	Group 3 Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN).	Group 4 Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN).	Group 5 Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and pegvisomant (mean of Screening IGF-1 values were ≤ULN).
Screening Period: up to 6 Weeks STARTED	25	10	5	4	3
Screening Period: up to 6 Weeks COMPLETED	25	10	5	4	3
Screening Period: up to 6 Weeks NOT COMPLETED	0	0	0	0	0
Treatment Period: up to 13 Weeks STARTED	25	10	5	4	3
Treatment Period: up to 13 Weeks COMPLETED	23	10	4	3	3
Treatment Period: up to 13 Weeks NOT COMPLETED	2	0	1	1	0
Follow-up Period: up to 4 Weeks STARTED	23	10	4	3	3
Follow-up Period: up to 4 Weeks COMPLETED	22	10	4	3	3
Follow-up Period: up to 4 Weeks NOT COMPLETED	1	0	0	0	0

Reasons for withdrawal

Measure	Group 1 Partial responders on a stable treatment of octreotide long-acting release (LAR) or lanreotide depot (at least 1 Screening IGF-1 value was \> upper limit of normal (ULN), and the V2 value was ≤2.5× ULN)	Group 2 Partial responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (at least 1 Screening IGF-1 value was \>ULN, and the V2 value was ≤2.5 × ULN).	Group 3 Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN).	Group 4 Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN).	Group 5 Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and pegvisomant (mean of Screening IGF-1 values were ≤ULN).
Treatment Period: up to 13 Weeks Prohibited Medication	1	0	0	1	0
Treatment Period: up to 13 Weeks COVID-19 Lockdown	0	0	1	0	0
Treatment Period: up to 13 Weeks Unable to Travel	1	0	0	0	0
Follow-up Period: up to 4 Weeks Withdrawal by Subject	1	0	0	0	0

Baseline Characteristics

Source missing for one participant, data removed and noted as protocol deviation.

