PATHFNDR-2
Research type
Research Study
Full title
A Randomized, Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Paltusotine in Subjects with Non-pharmacologically Treated Acromegaly
IRAS ID
1004169
Contact name
William Drake
Contact email
Sponsor organisation
Crinetics Pharmaceuticals, Inc.
Eudract number
2021-001703-32
Clinicaltrials.gov Identifier
Research summary
This is a multicenter, multinational study which will recruit approximately 76 patients with acromegaly. This study will investigate the safety and efficacy of paltusotine, taken by mouth, by patients who have never been treated medically for their acromegaly, have previously been treated or are currently being treated and are willing to come off their treatment to take part in this study.
These study participants will enter a screening phase of up to 16 weeks (depending on whether they have been medically treated for their acromegaly or not), consisting of up to 3 study visits. If a participant is eligible to take part after this screening period, they will be enrolled at random (via an interactive web-based system) with a 50/50 chance to receive either paltusotine or placebo. This is the treatment phase with about 6 study visits (and 4 at home or another suitable location) and lasting approximately 24 weeks.
At the end of the randomised treatment phase, if the Investigator thinks the participant will benefit from treatment with paltustotine, the participant may roll over into a 96 week treatment period with paltusotine, attending an additional 8 study visits (and 3 home visits). Everyone will receive paltusotine in this open-label extension phase.
Participants will undergo investigations such as an MRI scan of the pituitary, ultrasound of the gallbladder, and ECGs.
Blood will be sampled to determine safety laboratory results, growth hormone levels, pharmacokinetic information (what is happening to the study drug in your body) and if consented to, a genotype blood sample (participants' genetic material determines how they will respond to the treatment).
Some blood samples for growth hormone determination, will be performed under fasting conditions.
Individual participation in the study can last up to 3.0 years if the study, including the extension phase, is completed. The study will be conducted at about 5 NHS sites in the UK.REC name
London - Harrow Research Ethics Committee
REC reference
22/LO/0192
Date of REC Opinion
6 May 2022
REC opinion
Further Information Favourable Opinion