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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04406636
Recruitment Status : Recruiting
First Posted : May 28, 2020
Last Update Posted : July 27, 2020
Sponsor:
Collaborator:
Medpace, Inc.
Information provided by (Responsible Party):
Triplet Therapeutics, Inc.

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.


Condition or disease
Huntington Disease

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.

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
Actual Study Start Date : May 19, 2020
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : December 2022





Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Other Outcome Measures:
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 63 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Huntington disease (HD), genetically confirmed by direct DNA testing, either obtained previously or performed at Screening
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04406636


Contacts
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Contact: Irina A Antonijevic, MD PhD (781) 299-7830 ext 109 TripletTrials@triplettx.com
Contact: John Cavanaugh, MPH (781) 299-7830 ext 129 TripletTrials@triplettx.com

Locations
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United States, California
University of California, San Diego (UCSD) Recruiting
San Diego, California, United States, 920161
Contact: Chase Snell       cmsnell@ucsd.edu   
United States, Colorado
Rocky Mountain Movement Disorders Center Recruiting
Englewood, Colorado, United States, 80113
Contact: Kate Greenly       kgreenly@kumarneuro.com   
United States, Washington
Inland Northwest Research Recruiting
Spokane, Washington, United States, 99202
Contact: Melissa Bixby       mbixby@inwresearch.com   
Canada, Ontario
Centre for Movement Disorders Recruiting
Toronto, Ontario, Canada, M3B 257
Contact: Libby Kalman       lkalman@movementdisorders.ca   
Sponsors and Collaborators
Triplet Therapeutics, Inc.
Medpace, Inc.
Investigators
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Principal Investigator: Anne Rosser, PhD FRCP Cardiff University
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Responsible Party: Triplet Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04406636    
Other Study ID Numbers: TTX N1
First Posted: May 28, 2020    Key Record Dates
Last Update Posted: July 27, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Triplet Therapeutics, Inc.:
HD
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders