Histone Deacetylase Inhibitor FRM-0334 vs placebo in Dementia (V 1.0)

• Research type

  Research Study

• Full title

  A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 2a Safety, Tolerability, and Pharmacodynamic Study of Two Doses of an Histone Deacetylase Inhibitor (FRM-0334) in Subjects with Prodromal to Moderate Frontotemporal Dementia with Granulin Mutation

• IRAS ID

  160958

• Contact name

  Catherine Mummery

• Contact email

  cath.mummery@nhs.net

• Sponsor organisation

  FORUM Pharmaceuticals Inc.

• Eudract number

  2014-001489-85

• Clinicaltrials.gov Identifier

  NCT02149160

• Duration of Study in the UK

  0 years, 12 months, 0 days

• Research summary

  This is a randomized, doubleblind, placebo-controlled,dose escalating,Phase 2a study to evaluate the safety, tolerability, pharmacodynamic (PD) and pharmacokinetic (PK) effects of the investigational product, FRM-0334 against placebo for 28 days in subjects with prodromal to moderate Frontotemporal Dementia with Granulin Mutation (FTDGRN).

  The primary objectives are to
  • evaluate the safety and tolerability of 2 doses of FRM-0334 over 28days
  • Assess the pharmacodynamic effects of FRM-0334 on the change from baseline in plasma concentrations of
  progranulin (PGRN) after 28 days

  Once Group1 subjects have randomized and completed doubleblind treatment, safety, tolerability, and available pharmacokinetic data will be reviewed in blinded fashion. Randomization of subjects into Group2 will commence after day28, double blind safety, tolerability, and available pharmacokinetic data for Group 1 subjects are deemed acceptable by the Medical Monitor and Sponsor. Blinded safety, tolerability, and PK data for the 500 mg cohort will be reviewed continuously. Enrolment will be competitive, and recruitment will be terminated after randomization of 30 subjects.

  FRM-0334 will be provided to subjects once daily oral dosing of 300 or 500mg FRM0334 a white, opaque, size No.1, hard gelatin capsule for 28days, supplied as a 100mg capsule that will be size and
  color matched to the placebo capsule.

  Subjects will ingest drug (3 capsules daily for Group 1 and 5 capsules daily for Group 2) with food and water in the morning around the same time each day.

  Placebo will be provided to subjects via a once daily oral dosing regimen for 28 days, supplied as an identical capsule
  in appearance to the FRM-0334 capsule. Subjects will ingest drug (3 capsules daily for Group 1 and 5 capsules daily for Group 2) with food and water in the morning around the same time each day.

  The expected duration of the study for each subject is about 88 days.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  14/LO/2222

• Date of REC Opinion

  23 Feb 2015

• REC opinion

  Further Information Favourable Opinion