Clinical trial NCT01412424: Efficacy and Safety of Octreotide (MYCAPSSA™ [Formerly Octreolin™]) for Acromegaly
Official title: Efficacy and Safety of Oral Octreolin™ in Patients With Acromegaly Who Are Currently Receiving Parenteral Somatostatin Analogs
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Acromegaly
Keywords: acromegaly
IGF-1
growth hormone
Octreolin
octreotide
somatostatin analog
Lead sponsor: Chiasma, Inc.
Sponsor class: INDUSTRY
First posted: 2011-08-09
Enrollment: 155
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: MYCAPSSA™ (formerly Octreolin™) is a proprietary oral form of the approved injectable medical product octreotide used to treat acromegaly. This study will evaluate the efficacy and safety of MYCAPSSA™ treatment in patients with acromegaly.

Detailed description: The study consisted of 2 periods, a Core Treatment Period of up to 7 months and an optional Extension Treatment Period of up to 6 months, for a total study duration of up to 13 months. The Core Treatment Period consisted of 2 phases, a Dose Escalation Phase of at least 2 months to identify the therapeutic dose for each study participant and a Fixed Dose Phase of 2 to 5 months during which the therapeutic dose was maintained.

Participants were eligible to enter the Fixed Dose Phase of the Core Treatment Period if they were clinically and biochemically controlled. The same criteria were used to allow entry into the voluntary 6-month Extension Treatment Period.

The Core Treatment Period of the study was completed if the participant had at least 2 months of treatment in the Fixed Dose Phase and a total treatment duration of at least 7 months. Participants who elected to continue into the Extension Treatment Period maintained their therapeutic dose during this period. At the end of the study (after the last dose of MYCAPSSA in either the Core Treatment Period or the Extension Treatment Period), there was a 2-week follow-up period for safety assessments.

Eligibility criteria: Inclusion Criteria:

* Adult subjects, aged 18 to 75 years old, inclusive.
* Subjects with acromegaly defined as documented evidence of growth hormone-secreting pituitary tumor that is abnormally responsive to glucose, or documented elevated insulin-like growth factor-1 (IGF-1), who are currently receiving a stable dose of a somatostatin analog for at least the previous 3 months.
* A serum IGF-1 level \< 1.3 x the upper limit of normal (ULN) and a serum growth hormone (GH) level \< 2.5 ng/mL.
* Subjects able and willing to comply with the requirements of the protocol.
* Subjects able to swallow capsules.
* Subjects able to understand and sign written informed consent to participate in the study.

Exclusion Criteria:

* Receiving regular injections of a somatostatin analog less frequently than once a month, ie, longer than every 4 weeks.
* Symptomatic cholelithiasis.
* Received pituitary radiotherapy within ten years prior to screening.
* Undergone pituitary surgery within the prior 6 months.
* Any condition that may jeopardize study participation.
* Clinically significant gastrointestinal (GI), renal, or hepatic disease as determined by the Investigator.
* Conditions (eg, bariatric surgery) significantly affecting gastric acidity or emptying.
* Current use (within 1 month) of proton pump inhibitors (PPIs) and current chronic use of H2-antagonists.
* Female patients who are pregnant or lactating.
* Current or recent (\< 3 months) therapy with pegvisomant.
* Current or recent (\< 2 months) therapy with cabergoline.

