Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (ACROBAT Evolve) (NCT NCT03792555)
NCT ID: NCT03792555
Last Updated: 2025-03-17
Results Overview
Proportion of subjects who meet responder criteria (based on the mean of two consecutive IGF-1 measurements ≤ upper limit of normal \[ULN\])
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
13 Weeks
Results posted on
2025-03-17
Participant Flow
Medically stable male and female subjects 18 to 75 years of age, inclusive, with a confirmed acromegaly diagnosis that was controlled on a stable approved dose of octreotide LAR or lanreotide depot. At a minimum, there had to be documentation available of a pituitary tumour and elevated IGF-1 in the past.
Medically stable male and female participants 18 to 75 years of age, with a confirmed acromegaly diagnosis that was controlled on a stable approved dose of octreotide LAR or lanreotide depot. There had to be documentation available of a pituitary tumour and elevated IGF-1 in the past.
Participant milestones
| Measure |
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Paltusotine
Titration Period started with the first dose of study drug (10mg all subjects) at V3, approximately 4 weeks after V1b when the last dose of standard acromegaly treatment was administered. Subjects who enrolled in the Titration Period did not receive any depot somatostatin receptor ligands (SRL) injections or other standard acromegaly treatment until study completion or early termination.
At V6/W4, the study drug dose was titrated up in a blinded fashion, if the current dose was tolerated by the subject and the IGF-1 value at V4/W2 was \>0.9×ULN (upper limit of normal for normal IGF-1 values for a specific age and sex). At V9/W7, the study drug was up-titrated in a blinded fashion if the current dose was tolerated by the subject and the IGF-1 value at V7/W5 was \>ULN.
Investigator confirmed the subject eligibility for an increase in study drug dose based on tolerability at V5/W3, V6/W4, V8/W6, and V9/W7. If tolerability was confirmed and IGF-1 reader determined that IGF-1 had not met adequate suppression criteria, a blinded blister card containing the appropriate higher dose was assigned at the V6/W4 and/or V9/W7. Dose increases in 10 mg increments were allowed only at V6/W4 (from 10 mg to 20 mg) and V9/W7 (from 10 mg to 20 mg, or from 20 mg to 30 mg) visits. No further up-titration was allowed. The daily dose did not exceed 30 mg.
If eligible for randomization, the subject was randomized at V11/W10 in a blinded manner to continue the current dose of paltusotine.
|
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Placebo
Titration Period started with the first dose of study drug (10 mg for all subjects) at V3, approximately 4 weeks after V1b when the last dose of standard acromegaly treatment was administered. Subjects who enrolled in the Titration Period did not receive any depot somatostatin receptor ligands (SRL) depot injections or other standard acromegaly treatment until study completion or early termination.
At V6/W4, the study drug dose was titrated up in a blinded fashion, if the current dose was tolerated by the subject and the IGF-1 value at V4/W2 was \>0.9×ULN. Again, at V9/W7, the study drug was up-titrated in a blinded fashion if the current dose was tolerated by the subject and the IGF-1 value at V7/W5 was \>ULN.
The Investigator confirmed the subject was eligible for an increase in the study drug dose based on tolerability at V5/W3, V6/W4, V8/W6, and V9/W7. If tolerability was confirmed and the IGF-1 reader had determined that IGF-1 had not met adequate suppression criteria, a blinded blister card that contained the appropriate higher dose was assigned at the V6/W4 and/or V9/W7. Dose increases in 10 mg increments were allowed only at the V6/W4 (from 10 mg to 20 mg) and V9/W7 (from 10 mg to 20 mg, or from 20 mg to 30 mg) visits. No further up-titration was allowed. The daily dose did not exceed 30 mg.
If determined to be eligible for randomization, the subject was randomized at V11/W10 in a blinded manner and switched to placebo.
|
Titration Period: Paltusotine, Then Not Randomized
Titration period proceeded for these subjects in the same manner as other groups.
