Beta Testing of a New Assessment in Huntington's Disease (HD)

Huntington Disease Clinical Trial

— CAPIT-HD Beta  

Official title:

Beta Testing of a New Assessment Protocol for Assessment of Complex Therapies in Huntington's Disease (HD)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03119246
• Other study ID #: P150201
• Status: Recruiting
• Phase: N/A
• First received: August 25, 2016
• Last updated: April 13, 2017
• Start date: June 2016
• Est. completion date: March 2018

Study information

• Verified date: August 2016
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Anne-Catherine BACHOUD-LEVI, MD, PhD
• Phone: (0)1.49.81.43.01
• Email: anne-catherine.bachoud-levi[@]aphp.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Huntington's disease (HD) is an inherited neurodegenerative disease for which there are no existing disease-modifying treatments.

Repair-HD is an EU FP7 consortium that aims to establish all the preclinical requirements for transplantation of stem cell-derived neurons in HD in order to replace those lost to the disease process. These requirements include the generation of new clinical assessments for detailed monitoring of patients with HD who have undergone cell replacement therapy.

This protocol describes the beta testing of a new clinical assessment battery: Core Assessment Protocol for Intrastriatal Transplantation in HD version 2 (CAPIT-HD beta / CAPIT-HD2). CAPIT-HD beta represents a substantial revision of a previous CAPIT-HD battery published over 20 years ago, which is in need of updating in order to accommodate knowledge from clinical transplant studies over this time and to take advantage of technological advances in patient assessment.

HD is a complex disorder in which there is relentless deterioration of motor, cognitive and behavioural functions, usually from mid-life onwards. The original CAPIT battery aimed to capture elements of change in all three domains, but was based predominantly on subjective semi-quantitative assessment tools that have poor inter-rater reliability. Moreover, a number of deficits, such as impairments in social cognition, were not recognised when the original CAPIT-HD battery was constructed, so we have developed novel assessments of these deficits, some of which are included in CAPIT-HD beta. The beta testing will take place in established HD clinical centres in Cardiff, Manchester, Paris, and Munster by teams of researchers who are experienced in leading clinic research in HD. Patients with early to moderate HD will be assessed at baseline, and at one and twelve months later, to assess the reliability and sensitivity of the CAPIT-HD beta battery. Arrangements for data storage and analysis are in place.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: March 2018
• Est. primary completion date: March 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

1. Patients Inclusion criteria

• Must be confirmed to carry the HD gene through genetic testing (CAG = 36)

• Must be 18 years or above

• Stage I or II disease (TFC staging)

Exclusion criteria

• The inability to approve consent

• Any comorbid condition that has the potential to confound the results of the study

2. Controls Inclusion criteria - Must be 18 years or above

Exclusion criteria

• The inability to approve consent

• Any comorbid condition that has the potential to confound the results of the study

Related Conditions & MeSH terms

• Huntington Disease

Intervention

Other:
• CAPIT-HD beta
New assessment protocol for assessment of complex therapies in Huntington's disease for both groups

Locations

Country	Name	City	State
France	Henri Mondor Hospital	Creteil

Sponsors (7)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	George Huntington Institute - Muenster, Institut National de la Santé Et de la Recherche Médicale, France, Manchester Centre for Genomic Medicine - St. Mary’s Hospital University of Manchester, National Reference Center for Huntington's disease Cognitive Neurology Unit, School of Biosciences - Cardiff University, University Hospital of Wales

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	All dysfunction or disorder of huntington patient measured by means of a new battery assessments	The goal is to validate a battery of assessments for application in a wide range of complex therapies for Huntington's disease (HD)	1 year
Secondary	Difference on motor score between patient and healthy volontary measured by motor tests of the revised Core Assessment Protocol	The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the motor	1 year
Secondary	Difference on cognitive score between patient and healthy volontary measured by cognitive tests of the revised Core Assessment Protocol	The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the cognitive	1 year
Secondary	Difference on psychiatric score between patient and healthy volontary measured by psychiatric evaluation of the revised Core Assessment Protocol	The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the psychiatric	1 year
Secondary	Difference on functional scale between patient and healthy volontary measured by functional evaluation of the revised Core Assessment Protocol	The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the functional domains of impairment in HD	1 year
Secondary	Number of new assessment battery performed correctly		1 year