PRECISION-HD1

SAFETY AND TOLERABILITY OF WVE-120101 IN PATIENTS WITH HUNTINGTON’S DISEASE

Recruiting


UPDATES - March 30, 2021

Wave Life Sciences shared their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.

ABOUT

SPONSOR

Wave Life Sciences Ltd.

PARTICIPANTS

60

PRECISION-HD1 is testing a new drug that selectively lowers the mutant Huntington’s disease protein, whilst leaving the normal huntingtin protein relatively untouched. In order to be able to identify the gene with the mutant Huntingtin, they use a unique method and identify a so-called SNPs. Wave has identified two different snips that helps locate the mutant Huntingtin and therefore there are two studies (Presicion HD 1 and Presicion HD 2) with the same treatment, where the only difference is the targeted SNP. The primary objective is to understand whether the drug is safe in a small number of volunteers, before testing in a larger population and collecting evidence that the drug may work. The total of 60 participants receive either placebo or different dosages of the active drug every 4 weeks. Both the active drug and the placebo is delivered through a spinal injections. 

Estimated Study Completion Date: December 2020

Ages Eligible for Study:

25 Years to 65 Years (Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Inclusion Criteria

  • Prescreened with targeted SNP on the same allele as the pathogenic CAG expansion
  • Ambulatory, male or female patients aged ≥25 – ≤65 years
  • Clinical diagnostic motor features of HD, defined as Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
  • Early manifest HD, Stage I or Stage II based on UHDRS Total Functional Capacity Scores ≥7 and ≤13

Exclusion Criteria

  • Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
  • Received investigational drug or implantable device in prior 3 months or investigational oligonucleotide in prior 6 months or 5 half-lives of the oligonucleotide, whichever is longer
  • Clinically significant medical condition, unstable psychiatric symptoms, substance abuse, or pregnancy
  • Inability to undergo brain MRI
  • Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

COUNTRIES

DENMARK

TRIAL SITE:
University Hospital of Aarhus

Address: Aarhus, Denmark, 8000

CONTACT
Anette T Moller
Tel: Not available

Recruiting

TRIAL SITE: 
Rigshospitalet

Address: Copenhagen, Denmark, DK2100

CONTACT
Lena Hjermind
Tel: Not available

Recruiting

TRIAL SITE:
Odense University Hospital and University of Southern Denmark

Address: Odense, Denmark, 5000

Active, Not Recruiting

FRANCE

TRIAL SITE:
Hospital Henri Mondor

Address: Créteil, France, 94010

CONTACT
Katia Youssov
Tel: Not available

Recruiting

TRIAL SITE: 
Institut du Cerveau et de la Moelle Epinière

Address: Paris, France, 75646

CONTACT
Alexandra Dürr
Tel: Not available

Recruiting

GERMANY

TRIAL SITE:
George-Huntington- Institut GmbH

Address: Münster, Germany, 48149.

CONTACT
Ralf Reilmann
Tel: 49 (0)251 788 788 0 (George-Huntington- Institut)

Recruiting

POLAND

TRIAL SITE:
Szpital Sw. Wojciecha

Address: Gdańsk, Poland, 80-462

CONTACT
Jaroslaw Slawek
Tel: Not available

Recruiting

TRIAL SITE: 
Instytut Psychiatrii i Neurologii

Address: Warsaw, Poland, 02-957

CONTACT
Grzegorz Witkowski
Tel: Not available

Recruiting

UNITED KINGDOM

TRIAL SITE:
Royal Devon and Exeter Hospital NHS Trust

Address: Exeter, Devon, United Kingdom, EX2 5DW

CONTACT
Timothy P Harrower
Tel: Not available

Recruiting

TRIAL SITE: 
Queen Elizabeth University Hospital – PPDS

Address: Glasgow, Glasgow City, United Kingdom, G12 0XH

CONTACT
Stuart Richie
Tel: Not available

Recruiting