Huntington DiseaseSHIELD HDNCT04406636
Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
An observational study of Huntington Disease, run by CHDI Foundation, Inc. at 10 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 63 years
- Conditions the study is for
- Huntington Disease
- How many people
- 70 participants
- Where
- 10 sitesin 5 countries
- Study dates
- 19 May 2020to Apr 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 63 years
- All sexes
- No healthy volunteers
- Adult
Study population: Huntington disease (HD), genetically confirmed by direct DNA testing, either obtained previously or performed at Screening
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
10 sites in 5 countries.
10 sites in 5 countries
United States5 sites
Beth Israel Deaconess
Boston, Massachusetts, 02215
Rocky Mountain Movement Disorders Center
Englewood, Colorado, 80113
Columbia University
New York, New York, 10032
University of California, San Diego (UCSD)
San Diego, California, 920161
Inland Northwest Research
Spokane, Washington, 99202
Canada2 sites
Centre for Movement Disorders
Toronto, Ontario, M3B 257
North York General Hospital
Toronto, Ontario
United Kingdom1 site
University College London - Institute of Neurology & The National Hospital for Neurology and Neurosurgery
London
About this study
Official title: Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
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.
Read the 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.
- Conditions
- Huntington Disease
- Keywords
- HD
- Medical subject headings
- Huntington Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
DDR gene expression
To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
Measured: 2 years
Secondary outcomes (1)
Compare rates of change in biomarkers for disease progression
To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2020
- Study start19 May 2020
- Primary completionApr 2023 (estimated)
- Study completionApr 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHDI Foundation, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Medpace, Inc.
- Responsible party
- Sponsor
- Investigators
- Anne Rosser, PhD FRCP, Principal investigator, Cardiff University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04406636
- Sponsor's protocol ID
- TTX N1
- Acronym
- SHIELD HD
- Status last verified
- Sep 2022
- First submitted
- 20 May 2020
- Last updated
- 23 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
- ClinicalTrials.gov ID
- NCT04406636
- Acronym / sponsor ID
- SHIELD HD / TTX N1
- Conditions
- Huntington Disease
- Keywords
- HD
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (actual)
- Lead sponsor
- CHDI Foundation, Inc.University, hospital or other
- Collaborators
- Medpace, Inc.
- Study start
- 19 May 2020 (actual)
- Primary completion
- Apr 2023 (estimated)
- Study completion
- Apr 2023 (estimated)
- First posted
- 28 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 63 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Huntington disease (HD), genetically confirmed by direct DNA testing, either obtained previously or performed at Screening
Key inclusion criteria (5 listed)
- 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 (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
Compare rates of change in biomarkers for disease progression
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2022.
- Individual participant dataSharing planned: Undecided
Related trials for Huntington Disease
RecruitingPhase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Huntington Disease
RecruitingPhase 3
Pridopidine Phase 3 Study in Huntington's Disease
Huntington Disease
Not yet recruiting
Creatine and Beta-Hydroxy-Beta-Methylbutyrate (HMB) Supplementation Plus Home-Based Exercise for Sarcopenia in Huntington's Disease
Huntington Disease, Sarcopenia
Recruiting
Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study
Caregivers
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne Rosser, PhD FRCP principal investigator
Cardiff University
10 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Huntington Disease, run by CHDI Foundation, Inc. at 10 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada, France, Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DDR gene expression (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2022.