Baseline characteristics by cohort

Measure	Group 1 n=25 Participants Efficacy Analysis Set - Primary Analysis Population for all efficacy analyses. Only includes participants from Group 1. Eligibility to enter this group was specified as: Partial responders on a stable treatment of octreotide long-acting release (LAR) or lanreotide depot (at least 1 Screening IGF-1 value was greater than upper limit of normal (ULN), and the V2 value was ≤2.5× ULN)	Group 2 n=10 Participants Eligibility to enter this group was specified as: Partial responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (at least 1 Screening IGF-1 value was \>ULN, and the V2 value was ≤2.5 × ULN)	Group 3 n=5 Participants Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN)	Group 4 n=4 Participants Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)	Group 5 n=3 Participants Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and pegvisomant (mean of Screening IGF-1 values were ≤ULN).	Total n=47 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=25 Participants	0 Participants n=10 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	0 Participants n=47 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=25 Participants	8 Participants n=10 Participants	4 Participants n=5 Participants	3 Participants n=4 Participants	2 Participants n=3 Participants	39 Participants n=47 Participants
Age, Categorical >=65 years	3 Participants n=25 Participants	2 Participants n=10 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants	1 Participants n=3 Participants	8 Participants n=47 Participants
Age, Customized Age, y, median (range)	52.0 years n=25 Participants	52.5 years n=10 Participants	51.0 years n=5 Participants	56.0 years n=4 Participants	38.0 years n=3 Participants	51.0 years n=47 Participants
Sex: Female, Male Female	11 Participants n=25 Participants	7 Participants n=10 Participants	3 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=3 Participants	27 Participants n=47 Participants
Sex: Female, Male Male	14 Participants n=25 Participants	3 Participants n=10 Participants	2 Participants n=5 Participants	1 Participants n=4 Participants	0 Participants n=3 Participants	20 Participants n=47 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=25 Participants	6 Participants n=10 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=3 Participants	13 Participants n=47 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 Participants n=25 Participants	4 Participants n=10 Participants	3 Participants n=5 Participants	4 Participants n=4 Participants	2 Participants n=3 Participants	34 Participants n=47 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=25 Participants	0 Participants n=10 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	0 Participants n=47 Participants
Race/Ethnicity, Customized White	25 Participants n=25 Participants	5 Participants n=10 Participants	5 Participants n=5 Participants	4 Participants n=4 Participants	3 Participants n=3 Participants	42 Participants n=47 Participants
Race/Ethnicity, Customized Asian	0 Participants n=25 Participants	1 Participants n=10 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	1 Participants n=47 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=25 Participants	2 Participants n=10 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	2 Participants n=47 Participants
Race/Ethnicity, Customized Other	0 Participants n=25 Participants	2 Participants n=10 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	2 Participants n=47 Participants
Region of Enrollment United States	3 participants n=25 Participants	1 participants n=10 Participants	0 participants n=5 Participants	2 participants n=4 Participants	2 participants n=3 Participants	8 participants n=47 Participants
Region of Enrollment Germany	0 participants n=25 Participants	1 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	1 participants n=47 Participants
Region of Enrollment Greece	5 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	1 participants n=4 Participants	0 participants n=3 Participants	6 participants n=47 Participants
Region of Enrollment Hungary	5 participants n=25 Participants	1 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	6 participants n=47 Participants
Region of Enrollment Italy	0 participants n=25 Participants	0 participants n=10 Participants	1 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	1 participants n=47 Participants
Region of Enrollment Poland	2 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	2 participants n=47 Participants
Region of Enrollment Romania	0 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	1 participants n=4 Participants	1 participants n=3 Participants	2 participants n=47 Participants
Region of Enrollment Serbia	3 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	3 participants n=47 Participants
Region of Enrollment Slovakia	1 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	1 participants n=47 Participants
Region of Enrollment United Kingdom	2 participants n=25 Participants	0 participants n=10 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	2 participants n=47 Participants
Region of Enrollment Brazil	4 participants n=25 Participants	6 participants n=10 Participants	3 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	13 participants n=47 Participants
Region of Enrollment New Zealand	0 participants n=25 Participants	1 participants n=10 Participants	1 participants n=5 Participants	0 participants n=4 Participants	0 participants n=3 Participants	2 participants n=47 Participants
Height	173.95 centimeter n=24 Participants • Source missing for one participant, data removed and noted as protocol deviation.	167.75 centimeter n=10 Participants • Source missing for one participant, data removed and noted as protocol deviation.	173.00 centimeter n=5 Participants • Source missing for one participant, data removed and noted as protocol deviation.	169.45 centimeter n=4 Participants • Source missing for one participant, data removed and noted as protocol deviation.	157.50 centimeter n=3 Participants • Source missing for one participant, data removed and noted as protocol deviation.	170.25 centimeter n=46 Participants • Source missing for one participant, data removed and noted as protocol deviation.
Weight	90.95 kilogram(s) n=24 Participants • Source missing for one participant, data removed and noted as protocol deviation.	84.95 kilogram(s) n=10 Participants • Source missing for one participant, data removed and noted as protocol deviation.	87.30 kilogram(s) n=5 Participants • Source missing for one participant, data removed and noted as protocol deviation.	107 kilogram(s) n=4 Participants • Source missing for one participant, data removed and noted as protocol deviation.	77.50 kilogram(s) n=3 Participants • Source missing for one participant, data removed and noted as protocol deviation.	87.15 kilogram(s) n=46 Participants • Source missing for one participant, data removed and noted as protocol deviation.
BMI	29.25 kilogram(s)/square meter n=24 Participants • Source missing for one participant, data removed and noted as protocol deviation.	30.30 kilogram(s)/square meter n=10 Participants • Source missing for one participant, data removed and noted as protocol deviation.	28.50 kilogram(s)/square meter n=5 Participants • Source missing for one participant, data removed and noted as protocol deviation.	34.85 kilogram(s)/square meter n=4 Participants • Source missing for one participant, data removed and noted as protocol deviation.	31.20 kilogram(s)/square meter n=3 Participants • Source missing for one participant, data removed and noted as protocol deviation.	29.95 kilogram(s)/square meter n=46 Participants • Source missing for one participant, data removed and noted as protocol deviation.
Ring Size	12.5 Scores on a Scale n=24 Participants • Source missing for one participant, data removed and noted as protocol deviation.	12.0 Scores on a Scale n=10 Participants • Source missing for one participant, data removed and noted as protocol deviation.	11.0 Scores on a Scale n=5 Participants • Source missing for one participant, data removed and noted as protocol deviation.	12.5 Scores on a Scale n=4 Participants • Source missing for one participant, data removed and noted as protocol deviation.	8.0 Scores on a Scale n=3 Participants • Source missing for one participant, data removed and noted as protocol deviation.	12.0 Scores on a Scale n=46 Participants • Source missing for one participant, data removed and noted as protocol deviation.
IGF-1 (x ULN) at Baseline	1.335 ng/mL x ULN n=25 Participants	1.400 ng/mL x ULN n=10 Participants	0.922 ng/mL x ULN n=5 Participants	0.659 ng/mL x ULN n=4 Participants	0.909 ng/mL x ULN n=3 Participants	1.214 ng/mL x ULN n=47 Participants
Serum Growth Hormone Levels	0.6907 nanogram(s)/milliliter n=25 Participants	1.0490 nanogram(s)/milliliter n=10 Participants	0.8733 nanogram(s)/milliliter n=5 Participants	0.1976 nanogram(s)/milliliter n=4 Participants	2.8763 nanogram(s)/milliliter n=3 Participants	0.8733 nanogram(s)/milliliter n=47 Participants
IGFBP-3	0.803 ng/mL n=25 Participants	0.789 ng/mL n=10 Participants	0.684 ng/mL n=5 Participants	0.716 ng/mL n=4 Participants	0.585 ng/mL n=3 Participants	0.786 ng/mL n=47 Participants

