SHIELD HD

Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs)

Active, not recruiting

ABOUT

SPONSOR

Triplet Therapeutics, Inc.

PARTICIPANTS

70

SHIELD HD is an International study that will follow a group of people over time who have or are at risk of developing Huntington´s Disease. SHIELD HD will collect health information that includes medical history, blood and cerebrospinal fluid samples. Participants will also undergo MRIs, cognitive and motor function tests. The information will be used to evaluate the natural history of HD and its biomarkers that are associated with modulation of the number of CAG (cytosine-adenine-guanine) repeats in the mutant Huntingtin(HTT) gene. The results of this study will inform assessments for a future interventional treatment trial.

Estimated Study Completion Date: December 2022

Ages Eligible for Study:

18 Years and older (Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Inclusion Criteria

  • Capacity to comprehend the study objectives and procedures
  • Documentation of genetically confirmed disease by direct DNA testing, defined as a CAG repeat length ≥39 in the HTT gene
  • Ability to undergo and tolerate MRI scans
  • Ability to tolerate blood draws and lumbar punctures

Exclusion Criteria

  • Any conditions, including severe chorea and dementia, that would prevent either writing or performing pen and paper, tablet, or computer based tasks as determined by the Investigator
  • Treatment with an investigational drug within 30 days prior to screening or within 5 half lives of the investigational drug, whichever is longer
  • History of gene therapy or cell transplantation or any other experimental brain surgery

COUNTRIES

UPDATE

This two-year natural history study of HDGECs, sponsored by Triplet Therapeutics, is collecting longitudinal information related to somatic instability and DNA damage response genes, along with established assessments of disease progression. The study has enrolled 70 participants to date, and follow-up assessments are ongoing at sites in North America and Europe. The results will inform assessments ahead of a future treatment trial.

FRANCE

TRIAL SITE:
ICM – Institut du Cerveau et de la Moelle épinière

Address: Paris, France, 75013

Active, Not Recruiting

GERMANY

TRIAL SITE:
George-Huntington-Institut 

Address: Münster, Germany, 48149.

Active, Not Recruiting

UNITED KINGDOM

TRIAL SITE:
University College London – Institute of Neurology & The National Hospital for Neurology and Neurosurgery

Address: London, United Kingdom

Active, Not Recruiting