AcromegalyPhase 3OPTIMALNCT03252353
Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly
A Phase 3 study testing octreotide capsules in people with Acromegaly, run by Chiasma, Inc. at 60 sites in 17 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acromegaly
- What is being tested?
- octreotide capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 60 sitesin 17 countries
- Study dates
- 1 Sep 2017to May 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- Pegvisomant, within 24 weeks
- Dopamine agonists, within 12 weeks
- Pasireotide, within 24 weeks
Trial locations and contacts
60 sites in 17 countries.
60 sites in 17 countries
United States19 sites
The Emory Clinic
Atlanta, Georgia, 30322
University of Colorado
Aurora, Colorado, 80045
Johns Hopkins University Clinical Trials Unit
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
John H. Stroger Jr. Hospital of Cook County
Chicago, Illinois, 60612
Cleveland Clinic
Cleveland, Ohio, 44195
Ohio State University
Columbus, Ohio, 43203
Baylor College of Medicine
Houston, Texas, 77030
Keck Medical Center of University of Southern California
Los Angeles, California, 90033
Cedars-Sinai Medical Center
Los Angeles, California, 90048
UCLA Medical Center
Los Angeles, California, 90095
Columbia University Medical Center
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Stanford University School of Medicine
Palo Alto, California, 94304
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
Allegheny Endocrinology Associates
Pittsburgh, Pennsylvania, 15212
Oregon Health and Science University
Portland, Oregon, 97239
Huntsman Cancer Hospital
Salt Lake City, Utah, 84108
Washington University School of Medicine
St Louis, Missouri, 63110
Australia6 sites
St Vincent's Private Hospital-NSW
Darlinghurst, New South Wales, 2010
St Vincent's Hospital-VIC
Fitzroy, Victoria, 3065
The Alfred
Melbourne, Victoria, 3004
Keogh Institute (Sir Charles Gardner)
Nedlands, Western Australia, 6009
Melbourne Health
Parkville, Victoria, 3050
Royal North Shore Public Hospital
St Leonards, New South Wales, 2065
Poland5 sites
Katedra i Klinika Endokrynologii i Chorob Wewnetrznych
Gdansk, 80-211
Uniwersyteckie Centrum Okulistyki i Onkologii Samodzielny Publiczny Szpital Kliniczny Slaskiego Uniwersytetu Medycznego w Katowicach, Oddzial Endokrynologii
Katowice, 40-541
Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Endokrynologii
Krakow, 31-501
Klinika Chorob Wewnetrznych i Endokrynologii
Warsaw, 02-097
Samodzielny Publiczny Szpital Kliniczny nr 1 we Wroclawiu, Klinika Endokrynologii, Diabetologii i Leczenia Izotopami
Wroclaw, 50-367
Turkey (Türkiye)4 sites
Ankara University, Faculty of Medicine
Ankara, 6100
Hacettepe University Medical School
Ankara, 6100
Ege University Medical Faculty Internal Diseases
Izmir, 35040
Erciyes University Medical Faculty
Kayseri, 38080
Canada3 sites
St Joseph's Health Care
London, Ontario, N6A 4V2
McGill University Health Centre
Montreal, Quebec, H4A 3J1
University of British Columbia
Vancouver, British Columbia, V5Z 1M9
Hungary3 sites
Magyar Honvedseg Egeszsegugyi Kozpont, II. sz. Belgyogyaszati Osztaly
Budapest, H-1062
University of Semmelweiss, Budapest
Budapest, H-1088
Szegedi Tudományegyetem, I. Belgyógyászati Klinika
Szeged, H-6720
Israel3 sites
Hadassah Ein-Karem Medical Center
Jerusalem, 91120
Rabin Medical Center, Beilinson Hospital
Petah Tikva, 49100
Tel Aviv Sourasky Medical Center
Tel Aviv, 64239
United Kingdom3 sites
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Birmingham, B15 2GW
Central Manchester University Hospitals NHS Foundation Trust, Manchester Royal Infirmary
Manchester, M13 9WL
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Denmark2 sites
Aarhus University Hospital
Aarhus, 8000
Rigshospitalet The Department of Endocrinology
Copenhagen, 2100
Germany2 sites
RWTH Aachen University Hospital, Medical Clinic III Division of Endocrinology and Diabetes
Aachen, 52074
Klinikum der LMU Muenchen, Medizinische Klinik und Poliklinik IV, Endokrinologie
Munich, 80336
Italy2 sites
Policlinico di Monserrato U.O.C. Endocrinologia e Diabetologia
Monserrato, 09042
Azienda Ospedaliero-Universitaria Pisana, Università di Pisa
Pisa, 56124
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden, 2333 ZA
Erasmus Medisch Centrum
Rotterdam, 3015 CE
New Zealand2 sites
Dunedin Hospital
Dunedin, 9016
Wellington Hospital
Wellington, 6021
Bulgaria1 site
University Specialized Hospital for Active Treatment of Endocrinology "Acad. Iv. Pencev" EAD
Sofia, 1431
Latvia1 site
Riga Eastern Clinical University, Hospital Gailezers Department of Endocrinology
Riga, 1038
About this study
Official title: 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
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).
