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Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly

Phase 3
Conditions
Acromegaly
Interventions
Drug: Matching placebo
Registration Number
NCT03252353
Lead Sponsor
Chiasma, Inc.
Brief Summary

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

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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
56
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Octreotide capsulesoctreotide capsulesOctreotide capsules
Matching PlaceboMatching placeboMatching placebo capsules
Primary Outcome Measures
NameTimeMethod
Number of Patients Who Maintain Their Biochemical Response at the End of the Double Blind Placebo Controlled (DPC) Period.Week 36

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.

Secondary Outcome Measures
NameTimeMethod
Number of Patients Who Maintain Growth Hormone (GH) Response at the End of the Double Blind Placebo Controlled PeriodWeek 36

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.

Number of Patients Who Begin Rescue TreatmentWeek 36

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

Trial Locations

Locations (60)

Cedars-Sinai Medical Center

🇺🇸

Los Angeles, California, United States

UCLA Medical Center

🇺🇸

Los Angeles, California, United States

Ohio State University

🇺🇸

Columbus, Ohio, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

Royal Victoria Infirmary

🇬🇧

Newcastle upon Tyne, United Kingdom

Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

The Emory Clinic

🇺🇸

Atlanta, Georgia, United States

Johns Hopkins University Clinical Trials Unit

🇺🇸

Baltimore, Maryland, United States

Keck Medical Center of University of Southern California

🇺🇸

Los Angeles, California, United States

Ankara University, Faculty of Medicine

🇹🇷

Ankara, Turkey

Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Endokrynologii

🇵🇱

Krakow, Poland

Medical University Centre Ljubljana

🇸🇮

Ljubljana, Slovenia

Hacettepe University Medical School

🇹🇷

Ankara, Turkey

McGill University Health Centre

🇨🇦

Montreal, Quebec, Canada

St Vincent's Private Hospital-NSW

🇦🇺

Darlinghurst, New South Wales, Australia

Royal North Shore Public Hospital

🇦🇺

St Leonards, New South Wales, Australia

Keogh Institute (Sir Charles Gardner)

🇦🇺

Nedlands, Western Australia, Australia

Aarhus University Hospital

🇩🇰

Aarhus, Denmark

St Joseph's Health Care

🇨🇦

London, Ontario, Canada

University of Semmelweiss, Budapest

🇭🇺

Budapest, Hungary

Rigshospitalet The Department of Endocrinology

🇩🇰

Copenhagen, Denmark

Hadassah Ein-Karem Medical Center

🇮🇱

Jerusalem, Israel

Policlinico di Monserrato U.O.C. Endocrinologia e Diabetologia

🇮🇹

Monserrato, Italy

Klinika Chorob Wewnetrznych i Endokrynologii

🇵🇱

Warszawa, Poland

Erciyes University Medical Faculty

🇹🇷

Kayseri, Turkey

Rabin Medical Center, Beilinson Hospital

🇮🇱

Petah Tikva, Israel

Azienda Ospedaliero-Universitaria Pisana, Università di Pisa

🇮🇹

Pisa, Italy

Tel Aviv Sourasky Medical Center

🇮🇱

Tel Aviv, Israel

Riga Eastern Clinical University, Hospital Gailezers Department of Endocrinology

🇱🇻

Riga, Latvia

Sahlgrenska University Hospital

🇸🇪

Göteborg, Sweden

Ege University Medical Faculty Internal Diseases

🇹🇷

İzmir, Turkey

University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

🇬🇧

Birmingham, United Kingdom

University Specialized Hospital for Active Treatment of Endocrinology "Acad. Iv. Pencev" EAD

🇧🇬

Sofia, Bulgaria

Huntsman Cancer Hospital

🇺🇸

Salt Lake City, Utah, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Magyar Honvedseg Egeszsegugyi Kozpont, II. sz. Belgyogyaszati Osztaly

🇭🇺

Budapest, Hungary

Szegedi Tudományegyetem, I. Belgyógyászati Klinika

🇭🇺

Szeged, Hungary

Stanford University School of Medicine

🇺🇸

Palo Alto, California, United States

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

John H. Stroger Jr. Hospital of Cook County

🇺🇸

Chicago, Illinois, United States

University of Colorado

🇺🇸

Aurora, Colorado, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

University of British Columbia

🇨🇦

Vancouver, British Columbia, Canada

Allegheny Endocrinology Associates

🇺🇸

Pittsburgh, Pennsylvania, United States

RWTH Aachen University Hospital, Medical Clinic III Division of Endocrinology and Diabetes

🇩🇪

Aachen, Germany

Leids Universitair Medisch Centrum

🇳🇱

Leiden, Netherlands

Erasmus Medisch Centrum

🇳🇱

Rotterdam, Netherlands

Dunedin Hospital

🇳🇿

Dunedin, New Zealand

Uniwersyteckie Centrum Okulistyki i Onkologii Samodzielny Publiczny Szpital Kliniczny Slaskiego Uniwersytetu Medycznego w Katowicach, Oddzial Endokrynologii

🇵🇱

Katowice, Poland

Wellington Hospital

🇳🇿

Wellington, New Zealand

Central Manchester University Hospitals NHS Foundation Trust, Manchester Royal Infirmary

🇬🇧

Manchester, United Kingdom

Klinikum der LMU Muenchen, Medizinische Klinik und Poliklinik IV, Endokrinologie

🇩🇪

Munich, Germany

Thomas Jefferson University Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

St Vincent's Hospital-VIC

🇦🇺

Fitzroy, Victoria, Australia

Melbourne Health

🇦🇺

Parkville, Victoria, Australia

Katedra i Klinika Endokrynologii i Chorob Wewnetrznych

🇵🇱

Gdansk, Poland

Samodzielny Publiczny Szpital Kliniczny nr 1 we Wroclawiu, Klinika Endokrynologii, Diabetologii i Leczenia Izotopami

🇵🇱

Wroclaw, Poland

The Alfred

🇦🇺

Melbourne, Victoria, Australia

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