PRIMARY outcome

Timeframe: 13 Weeks

Population: Efficacy Analysis Set includes all subjects in Group 1 who received at least one dose of study drug.

Change from baseline in IGF-1 level at Week 13/End of Treatment (W13/EoT) in Group 1 subjects Efficacy Analysis Set (EAS).

Outcome measures

Measure	Group 1 n=25 Participants Subjects in Group 1 previously treated with octreotide or lanreotide	Group 4 Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN)	Group 5 Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)	Group 4 Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist(bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ ULN)	Group 5 Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)
Change From Baseline (Median of Screening Values) in Insulin-like Growth Factor-1 (IGF-1) Level	-0.034 ng/mL x ULN Interval -0.107 to 1.07	—	—	—	—

PRIMARY outcome

Timeframe: 13 Weeks

Population: Efficacy Analysis Set includes all subjects in Group 1 who received at least one dose of study drug.

Change from baseline in IGF-1 level at Week 13/End of Treatment (W13/EoT) in Group 1 subjects Efficacy Analysis Set (EAS).

Outcome measures

Measure	Group 1 n=25 Participants Subjects in Group 1 previously treated with octreotide or lanreotide	Group 4 Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN)	Group 5 Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)	Group 4 Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist(bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ ULN)	Group 5 Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)
Change From Baseline (Median of Screening Values) in Insulin-like Growth Factor-1 (IGF-1) Level Baseline	1.335 ng/mL x ULN Interval 1.078 to 1.471	—	—	—	—
Change From Baseline (Median of Screening Values) in Insulin-like Growth Factor-1 (IGF-1) Level Week 13 (Visit 14)/ End of Treatment	1.343 ng/mL x ULN Interval 1.169 to 1.448	—	—	—	—

SECONDARY outcome

Timeframe: 13 Weeks

Population: This endpoint was limited to only subjects in Groups 3, 4 and 5 who started the study with IGF-1 ≤ULN.

The secondary endpoint was the proportion of participants who maintained IGF-1 response, defined as the last assessment before the EoT with IGF-1 ≤1.0× ULN meet responder criteria, in Group 3, 4, and 5 subjects only at W13/EoT

Outcome measures

Measure	Group 1 n=5 Participants Subjects in Group 1 previously treated with octreotide or lanreotide	Group 4 n=4 Participants Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN)	Group 5 n=3 Participants Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)	Group 4 Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist(bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ ULN)	Group 5 Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)
Proportion of Subjects With Their Last IGF-1 Measurement ≤ Upper Limit of Normal (ULN) Baseline	5 Participants	4 Participants	3 Participants	—	—
Proportion of Subjects With Their Last IGF-1 Measurement ≤ Upper Limit of Normal (ULN) W13/EoT (Response)	2 Participants	1 Participants	1 Participants	—	—
Proportion of Subjects With Their Last IGF-1 Measurement ≤ Upper Limit of Normal (ULN) W13/EoT (No Response)	3 Participants	3 Participants	2 Participants	—	—

SECONDARY outcome

Timeframe: 13 Weeks

Population: This endpoint was estimated for all the subjects in the Full Analysis Set (FAS)

Proportion of participants with IGF-1 ≤1.5× ULN at W13/EoT.

Outcome measures

Measure	Group 1 n=25 Participants Subjects in Group 1 previously treated with octreotide or lanreotide	Group 4 n=10 Participants Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist (bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ULN)	Group 5 n=5 Participants Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)	Group 4 n=4 Participants Eligibility to enter this group was specified as: Complete responders on a stable combination treatment of SRL (ie, octreotide LAR or lanreotide depot) and a dopamine agonist(bromocriptine or cabergoline) (mean of Screening IGF-1 values were ≤ ULN)	Group 5 n=3 Participants Eligibility to enter this group was specified as: Complete responders on a stable dose of pasireotide LAR (mean of Screening IGF-1 values were ≤ULN)
Proportion of Subjects With Their Last IGF-1 Measurements ≤1.5×ULN Baseline (Response)	19 Participants	6 Participants	5 Participants	4 Participants	2 Participants
Proportion of Subjects With Their Last IGF-1 Measurements ≤1.5×ULN W13/EoT (Response)	20 Participants	4 Participants	4 Participants	3 Participants	1 Participants
Proportion of Subjects With Their Last IGF-1 Measurements ≤1.5×ULN W13/EoT (No Response)	5 Participants	6 Participants	1 Participants	1 Participants	2 Participants

