Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly

Study Overview
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Brief Summary
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Octreotide capsule is a novel, orally-administered formulation of the commercially-available injectable drug octreotide. In a recent phase 3 trial, oral octreotide capsules demonstrated maintenance of biochemical response up to 13 months in the majority of patients with acromegaly previously managed with somatostatin analog injections (reference below).

Detailed Description
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This is a double blind, randomized study that assesses the efficacy and safety of octreotide capsules vs. placebo. Eligible acromegaly patients, treated with injectable somatostatin analogs, who are biochemically controlled and have prior evidence of active disease, will be randomized to receive either octreotide capsules or placebo for up to 36 weeks. At the end of this double blind, placebo controlled period, eligible patients will receive octreotide capsules in an open-labeled extension for at least one year. Patients failing to respond (per protocol), to oral treatment, (either placebo or octreotide capsules), will be rescued with the standard of care and upon meeting the eligibility criteria could also enroll into the long term extension with octreotide capsules. This study received agreement from the FDA, under a special protocol assessment.

Official Title
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A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of Octreotide Capsules in Patients Who Demonstrated Biochemical Control on Injectable Somatostatin Receptor Ligands (SRL) Treatment

Conditions
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Acromegaly

Intervention / Treatment
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* Drug: octreotide capsules
* Drug: Matching placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Documented evidence of active acromegaly Treatment with Somatostatin analogs injections (octreotide or lanreotide) for at least 6 months with a stable dose for at least the last three months of therapy Biochemically controlled Exclusion Criteria: Patients taking injections of long-acting Somatostatin Receptor Ligands (SRLs) not as indicated in the label Pituitary surgery within six months Conventional or stereotactic pituitary radiotherapy any time in the past Patients who previously participated in CH-ACM-01 or OOC-ACM-302 Any clinically significant uncontrolled concomitant disease Symptomatic cholelithiasis Pegvisomant, within 24 weeks Dopamine agonists, within 12 weeks Pasireotide, within 24 weeks

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Octreotide capsules<br>Octreotide capsules | Drug: octreotide capsules<br>* octreotide capsules 40 mg/day, 60 mg/day, 80 mg/day (individual dose titration)<br>* Other names: MYCAPSSA;|
| Placebo Comparator: Matching Placebo<br>Matching placebo capsules | Drug: Matching placebo<br>* Matching placebo capsules<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Patients Who Maintain Their Biochemical Response at the End of the Double Blind Placebo Controlled (DPC) Period. | 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. | Week 36 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Week 36 | 
| Number of Patients Who Begin Rescue Treatment | Number of Patients who Began Rescue Treatment Prior to and Including Week 36 | Week 36 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Octreotide Capsules, Acromegaly, OPTIMAL, MYCAPSSA