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Screening Period: 4-6 Weeks
STARTED
|
3
|
4
|
6
|
|
Screening Period: 4-6 Weeks
COMPLETED
|
3
|
4
|
6
|
|
Screening Period: 4-6 Weeks
NOT COMPLETED
|
0
|
0
|
0
|
|
Titration Period: up to 9 Weeks
STARTED
|
3
|
4
|
6
|
|
Titration Period: up to 9 Weeks
COMPLETED
|
3
|
4
|
5
|
|
Titration Period: up to 9 Weeks
NOT COMPLETED
|
0
|
0
|
1
|
|
Randomized Withdrawal: up to 4 Weeks
STARTED
|
3
|
4
|
5
|
|
Randomized Withdrawal: up to 4 Weeks
COMPLETED
|
3
|
4
|
0
|
|
Randomized Withdrawal: up to 4 Weeks
NOT COMPLETED
|
0
|
0
|
5
|
|
Follow-up Period: up to 4 Weeks
STARTED
|
3
|
4
|
5
|
|
Follow-up Period: up to 4 Weeks
COMPLETED
|
3
|
3
|
5
|
|
Follow-up Period: up to 4 Weeks
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Paltusotine
Titration Period started with the first dose of study drug (10mg all subjects) at V3, approximately 4 weeks after V1b when the last dose of standard acromegaly treatment was administered. Subjects who enrolled in the Titration Period did not receive any depot somatostatin receptor ligands (SRL) injections or other standard acromegaly treatment until study completion or early termination.
At V6/W4, the study drug dose was titrated up in a blinded fashion, if the current dose was tolerated by the subject and the IGF-1 value at V4/W2 was \>0.9×ULN (upper limit of normal for normal IGF-1 values for a specific age and sex). At V9/W7, the study drug was up-titrated in a blinded fashion if the current dose was tolerated by the subject and the IGF-1 value at V7/W5 was \>ULN.
Investigator confirmed the subject eligibility for an increase in study drug dose based on tolerability at V5/W3, V6/W4, V8/W6, and V9/W7. If tolerability was confirmed and IGF-1 reader determined that IGF-1 had not met adequate suppression criteria, a blinded blister card containing the appropriate higher dose was assigned at the V6/W4 and/or V9/W7. Dose increases in 10 mg increments were allowed only at V6/W4 (from 10 mg to 20 mg) and V9/W7 (from 10 mg to 20 mg, or from 20 mg to 30 mg) visits. No further up-titration was allowed. The daily dose did not exceed 30 mg.
If eligible for randomization, the subject was randomized at V11/W10 in a blinded manner to continue the current dose of paltusotine.
|
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Placebo
Titration Period started with the first dose of study drug (10 mg for all subjects) at V3, approximately 4 weeks after V1b when the last dose of standard acromegaly treatment was administered. Subjects who enrolled in the Titration Period did not receive any depot somatostatin receptor ligands (SRL) depot injections or other standard acromegaly treatment until study completion or early termination.
At V6/W4, the study drug dose was titrated up in a blinded fashion, if the current dose was tolerated by the subject and the IGF-1 value at V4/W2 was \>0.9×ULN. Again, at V9/W7, the study drug was up-titrated in a blinded fashion if the current dose was tolerated by the subject and the IGF-1 value at V7/W5 was \>ULN.
The Investigator confirmed the subject was eligible for an increase in the study drug dose based on tolerability at V5/W3, V6/W4, V8/W6, and V9/W7. If tolerability was confirmed and the IGF-1 reader had determined that IGF-1 had not met adequate suppression criteria, a blinded blister card that contained the appropriate higher dose was assigned at the V6/W4 and/or V9/W7. Dose increases in 10 mg increments were allowed only at the V6/W4 (from 10 mg to 20 mg) and V9/W7 (from 10 mg to 20 mg, or from 20 mg to 30 mg) visits. No further up-titration was allowed. The daily dose did not exceed 30 mg.
