A Study to Evaluate ALN-HTT02 in Adult Patients with Huntington’s Disease

• Research type

  Research Study

• Full title

  A Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered Single Ascending Doses of ALN-HTT02 in Adult Patients with Huntington’s Disease

• IRAS ID

  1010230

• Contact name

  Satinder Chettra

• Contact email

  schettra@alnylam.com

• Sponsor organisation

  Alnylam UK Limited

• Clinicaltrials.gov Identifier

  NCT06585449

• Research summary

  This study is investigating the safety, tolerability and effects of a new drug called ALN-HTT02 in participants with Huntington's Disease. The study is being conducted to understand if ALN-HTT02 is safe and well tolerated in people with Huntington's Disease and collect initial data on how ALN-HTT02 affects the body.
  Between 36 and 102 study participants will participate worldwide.

  Potential participants may be able to take part if they:
  - Aged between 25 to 70 years inclusive
  - Have early stage Huntington's Disease

  There are two parts to this study, a double-blind part and an open-label (OL) part for placebo treated patients only.
  Part 1: Double-blind part includes 5 planned cohorts. Cohort 1: 6 participants with 4 participants receiving a single dose of ALN-HTT02 and 2 participants receiving placebo, Cohort 2: A maximum (max) of 12 participants with 8 participants receiving a single dose of ALN-HTT02 and 4 participants receiving placebo. Cohorts 3 through to 5: A max of 12 participants with 9 participants receiving a single dose of ALN-HTT02 and 3 participants receiving placebo. Four optional cohorts may be enrolled, each with a max of 12 participants with 9 participants receiving a single dose of ALN-HTT02 and 3 participants receiving placebo.
  Part 2: For those participants who received placebo in part 1 of the study and have completed the Month 6 End Of Treatment visit, and their cohort has been unblinded . The participant if eligible will have the option to receive a single dose of ALN-HTT02 in the 12-month Open label part of the study.
  A single intrathecal administration (injection into the fluid around spinal cord) of or placebo will be given to participants in Part 1.
  In Part 2 the participant who received placebo in part 1 will have the option to receive a single dose of ALN-HTT02 in the open label Study.
  Estimated duration of Part 1 is 14 months and total duration for Parts 1 & 2 is 26 months.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  24/LO/0538

• Date of REC Opinion

  4 Sep 2024

• REC opinion

  Further Information Favourable Opinion