Trial Outcomes & Findings for Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly (NCT NCT03252353)

Last Updated: 2020-11-23

Results Overview

Maintenance of response was defined by using the average IGF-1 level of the last 2 available assessments in the DPC period. If the average IGF-1 is ≤ 1×ULN, a patient was classified as a responder (i.e., maintained their biochemical response). If the average IGF-1 is \> 1×ULN, a patient was classified as a non-responder. Patients who discontinue study medication during the DPC period for any reason was classified as non-responders for the primary analysis, regardless of their IGF-1 values.

Recruitment status

UNKNOWN

Study phase

PHASE3

Target enrollment

56 participants

Primary outcome timeframe

Week 36

Results posted on

2020-11-23

Participant Flow

Participant milestones

Participant milestones
Measure	Octreotide Capsules Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo Matching placebo capsules Matching placebo: Matching placebo capsules
Overall Study STARTED	28	28
Overall Study COMPLETED	28	28
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Octreotide Capsules n=28 Participants Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 Participants Matching placebo capsules Matching placebo: Matching placebo capsules	Total n=56 Participants Total of all reporting groups
Age, Continuous Age	55.3 years STANDARD_DEVIATION 11.97 • n=5 Participants	54.2 years STANDARD_DEVIATION 10.96 • n=7 Participants	54.7 years STANDARD_DEVIATION 11.38 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants	14 Participants n=7 Participants	30 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	14 Participants n=7 Participants	26 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=5 Participants	25 Participants n=7 Participants	53 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	27 Participants n=5 Participants	24 Participants n=7 Participants	51 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Baseline average Insulin-like Growth Factor 1 (IGF-1) (categorical) IGF≤ 1 Upper Limit of Normal (ULN)	27 Participants n=5 Participants	23 Participants n=7 Participants	50 Participants n=5 Participants
Baseline average Insulin-like Growth Factor 1 (IGF-1) (categorical) IGF > 1 to < 1.3 ULN	1 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants
Baseline average Insulin-like Growth Factor 1 (IGF-1) (categorical) IGF≥ 1.3 ULN	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 36

Population: Number of Patients who Maintained Their Biochemical Response at the End of the DPC Period

Outcome measures

Outcome measures
Measure	Octreotide Capsules n=28 Participants Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 Participants Matching placebo capsules
Number of Patients Who Maintain Their Biochemical Response at the End of the Double Blind Placebo Controlled (DPC) Period.	16 Participants	5 Participants

SECONDARY outcome

Timeframe: Week 36

Population: Number of patients who maintain Growth Hormone (GH) response at the end of the double blind placebo controlled period

Maintenance of GH response was defined as having mean Growth Hormone (5 measurements 30 minutes apart) \< 2.5 ng/mL at the end of the double blind placebo controlled period, out of those who were responders on Somatostatin Receptor Ligands (SRLs) injections at Screening.

Outcome measures

Outcome measures
Measure	Octreotide Capsules n=28 Participants Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 Participants Matching placebo capsules
Number of Patients Who Maintain Growth Hormone (GH) Response at the End of the Double Blind Placebo Controlled Period	21 Participants	7 Participants

SECONDARY outcome

Timeframe: Week 36

Population: The number of patients who began rescue treatment

Number of Patients who Began Rescue Treatment Prior to and Including Week 36

Outcome measures

Outcome measures
Measure	Octreotide Capsules n=28 Participants Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 Participants Matching placebo capsules
Number of Patients Who Begin Rescue Treatment	7 Participants	19 Participants

Adverse Events

Octreotide Capsules

Serious events: 2 serious events

Other events: 28 other events

Deaths: 0 deaths

Matching Placebo

Serious events: 1 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Octreotide Capsules n=28 participants at risk Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 participants at risk Matching placebo capsules Matching placebo: Matching placebo capsules
Hepatobiliary disorders Cholecystitis acute	3.6% 1/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Injury, poisoning and procedural complications Joint dislocation	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Arthritis	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.

Other adverse events

Additional Information

Asi Haviv, VP Clinical development

Chiasma

Phone: 972-8-939-3888

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER

Measure	Octreotide Capsules n=28 participants at risk Octreotide capsules octreotide capsules: octreotide capsules 40mg/day, 60mg/day, 80 mg/day (individual dose titration)	Matching Placebo n=28 participants at risk Matching placebo capsules Matching placebo: Matching placebo capsules
Gastrointestinal disorders Abdominal discomfort	14.3% 4/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Abdominal pain	7.1% 2/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Abdominal pain upper	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Constipation	10.7% 3/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	14.3% 4/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Diarrhoea	28.6% 8/28 • Number of events 9 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	21.4% 6/28 • Number of events 6 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Dyspepsia	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	3.6% 1/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Flatulence	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Large intestinal polyp	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Nausea	21.4% 6/28 • Number of events 7 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Tongue disorder	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Gastrointestinal disorders Vomiting	14.3% 4/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
General disorders Fatigue	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	25.0% 7/28 • Number of events 8 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
General disorders Influenza-like illness	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
General disorders Oedema peripheral	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
General disorders Pain	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
General disorders Peripheral swelling	10.7% 3/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	14.3% 4/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Hepatobiliary disorders Cholelithiasis	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Infections and infestations Nasopharyngitis	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	14.3% 4/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Infections and infestations Sinusitis	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Infections and infestations Upper respiratory tract infection	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Infections and infestations Urinary tract infection	7.1% 2/28 • Number of events 6 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	3.6% 1/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Investigations Blood glucose increased	10.7% 3/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	3.6% 1/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Investigations Gamma-glutamyltransferase increased	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Investigations Insulin-like growth factor increased	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Investigations Weight increased	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Metabolism and nutrition disorders Hypercholesterolaemia	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Arthralgia	32.1% 9/28 • Number of events 11 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	57.1% 16/28 • Number of events 28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Arthritis	7.1% 2/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Back pain	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	14.3% 4/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 4 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Osteoarthritis	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Musculoskeletal and connective tissue disorders Soft tissue swelling	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	10.7% 3/28 • Number of events 3 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Nervous system disorders Carpal tunnel syndrome	14.3% 4/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	14.3% 4/28 • Number of events 5 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Nervous system disorders Headache	14.3% 4/28 • Number of events 7 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	32.1% 9/28 • Number of events 17 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Nervous system disorders Hypoaesthesia	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Nervous system disorders Paraesthesia	0.00% 0/28 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Psychiatric disorders Anxiety	3.6% 1/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Skin and subcutaneous tissue disorders Hyperhidrosis	21.4% 6/28 • Number of events 10 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	25.0% 7/28 • Number of events 8 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.
Skin and subcutaneous tissue disorders Night sweats	3.6% 1/28 • Number of events 1 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.	7.1% 2/28 • Number of events 2 • 1 year, 10 months Safety population: All participants enrolled in the study who received any amount of the study drug.