Primary outcomes:
measure: Percentage of Responders at the End of the Core Treatment Period
timeFrame: End of the core treatment period (up to 7 months)
measure: Percentage of Responders at the End of the Extension Treatment Period
timeFrame: End of the extension treatment period (up to 13 months)
Secondary outcomes:
measure: Percentage of Participants With Specified IGF-1 and GH Concentrations at Baseline and at the End of the Core Treatment Period
timeFrame: Baseline and the end of the core treatment period (up to 7 months)
measure: Maintenance of Response During the Fixed Dose Phase of the Core Treatment Period
timeFrame: Beginning of the fixed dose phase of the core treatment period and the end of the core treatment period (up to 7 months)
measure: Percentage of Participants With Specified IGF-1 and GH Concentrations at the Beginning and at the End of the Extension Treatment Period
timeFrame: Beginning and the end of the extension treatment period (up to 6 months)
measure: Maintenance of Response During the Extension Treatment Period
timeFrame: Beginning of the extension treatment period and the end of the extension treatment period (up to 13 months)
measure: Percentage of Participants With Improved or Maintained Acromegaly Symptoms at the End of the Extension Treatment Period
timeFrame: Baseline and the end of the extension treatment period (up to 13 months)
measure: Percentage of Participants With ≥ 1, 2, or 3 Acromegaly Symptoms at Baseline and at the End of the Extension Treatment Period
timeFrame: Baseline and the end of the extension treatment period (up to 13 months)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Berlin
country: Germany
facility: Campus Charité Mitte
city: Hamburg
country: Germany
facility: ENDOC Center for Endocrine Tumors
city: Munich
country: Germany
facility: Medizinische Klinik Innenstadt
city: Munich
country: Germany
facility: Max Planck Institute of Psychiatry
city: Oldenburg
country: Germany
facility: Praxis for Endocrimology and Diabetology in Oldenburg
city: Budapest
country: Hungary
facility: Military Hospital, State Health Center 2nd Department of Internal Medicine
city: Budapest
country: Hungary
facility: Semmelweiss University
city: Pecs
country: Hungary
facility: University of Pecs
city: Szeged
country: Hungary
facility: University of Szeged
city: Brescia
country: Italy
facility: Servizio di Endocrinologia A.O. Spedali Civili di Brescia
city: Messina
country: Italy
facility: Dipartimento Clinico Sperimentale di Medicina e Farmacologia
city: Torino
country: Italy
facility: Ospedale Molinette
city: Kaunas
country: Lithuania
facility: Hospital of Lithuanian University of Health Sciences Kauno Klinikos
city: Vilnius
country: Lithuania
facility: Vilnius University Hospital Santariskiu Clinics Center of Endocrinology
city: Guadalajara Jalisco
country: Mexico
facility: Unidad de Investigacion Clinica Cardiometabolica de Occidente
city: Mexico City
country: Mexico
facility: Instituto Nacional de Neurologia y Neurocirugía - National Institute of Neurology and Neurosurgery
city: Mexico D.F.
country: Mexico
facility: Centro Medico ABC
city: Leiden
country: Netherlands
facility: Leiden University Medical Centre
city: Rotterdam
country: Netherlands
facility: Erasmus University Medical Center
city: Katowice
country: Poland
facility: Autonomous Public Clinical Hospital No. 5
city: Krakow
country: Poland
facility: Department of Endocrinology - Jagiellonian University, Krakow
city: Poznan
country: Poland
facility: Clinical Hospital of Medical University in Poznan
city: Warsaw
country: Poland
facility: Bielanski Hospital
city: Wroclaw
country: Poland
facility: Wroclaw Medical University
city: Bucharest
country: Romania
facility: Endocrinology Institute C.I.Parhon
city: Iasi
country: Romania
facility: County Emergency Hospital, Sf. Spiridon, Department of Endocrinology
city: Belgrade
country: Serbia
facility: Clinic for Endocrinology, Diabetes and Metabolism Diseases, Clinical Center of Serbia
city: Novi Sad
country: Serbia
facility: Clinical Center of Vojvodina
city: Bratislava
country: Slovakia
facility: University Hospital Bratislava, Hospital of L.Derer
city: Ľubochňa
country: Slovakia
facility: National Institute of Endocrinology and Diabetology
city: Ljubliana
country: Slovenia
facility: Department of Endocrinology and Diabetes, University Medical Centre
city: Coventry
country: United Kingdom
facility: University of Warwick - Medical School
city: London
country: United Kingdom
facility: St Bartholomew's Hospital West
city: Manchester
country: United Kingdom
facility: The Christie Hospital NHS Trust
city: Oxford
country: United Kingdom
facility: Oxford Centre for Diabetes, Endocrinology and Metabolism
Study officials:
affiliation: Cedars-Sinai Medical Center
name: Shlomo Melmed, MD
role: STUDY_CHAIR
References:
citation: Labadzhyan 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.
citation: 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.