Efficacy and Safety of Octreotide (MYCAPSSA™ [Formerly Octreolin™]) for Acromegaly

Efficacy and Safety of Oral Octreolin™ in Patients With Acromegaly Who Are Currently Receiving Parenteral Somatostatin Analogs

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.

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.

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.