Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD (SHIELD HD)
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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 |
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Recruitment Status :
Recruiting
First Posted : May 28, 2020
Last Update Posted : August 7, 2020
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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 |
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| Huntington Disease |
| 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 |
- 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.
- 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.
- 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.
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.
| Ages Eligible for Study: | 18 Years to 63 Years (Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Key Inclusion Criteria
Patients who meet all of the following criteria will be eligible to participate in the study:
- 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
Key Exclusion Criteria
Patients who meet any of the following criteria will be excluded from participation in the study:
- 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
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
| 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 |
| 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, Massachusetts | |
| Beth Israel Deaconess | Recruiting |
| Boston, Massachusetts, United States, 02215 | |
| Contact: Jacqueline Fung jfung@bidmc.harvard.edu | |
| 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 | |
| Principal Investigator: | Anne Rosser, PhD FRCP | Cardiff University |
| 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: | August 7, 2020 |
| Last Verified: | July 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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HD |
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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 |