Read the 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.
- Conditions
- Acromegaly
- Being tested
- octreotide capsules, Matching placebo
- Keywords
- Octreotide Capsules, Acromegaly, OPTIMAL, MYCAPSSA
- Medical subject headings
- Acromegaly
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
octreotide capsulesDrug
Also called MYCAPSSA
octreotide capsules 40 mg/day, 60 mg/day, 80 mg/day (individual dose titration)
Matching placeboDrug
Also called Placebo
Matching placebo capsules
| Study group | Role | What they receive |
|---|---|---|
| Octreotide capsules | Active comparator | Octreotide capsules Receives: Drug: octreotide capsules |
| Matching Placebo | Placebo comparator | Matching placebo capsules Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 36
Secondary outcomes (2)
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.
Measured: Week 36
Number of Patients Who Begin Rescue Treatment
Number of Patients who Began Rescue Treatment Prior to and Including Week 36
Measured: Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2017
- Study start1 Sep 2017
- Primary completion13 Jun 2019
- Study completionMay 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for AcromegalyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiasma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Susan L Samson, MD PhD, Study chair, Pituitary Center at Baylor St. Luke's Medical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03252353
- Sponsor's protocol ID
- OOC-ACM-303
- Acronym
- OPTIMAL
- Status last verified
- Nov 2020
- First submitted
- 10 Aug 2017
- Last updated
- 23 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Melmed S, Popovic V, Bidlingmaier M, Mercado M, van der Lely AJ, Biermasz N, Bolanowski M, Coculescu M, Schopohl J, Racz K, Glaser B, Goth M, Greenman Y, Trainer P, Mezosi E, Shimon I, Giustina A, Korbonits M, Bronstein MD, Kleinberg D, Teichman S, Gliko-Kabir I, Mamluk R, Haviv A, Strasburger C. Safety and efficacy of oral octreotide in acromegaly: results of a multicenter phase III trial. J Clin Endocrinol Metab. 2015 Apr;100(4):1699-708. doi: 10.1210/jc.2014-4113. Epub 2015 Feb 9.
PubMed 25664604Tuvia S, Atsmon J, Teichman SL, Katz S, Salama P, Pelled D, Landau I, Karmeli I, Bidlingmaier M, Strasburger CJ, Kleinberg DL, Melmed S, Mamluk R. Oral octreotide absorption in human subjects: comparable pharmacokinetics to parenteral octreotide and effective growth hormone suppression. J Clin Endocrinol Metab. 2012 Jul;97(7):2362-9. doi: 10.1210/jc.2012-1179. Epub 2012 Apr 26.
PubMed 22539587Labadzhyan A, Nachtigall LB, Fleseriu M, Gordon MB, Molitch M, Kennedy L, Samson SL, Greenman Y, Biermasz N, Bolanowski M, Haviv A, Ludlam W, Patou G, Strasburger CJ. Oral octreotide capsules for the treatment of acromegaly: comparison of 2 phase 3 trial results. Pituitary. 2021 Dec;24(6):943-953. doi: 10.1007/s11102-021-01163-2. Epub 2021 Jun 25.
PubMed 34173129Samson SL, Nachtigall LB, Fleseriu M, Gordon MB, Bolanowski M, Labadzhyan A, Ur E, Molitch M, Ludlam WH, Patou G, Haviv A, Biermasz N, Giustina A, Trainer PJ, Strasburger CJ, Kennedy L, Melmed S. Maintenance of Acromegaly Control in Patients Switching From Injectable Somatostatin Receptor Ligands to Oral Octreotide. J Clin Endocrinol Metab. 2020 Oct 1;105(10):e3785-97. doi: 10.1210/clinem/dgaa526.
