A phase 2, randomized, parallel-group study to evaluate the safety, pharmacokinetics, and dose response of paltusotine treatment in patients with carcinoid syndrome

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Introduction: While long-acting somatostatin receptor ligands (SRLs) are mainstay treatments for carcinoid syndrome (CS), symptom relief at labelled doses is inadequate for many patients. Paltusotine is a novel oral, nonpeptide, selective somatostatin receptor type 2 agonist. In patients with acromegaly, paltusotine maintains plasma IGF-1 levels in patients switching from long-acting SRLs. Paltusotine was well tolerated in phase 2 acromegaly studies.

Aim(s): This randomized, open-label, parallel-group, multicenter study examines the safety, tolerability, pharmacokinetics, and exploratory efficacy of paltusotine in patients with CS.

Materials and methods: Patients with documented, well-differentiated, grade I or II neuroendocrine tumors (NETs) and CS, who are naïve to therapy with SRLs or have symptomatic control (bowel movement and flushing frequency) on SRLs, are eligible to participate. An electronic diary is used to capture symptom frequency and severity. Patients discontinuing SRLs will be eligible for randomization when symptom worsening occurs over any 7-day period. After screening, patients will be randomly assigned to open-label paltusotine 40 mg or 80 mg daily for 8 weeks. In addition to collection of safety data and serum paltusotine levels, symptom and biomarker responses will be assessed. Patients who complete the randomized treatment phase may be eligible to enter the extension phase and receive paltusotine for an additional 50 weeks.

Conference:

Presenting Author:

Authors: Chauhan A, Shaheen S, Usiskin K, Odueyungbo A, Mui C,

Keywords: Paltusotine, Phase 2 trial, Somatostatin receptor agonist, Safety, Pharmacokinetics, Carcinoid syndrome,