If determined to be eligible for randomization, the subject was randomized at V11/W10 in a blinded manner and switched to placebo.
|
Titration Period: Paltusotine, Then Not Randomized
Titration period proceeded for these subjects in the same manner as other groups.
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Titration Period: up to 9 Weeks
COVID-19
|
0
|
0
|
1
|
|
Randomized Withdrawal: up to 4 Weeks
Participants who did not meet randomization criteria did not enter the Randomized Withdrawal Period.
|
0
|
0
|
5
|
|
Follow-up Period: up to 4 Weeks
COVID-19
|
0
|
1
|
0
|
Baseline Characteristics
All demographics and baseline characteristics presented are based on the titration period baseline.
Baseline characteristics by cohort
| Measure |
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Paltusotine
n=3 Participants
During the titration period, the study drug dose was titrated up in a blinded fashion. At randomization for the randomized withdrawal period, these subjects continued to receive paltusotine at the same level as end of the titration period.
|
Titration Period: Paltusotine, Then Randomized Withdrawal Period: Placebo
n=4 Participants
During the titration period, the study drug dose was titrated up in a blinded fashion. At randomization for the randomized withdrawal period, these subjects were switched to placebo.
|
Titration Period: Paltusotine, Then Not Randomized
n=6 Participants
Titration period proceeded for these subjects in the same manner as other groups.
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
65.0 years
STANDARD_DEVIATION 3.00 • n=5 Participants
|
38.8 years
STANDARD_DEVIATION 8.66 • n=7 Participants
|
57.7 years
STANDARD_DEVIATION 11.48 • n=5 Participants
|
53.5 years
STANDARD_DEVIATION 13.76 • n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
10 Participants
n=4 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
|
|
Race/Ethnicity, Customized
White
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
UGT1A1 Genotype
*1/*1
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
UGT1A1 Genotype
*1/*80
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
UGT1A1 Genotype
*80/*80
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
UGT1A1 Genotype
Not reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
UGT1A1 Phenotype
Normal Metabolizer
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
UGT1A1 Phenotype
Intermediate Metabolizer
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
UGT1A1 Phenotype
Poor Metabolizer
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
UGT1A1 Phenotype
Not reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Height
|
163.07 cm
STANDARD_DEVIATION 8.100 • n=5 Participants
|
181.90 cm
STANDARD_DEVIATION 14.461 • n=7 Participants
|
167.73 cm
STANDARD_DEVIATION 12.502 • n=5 Participants
|
171.02 cm
STANDARD_DEVIATION 13.748 • n=4 Participants
|
|
Weight
|
70.83 kg
STANDARD_DEVIATION 15.942 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
89.05 kg
STANDARD_DEVIATION 10.120 • n=7 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
80.65 kg
STANDARD_DEVIATION 22.542 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
80.97 kg
STANDARD_DEVIATION 18.088 • n=4 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
|
BMI
|
26.37 kg/m2
STANDARD_DEVIATION 3.371 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
26.95 kg/m2
STANDARD_DEVIATION 1.760 • n=7 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
28.48 kg/m2
STANDARD_DEVIATION 7.008 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
27.52 kg/m2
STANDARD_DEVIATION 4.903 • n=4 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
|
Ring Size
|
13.7 Scores on a Scale
STANDARD_DEVIATION 3.51 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
14.5 Scores on a Scale
STANDARD_DEVIATION 5.45 • n=7 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
11.3 Scores on a Scale
STANDARD_DEVIATION 1.86 • n=5 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
12.8 Scores on a Scale
STANDARD_DEVIATION 3.63 • n=4 Participants • All demographics and baseline characteristics presented are based on the titration period baseline.
|
PRIMARY outcome
Timeframe: 13 WeeksPopulation: The Analysis Population includes all subjects who received treatment in the titration period. This includes subjects who were randomized in the randomized withdrawal period to receive placebo or continue receiving paltusotine and subjects who were not randomized and continued receiving paltusotine. The primary endpoint of the proportion of subjects who met responder criteria (based on the mean of two consecutive IGF-1 measurements ≤ULN) at Week 13 is presented for all subjects.
