PIVOT-HD

• Research type

  Research Study

• Full title

  A PHASE 2A, RANDOMIZED, PLACEBO-CONTROLLED, DOSE-RANGING STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PTC518 IN SUBJECTS WITH HUNTINGTON'S DISEASE

• IRAS ID

  1004960

• Contact name

  Madison Gray

• Contact email

  medinfo@ptcbio.com

• Sponsor organisation

  PTC Therapeutics, INC

• Eudract number

  2021-003852-18

• ISRCTN Number

  ISRCTN97180769

• Clinicaltrials.gov Identifier

  NCT05551741

• Research summary

  The safety and efficacy of three doses of PTC518 (investigational medicine), for participants with Huntington's Disease (HD), will be studied in this Clinical Trial.
  PTC518 is being studied to determine if it can slow the progression of HD and its associated symptoms by reducing the production of an abnormal version of a protein called huntingtin protein (sometimes called a mutant version or mHTT).
  Approximately 156 participants, aged 25 years and older will participate across approximately 20 sites in North America, Europe, and Australia.

  The Trial is divided into the below periods:
  - Screening period (to check eligibility to join the study)
  - Baseline Visit (Another visit to site for further assessment of participants to confirm eligibility and dosing)
  - Treatment period (3 doses of PTC518. Part A, 108 participants randomized to 1 of 2 active treatments - 5 mg or 10 mg - or placebo in a 1:1:1 randomization. Part B, 48 participants randomized in a 1:3 ratio to placebo or 20 mg. Participants in part A and part B will have a 2 in 3 and 3 in 4 chance of receiving study drug vs placebo respectively).
  - Safety follow-up period (to check participants overall health and safety).

  The study medicine comes in tablet form and is to be taken orally, in the morning, at least 2 hours before the first meal of the day (fasting).
  
  Participation in the study will last up to 24 weeks, from screening to the final follow-up visit. Study treatment period will last approximately 85 days (12 weeks). Participation is voluntary.
  Participants will be expected to attend clinic visit whereby study assessments will take place e.g., blood and urine collection, vital signs monitoring, assessments of motor function & gait, cognition & behavioral and mental health, and completion of questionnaires. If participants are unable to conduct an on-site visit, they may be allowed to have some study assessments performed at their home by the designated Home Care Services.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  22/LO/0229

• Date of REC Opinion

  3 May 2022

• REC opinion

  Further Information Favourable Opinion