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

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

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.

Study Overview

• Status: Unknown
• Conditions: Huntington Disease
• Intervention / Treatment: Other: CAPIT-HD beta

• Study Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Anne-Catherine BACHOUD-LEVI, MD, PhD; Phone Number: +33 (0)1.49.81.43.01; Email: anne-catherine.bachoud-levi@aphp.fr

Study Locations

• France
  • Creteil, France, 94010
    • Recruiting
    • Henri Mondor Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: HD patients	Other: CAPIT-HD beta; New assessment protocol for assessment of complex therapies in Huntington's disease for both groups
EXPERIMENTAL: Controls	Other: CAPIT-HD beta; New assessment protocol for assessment of complex therapies in Huntington's disease for both groups

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
Number of new assessment battery performed correctly		1 year

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Institut National de la Santé Et de la Recherche Médicale, France; University Hospital of Wales; School of Biosciences - Cardiff University; National Reference Center for Huntington's disease Cognitive Neurology Unit; Manchester Centre for Genomic Medicine - St. Mary's Hospital University of Manchester; George-Huntington-Institut GmbH
• Investigators: Principal Investigator: Anne-Catherine BACHOUD-LEVI, MD, PhD, Assistance Publique - Hôpitaux de Paris; Principal Investigator: Anne ROSSER, MD, PhD, School of Biosciences - Cardiff University; Principal Investigator: David CRAUFURD, MD, PhD, Manchester Centre for Genomic Medicine - St. Mary's Hospital; Principal Investigator: Ralf REILMANN, MD, PhD, George-Huntington-Institut GmbH

Study record dates

Study Major Dates

• Study Start: June 1, 2016
• Primary Completion (ANTICIPATED): March 1, 2018
• Study Completion (ANTICIPATED): March 1, 2018

Study Registration Dates

• First Submitted: August 25, 2016
• First Submitted That Met QC Criteria: April 13, 2017
• First Posted (ACTUAL): April 18, 2017

Study Record Updates

• Last Update Posted (ACTUAL): April 18, 2017
• Last Update Submitted That Met QC Criteria: April 13, 2017
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Keywords: HD; beta testing; assessment battery
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease
• Other Study ID Numbers: P150201