Proportion of subjects who meet responder criteria (based on the mean of two consecutive IGF-1 measurements ≤ upper limit of normal \[ULN\])
Outcome measures
| Measure |
Randomized Withdrawal Period: Paltusotine
n=3 Participants
Subjects receiving paltusotine during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to continue receiving paltusotine at the same level as end of titration period.
|
Randomized Withdrawal Period: Placebo
n=4 Participants
Subjects receiving placebo during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to be switched to a placebo.
|
Randomized Withdrawal Period: Not Randomized
n=6 Participants
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Responder Criteria Was Based on the Mean of Two Consecutive Insulin-like Growth Factor-1 [IGF-1] Measurements ≤ULN at Week 13
|
66.7 percentage of responders
Interval 9.4 to 99.2
|
25.0 percentage of responders
Interval 0.6 to 80.6
|
16.7 percentage of responders
Interval 0.4 to 64.1
|
SECONDARY outcome
Timeframe: From Week 10 to Week 13Population: Analysis Population includes all subjects who met protocol pre-specified criteria for randomization into paltusotine/placebo. Per the study design, Randomized Withdrawal Period (RWP) baseline only applies to those subjects who met criteria for randomization. RWP baseline is undefined for non-randomized subjects. Demographics and Baseline Characteristics were reported for all subjects at Titration Period Baseline, but not RWP baseline.
Change in IGF-1 levels between RWP Baseline/Week 10 and Week 13
Outcome measures
| Measure |
Randomized Withdrawal Period: Paltusotine
n=3 Participants
Subjects receiving paltusotine during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to continue receiving paltusotine at the same level as end of titration period.
|
Randomized Withdrawal Period: Placebo
n=4 Participants
Subjects receiving placebo during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to be switched to a placebo.
|
Randomized Withdrawal Period: Not Randomized
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Change in IGF-1 Levels
|
4.04 percent change in IGF1
Standard Deviation 5.064
|
48.17 percent change in IGF1
Standard Deviation 41.408
|
—
|
SECONDARY outcome
Timeframe: From Week 8 to Week 13Population: Analysis Population includes all subjects who met protocol pre-specified criteria for randomization into paltusotine/placebo. Per the study design, Randomized Withdrawal Period (RWP) baseline only applies to those subjects who met criteria for randomization. RWP baseline is undefined for non-randomized subjects. Demographics and Baseline Characteristics were reported for all subjects at Titration Period Baseline, but not RWP baseline.
Change in growth hormone levels between RWP Baseline/Week 8 and Week 13
Outcome measures
| Measure |
Randomized Withdrawal Period: Paltusotine
n=3 Participants
Subjects receiving paltusotine during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to continue receiving paltusotine at the same level as end of titration period.
|
Randomized Withdrawal Period: Placebo
n=4 Participants
Subjects receiving placebo during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to be switched to a placebo.
|
Randomized Withdrawal Period: Not Randomized
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Change in Growth Hormone (GH) Levels
|
71.96 percent change in GH
Standard Deviation 48.865
|
108.30 percent change in GH
Standard Deviation 179.977
|
—
|
SECONDARY outcome
Timeframe: From Week 10 to Week 13Population: Analysis Population includes all subjects who met protocol pre-specified criteria for randomization into paltusotine/placebo. Per the study design, Randomized Withdrawal Period (RWP) baseline only applies to those subjects who met criteria for randomization. RWP baseline is undefined for non-randomized subjects. Demographics and Baseline Characteristics were reported for all subjects at Titration Period Baseline, but not RWP baseline.
