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

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ClinicalTrials.gov Identifier: NCT03119246
Recruitment Status : Recruiting
First Posted : April 18, 2017
Last Update Posted : April 18, 2017
Sponsor:
Collaborators:
School of Biosciences - Cardiff University
University Hospital of Wales
National Reference Center for Huntington's disease Cognitive Neurology Unit
Institut National de la Santé Et de la Recherche Médicale, France
Manchester Centre for Genomic Medicine - St. Mary’s Hospital University of Manchester
George Huntington Institute - Muenster
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief 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.


Condition or disease Intervention/treatment Phase
Huntington Disease Other: CAPIT-HD beta Not Applicable

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Beta Testing of a New Assessment Protocol for Assessment of Complex Therapies in Huntington's Disease (HD)
Study Start Date : June 2016
Estimated Primary Completion Date : March 2018
Estimated Study Completion Date : March 2018


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



Primary Outcome Measures :
  1. All dysfunction or disorder of huntington patient measured by means of a new battery assessments [ Time Frame: 1 year ]
    The goal is to validate a battery of assessments for application in a wide range of complex therapies for Huntington's disease (HD)


Secondary Outcome Measures :
  1. Difference on motor score between patient and healthy volontary measured by motor tests of the revised Core Assessment Protocol [ Time Frame: 1 year ]
    The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the motor

  2. Difference on cognitive score between patient and healthy volontary measured by cognitive tests of the revised Core Assessment Protocol [ Time Frame: 1 year ]
    The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the cognitive

  3. Difference on psychiatric score between patient and healthy volontary measured by psychiatric evaluation of the revised Core Assessment Protocol [ Time Frame: 1 year ]
    The goal is to validate the discriminative ability of the revised Core Assessment Protocol for Intracranial transplantation in HD for the psychiatric

  4. Difference on functional scale between patient and healthy volontary measured by functional evaluation of the revised Core Assessment Protocol [ Time Frame: 1 year ]
    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

  5. Number of new assessment battery performed correctly [ Time Frame: 1 year ]


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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria
  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

Information from the National Library of Medicine

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): NCT03119246


Contacts
Contact: Anne-Catherine BACHOUD-LEVI, MD, PhD (0)1.49.81.43.01 ext +33 anne-catherine.bachoud-levi@aphp.fr

Locations
France
Henri Mondor Hospital Recruiting
Creteil, France, 94010
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
School of Biosciences - Cardiff University
University Hospital of Wales
National Reference Center for Huntington's disease Cognitive Neurology Unit
Institut National de la Santé Et de la Recherche Médicale, France
Manchester Centre for Genomic Medicine - St. Mary’s Hospital University of Manchester
George Huntington Institute - Muenster
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 Institute - Muenster

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03119246     History of Changes
Other Study ID Numbers: P150201
First Posted: April 18, 2017    Key Record Dates
Last Update Posted: April 18, 2017
Last Verified: August 2016

Keywords provided by Assistance Publique - Hôpitaux de Paris:
beta testing
assessment battery
HD

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Dementia
Chorea
Dyskinesias