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

Huntington Disease Clinical Trial

— SHIELD HD  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04406636
• Other study ID #: TTX N1
• Status: Active, not recruiting
• Start date: May 19, 2020
• Est. completion date: April 2023

Study information

• Verified date: September 2022
• Source: CHDI Foundation, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 70
• Est. completion date: April 2023
• Est. primary completion date: April 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 63 Years

Eligibility

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

Related Conditions & MeSH terms

• Huntington Disease

Locations

Country	Name	City	State
Canada	Centre for Movement Disorders	Toronto	Ontario
Canada	North York General Hospital	Toronto	Ontario
France	ICM - Institut du Cerveau et de la Moelle épinière	Paris
Germany	George-Huntington-Institut (GHI)	Muenster
United Kingdom	University College London - Institute of Neurology & The National Hospital for Neurology and Neurosurgery	London
United States	Beth Israel Deaconess	Boston	Massachusetts
United States	Rocky Mountain Movement Disorders Center	Englewood	Colorado
United States	Columbia University	New York	New York
United States	University of California, San Diego (UCSD)	San Diego	California
United States	Inland Northwest Research	Spokane	Washington

Sponsors (2)

Lead Sponsor	Collaborator
CHDI Foundation, Inc.	Medpace, Inc.

Countries where clinical trial is conducted

United States,  Canada,  France,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Additional biomarkers to be examined	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.	2 years
Primary	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.	2 years
Secondary	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.	2 years