The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and exploratory dose response of paltusotine treatment in subjects with carcinoid syndrome. This study consists of a Randomized Treatment Phase followed by an Open-Label Extension (OLE) Phase.
This is a Phase 2, randomized, open-label, parallel-group, multicenter study designed to evaluate the safety, pharmacokinetics, and efficacy of paltusotine treatment in subjects with carcinoid syndrome. The study was conducted in 2 parts: a Randomized Treatment Phase (RTP) which is completed, and an Open-label Extension (OLE) Phase which is still ongoing. The RTP consisted of paltusotine treatment for 8 weeks. Subjects who completed the RTP were eligible to enter the OLE Phase at the recommendation of the Investigator. In the ongoing OLE Phase, paltusotine is being administered for a further 102 weeks. The total duration of paltusotine treatment for the combined RTP and OLE Phase is up to 110 weeks (28 months).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Two 20 mg tablets QD (Potential post-randomization dose escalation based on efficacy and acceptable tolerability: up to 80 mg)
Four 20 mg tablets QD (Potential post-randomization dose escalation based on efficacy and acceptable tolerability: up to 120 mg)
Crinetics Study Site
Los Angeles, California, United States
Crinetics Study Site
Los Angeles, California, United States
Crinetics Study Site
Newport Beach, California, United States
Crinetics Study Site
Stanford, California, United States
Crinetics Study Site
Miami, Florida, United States
Safety - Incidence of Treatment-emergent Adverse Events (TEAEs)
Incidence of TEAEs, including serious TEAEs and TEAEs leading to discontinuation; change from baseline to the EOR in safety parameters: clinical laboratory tests, physical exam findings, vital signs, 12-lead ECG, and 24-hour continuous cardiac monitoring (only for subjects on 120 mg dose). In addition to all-cause mortality, deaths were categorized by primary cause (e.g., cardiovascular) based on medical adjudication. Events were coded using MedDRA and summarized by treatment group, system organ class, and preferred term. TEAEs were reported per randomized arm. There were 2 randomized arms (40 mg and 80 mg paltusotine, with up-or down-dose titration permitted for symptom control). Results are analyzed based on the initially assigned randomization arm, regardless of the actual dose received.
Time frame: First dose of investigational medicine to End of Randomized Treatment Phase (8 weeks)
Pharmacokinetics (PK) of Paltusotine
Steady state trough levels by dose and visit for Randomized Treatment Phase (RTP). The "Overall Number of Participants Analyzed" displayed at the top of the PK summary table represents the total number of participants who received each dose at any point during the trial, factoring in dose titrations. PK results are summarized by actual dose taken right before the time of sample collection, rather than by randomized dose. Participants may be included in different dose groups for different visits due to dose titration.
Time frame: Measured at each visit (pre and post dose) up to Week 8 (i.e., End of Randomized Treatment Phase [EOR])
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Crinetics Study Site
Iowa City, Iowa, United States
Crinetics Study Site
Lexington, Kentucky, United States
Crinetics Study Site
New Orleans, Louisiana, United States
Crinetics Study Site
Boston, Massachusetts, United States
Crinetics Study Site
Rochester, Minnesota, United States
...and 25 more locations