Measured by total Acromegaly symptom diary (ASD) score from W10-W13. ASD is a sponsor-developed daily diary to assess important acromegaly symptoms from the patient perspective. It covers 7 symptoms (headache pain, joint pain, sweating, fatigue, weakness in legs, swelling, and numbness or tingling). Patients rate the severity of each experience in the past 24 hours on an 11-point numeric scale that ranges from 0 (no symptom) to 10 (worst symptom). A weekly average ASD score is calculated for each item as the sum of the item responses for a specific item over the course of the study week divided by the number of days on which the item was completed. The weekly average ASD total score is calculated by computing the sum of the weekly average item scores for the 7 items (total range from 0 to 70), where higher score = higher symptom severity.
Outcome measures
| Measure |
Randomized Withdrawal Period: Paltusotine
n=3 Participants
Subjects receiving paltusotine during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to continue receiving paltusotine at the same level as end of titration period.
|
Randomized Withdrawal Period: Placebo
n=4 Participants
Subjects receiving placebo during the Randomized Withdrawal Period (RWP):
Eligibility for randomization into the RWP was made based on information at the V10/W8 and V11/W10. To be eligible for randomization, the participant had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the participant tolerated the study drug. The participants were randomized at V11/W10 in a blinded manner to be switched to a placebo.
|
Randomized Withdrawal Period: Not Randomized
To be eligible for randomization, the subject had to have IGF-1 value ≤ULN at V10/W8 and an Investigator determination at V11/W10 that the subject tolerated the study drug. Subjects who did not meet both criteria were not to be randomized but were to be allowed to stay in the study and continue on a study drug dose that was tolerated until completion of all study visits or until criteria to resume standard acromegaly therapy and discontinuation from the study were met. The dose of study drug used at V9/W7 remained stable through V11/W10 if tolerated.
|
|---|---|---|---|
|
Change in Total ASD Score Between RWP Baseline/Week 10 and Week 13
|
1.571 Total ASD score change -units on a scale
Standard Deviation 3.1004
|
7.962 Total ASD score change -units on a scale
Standard Deviation 7.1591
|
—
|
Adverse Events
Safety Analysis Set/Titration Period - Paltusotine
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Randomized Withdrawal Period - Paltusotine
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Randomized Withdrawal Period - Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Total Paltusotine
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Safety Analysis Set/Titration Period - Paltusotine
n=13 participants at risk
All participants who received a dose of Paltusotine during the titration period.
|
Randomized Withdrawal Period - Paltusotine
n=3 participants at risk
Adverse events reported during the randomised withdrawal period by those receiving Paltusotine treatment
|
Randomized Withdrawal Period - Placebo
n=4 participants at risk
Adverse events reported during the randomised withdrawal period by those receiving Placebo
|
Total Paltusotine
n=13 participants at risk
All participants who received a dose of paltusotine. Includes all participants randomized into the RWP, and those not randomized that were allowed to stay in the study and continue dosing at a study drug dose that was tolerated, until the participant completed all study visits.
|
|---|---|---|---|---|
|
Vascular disorders
Hypertension
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Investigations
Blood glucose increased
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Investigations
Blood triglycerides increased
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Nervous system disorders
Headache
|
23.1%
3/13 • Number of events 3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
30.8%
4/13 • Number of events 4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Nervous system disorders
Paraesthesia
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
General disorders
Fatigue
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
23.1%
3/13 • Number of events 3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
General disorders
Peripheral swelling
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
23.1%
3/13 • Number of events 3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
General disorders
Pain
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Psychiatric disorders
Anxiety
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Psychiatric disorders
Sleep disorder
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
50.0%
2/4 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
33.3%
1/3 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Infections and infestations
Nasopharyngitis
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
15.4%
2/13 • Number of events 2 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Infections and infestations
COVID-19
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/4 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
7.7%
1/13 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/3 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
25.0%
1/4 • Number of events 1 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
0.00%
0/13 • Duration of participation for each subject after obtaining a signed informed consent was up to 23 weeks.
Safety analysis set: All participants enrolled in the study who received any amount of the study drug. All AEs reported in ≥ 1 subject are presented.
|
Additional Information
Crinetics Clinical Trials
Crinetics Pharmaceuticals Inc.
Phone: +1 858-450-6464
Email: [email protected]
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