Adverse Events

Dose 5 mg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dose 10 mg

Serious events: 0 serious events

Other events: 23 other events

Deaths: 0 deaths

Dose 20 mg

Serious events: 1 serious events

Other events: 24 other events

Deaths: 0 deaths

Dose 30 mg

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Dose 40 mg

Serious events: 0 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	Dose 5 mg n=2 participants at risk Adverse event occurred when subject was receiving 5 mg dose paltusotine	Dose 10 mg n=47 participants at risk Adverse event occurred when subject was receiving 10 mg dose paltusotine	Dose 20 mg n=43 participants at risk Adverse event occurred when subject was receiving 20 mg dose paltusotine	Dose 30 mg n=37 participants at risk Adverse event occurred when subject was receiving 30 mg dose paltusotine	Dose 40 mg n=32 participants at risk Adverse event occurred when subject was receiving 40 mg dose paltusotine
Nervous system disorders Headache	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/47 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.3% 1/43 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.

Other adverse events

Measure	Dose 5 mg n=2 participants at risk Adverse event occurred when subject was receiving 5 mg dose paltusotine	Dose 10 mg n=47 participants at risk Adverse event occurred when subject was receiving 10 mg dose paltusotine	Dose 20 mg n=43 participants at risk Adverse event occurred when subject was receiving 20 mg dose paltusotine	Dose 30 mg n=37 participants at risk Adverse event occurred when subject was receiving 30 mg dose paltusotine	Dose 40 mg n=32 participants at risk Adverse event occurred when subject was receiving 40 mg dose paltusotine
Cardiac disorders Palpitations	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/47 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.7% 2/43 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Nervous system disorders Headache	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	12.8% 6/47 • Number of events 6 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	11.6% 5/43 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	10.8% 4/37 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	21.9% 7/32 • Number of events 7 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Nervous system disorders Paresthesia	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.4% 3/47 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	11.6% 5/43 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	5.4% 2/37 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	9.4% 3/32 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
General disorders Fatigue	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	10.6% 5/47 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	9.3% 4/43 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	8.1% 3/37 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	12.5% 4/32 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Peripheral swelling	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.4% 3/47 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.7% 2/43 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.7% 1/37 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.2% 2/32 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Diarrhea	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	8.5% 4/47 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.7% 2/43 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.7% 1/37 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Abdominal pain	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	8.5% 4/47 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.3% 1/43 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Abdominal distension	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.4% 3/47 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/43 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Dyspepsia	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.1% 1/47 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.3% 1/43 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.7% 1/37 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.2% 2/32 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Flatulence	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.3% 2/47 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/43 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Respiratory, thoracic and mediastinal disorders Sleep apnea syndrome	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.3% 2/47 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.3% 1/43 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	2.7% 1/37 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	3.1% 1/32 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Skin and subcutaneous tissue disorders Hyperhidrosis	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	10.6% 5/47 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	9.3% 4/43 • Number of events 4 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	6.2% 2/32 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	10.6% 5/47 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	7.0% 3/43 • Number of events 3 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	5.4% 2/37 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	15.6% 5/32 • Number of events 5 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/47 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/43 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	5.4% 2/37 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	3.1% 1/32 • Number of events 1 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Metabolism and nutrition disorders Hyperglycemia	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/47 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.7% 2/43 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/37 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.
Gastrointestinal disorders Abdominal discomfort	0.00% 0/2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	4.3% 2/47 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/43 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	5.4% 2/37 • Number of events 2 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.	0.00% 0/32 • Participants were monitored until the end of the 4-week follow-up period (up to 17 weeks total). Some participants received uptitrated dose during the treatment period and are therefore counted in multiple dosing groups.

Additional Information

Crinetics Clinical Trials

Crinetics Pharmaceuticals, Inc

Phone: +1 858-450-6464

Email: clinicaltrials@crinetics.com

Results disclosure agreements

• Principal investigator is a sponsor employee The only disclosure restriction on the PI is that PIs cannot publish until the multi-center publication is first published. If no multi-center publication is published within 18 months of the completion date, the PI may publish their results, provided that the Sponsor is given the opportunity to review the proposed publication in advance.
• Publication restrictions are in place

Restriction type: OTHER