PRECISION-HD1

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

Recruiting

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


UPDATES

WAVE Life Sciences expects to report data from both its ongoing PRECISION-HD phase 1b/2a trials, and available data from the related OLE studies this spring. The analysis will include biomarker and safety data from all dosing cohorts up to and including the 32mg cohort for PRECISION-HD2 and completed dosing cohorts for PRECISION-HD1 (including all participants from the 16mg cohort). These data are expected to support a decision regarding a potential phase 3 study.

Participants from the phase 1b/2a trials have been enrolling into the OLE studies since 2020, and the vast majority of eligible patients have been enrolled. In December 2020, WAVE submitted a clinical trial application for WVE-003 (SNP3). This is WAVE’s first HD candidate to use its novel PN backbone chemistry modifications which, according to preclinical data, increase the potency, exposure and durability of a compound. WVE-003 is also designed to selectively target the mutant huntingtin mRNA transcript while leaving the wild-type protein relatively intact.

The protocol was also submitted to EHDN, which endorsed it in December 2020. WAVE expects to start dosing HD participants with SNP3 this year, once regulatory and ethical approvals are in place.

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