PubMed 32882036
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03252353
- Acronym / sponsor ID
- OPTIMAL / OOC-ACM-303
- Conditions
- Acromegaly
- Keywords
- Octreotide Capsules, Acromegaly, OPTIMAL, MYCAPSSA
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Chiasma, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2017 (actual)
- Primary completion
- 13 Jun 2019 (actual)
- Study completion
- May 2022 (estimated)
- First posted
- 17 Aug 2017 (actual)
- Results
- Posted 19 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Octreotide capsules | Active comparator | Drug: octreotide capsulesOctreotide capsules |
| Matching Placebo | Placebo comparator | Drug: Matching placeboMatching placebo capsules |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| octreotide capsulesAlso: MYCAPSSAoctreotide capsules 40 mg/day, 60 mg/day, 80 mg/day (individual dose titration) | Drug | Octreotide capsules |
| Matching placeboAlso: PlaceboMatching placebo capsules | Drug | Matching Placebo |
Primary outcome
Number of Patients Who Maintain Their Biochemical Response at the End of the Double Blind Placebo Controlled (DPC) Period.
Time frame: 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.
2 secondary outcomes
Number of Patients Who Maintain Growth Hormone (GH) Response at the End of the Double Blind Placebo Controlled Period
Time frame: Week 36
Number of Patients Who Begin Rescue Treatment
Time frame: Week 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 25664604Melmed S, Popovic V, Bidlingmaier M, Mercado M, van der Lely AJ, Biermasz N, Bolanowski M, Coculescu M, Schopohl J, Racz K, Glaser B, Goth M, Greenman Y, Trainer P, Mezosi E, Shimon I, Giustina A, Korbonits M, Bronstein MD, Kleinberg D, Teichman S, Gliko-Kabir I, Mamluk R, Haviv A, Strasburger C. Safety and efficacy of oral octreotide in acromegaly: results of a multicenter phase III trial. J Clin Endocrinol Metab. 2015 Apr;100(4):1699-708. doi: 10.1210/jc.2014-4113. Epub 2015 Feb 9.
- Background: PubMed 22539587Tuvia S, Atsmon J, Teichman SL, Katz S, Salama P, Pelled D, Landau I, Karmeli I, Bidlingmaier M, Strasburger CJ, Kleinberg DL, Melmed S, Mamluk R. Oral octreotide absorption in human subjects: comparable pharmacokinetics to parenteral octreotide and effective growth hormone suppression. J Clin Endocrinol Metab. 2012 Jul;97(7):2362-9. doi: 10.1210/jc.2012-1179. Epub 2012 Apr 26.
- Derived from this study: PubMed 34173129Labadzhyan A, Nachtigall LB, Fleseriu M, Gordon MB, Molitch M, Kennedy L, Samson SL, Greenman Y, Biermasz N, Bolanowski M, Haviv A, Ludlam W, Patou G, Strasburger CJ. Oral octreotide capsules for the treatment of acromegaly: comparison of 2 phase 3 trial results. Pituitary. 2021 Dec;24(6):943-953. doi: 10.1007/s11102-021-01163-2. Epub 2021 Jun 25.
- Derived from this study: PubMed 32882036Samson SL, Nachtigall LB, Fleseriu M, Gordon MB, Bolanowski M, Labadzhyan A, Ur E, Molitch M, Ludlam WH, Patou G, Haviv A, Biermasz N, Giustina A, Trainer PJ, Strasburger CJ, Kennedy L, Melmed S. Maintenance of Acromegaly Control in Patients Switching From Injectable Somatostatin Receptor Ligands to Oral Octreotide. J Clin Endocrinol Metab. 2020 Oct 1;105(10):e3785-97. doi: 10.1210/clinem/dgaa526.
- Chiasma Web PageLink provided by the sponsor.
- Melmed et al. JCEM 2015Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susan L Samson, MD PhD study chair
Pituitary Center at Baylor St. Luke's Medical
60 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing octreotide capsules in people with Acromegaly, run by Chiasma, Inc. at 60 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 60 sites in United States, Australia, Poland, Turkey (Türkiye), Canada and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Who Maintain Their Biochemical Response at the End of the Double Blind Placebo Controlled (DPC) Period. (time frame: Week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03252353, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Nov 2020.