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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04406636
Recruitment Status : Active, not recruiting
First Posted : May 28, 2020
Last Update Posted : October 18, 2021
Medpace, Inc.
Information provided by (Responsible Party):
Triplet Therapeutics, Inc.

Tracking Information
First Submitted Date May 20, 2020
First Posted Date May 28, 2020
Last Update Posted Date October 18, 2021
Actual Study Start Date May 19, 2020
Estimated Primary Completion Date November 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 22, 2020)
DDR gene expression [ Time Frame: 2 years ]
To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: May 22, 2020)
Compare rates of change in biomarkers for disease progression [ Time Frame: 2 years ]
To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures
 (submitted: May 22, 2020)
Additional biomarkers to be examined [ Time Frame: 2 years ]
The exploratory objectives of this study are to be determined and may include the examination of additional biomarkers present in CSF, plasma, and whole blood, including but not limited to mutant HTT (mHTT) protein, cytokines, and others, as well as clinical markers of progression.
Original Other Pre-specified Outcome Measures Same as current
Descriptive Information
Brief Title Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
Official Title Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
Brief Summary

SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history study to assess the natural history of HD and its biomarkers that are associated with modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin (HTT) gene.

Approximately 60 patients will be enrolled into the study and followed for up to 24 months at clinical sites in North America and Europe.

The results of this study will inform assessments for a future interventional treatment trial.

Detailed Description The rationale for this study is to obtain longitudinal information related to Somatic Instability and DNA damage response genes in HDGECs at various stages of the disease. Established assessments of disease progression will also be recorded.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Huntington disease (HD), genetically confirmed by direct DNA testing, either obtained previously or performed at Screening
Condition Huntington Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: November 18, 2020)
Original Estimated Enrollment
 (submitted: May 22, 2020)
Estimated Study Completion Date December 2022
Estimated Primary Completion Date November 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

Key Inclusion Criteria

Patients who meet all of the following criteria will be eligible to participate in the study:

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

Key Exclusion Criteria

Patients who meet any of the following criteria will be excluded from participation in the study:

  1. 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
  2. Treatment with an investigational drug within 30 days prior to screening or within 5 half lives of the investigational drug, whichever is longer
  3. History of gene therapy or cell transplantation or any other experimental brain surgery
Sexes Eligible for Study: All
Ages 18 Years to 63 Years   (Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   France,   Germany,   United Kingdom,   United States
Removed Location Countries  
Administrative Information
NCT Number NCT04406636
Other Study ID Numbers TTX N1
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party Triplet Therapeutics, Inc.
Study Sponsor Triplet Therapeutics, Inc.
Collaborators Medpace, Inc.
Principal Investigator: Anne Rosser, PhD FRCP Cardiff University
PRS Account Triplet Therapeutics, Inc.
Verification Date October 2021