Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03119246
Recruitment Status : Unknown
Verified August 2016 by Assistance Publique - Hôpitaux de Paris.
Recruitment status was:  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-Institut GmbH
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Tracking Information
First Submitted Date  ICMJE August 25, 2016
First Posted Date  ICMJE April 18, 2017
Last Update Posted Date April 18, 2017
Study Start Date  ICMJE June 2016
Estimated Primary Completion Date March 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 13, 2017)
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)
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: April 13, 2017)
  • 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
  • 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
  • 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
  • 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
  • Number of new assessment battery performed correctly [ Time Frame: 1 year ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Beta Testing of a New Assessment in Huntington's Disease (HD)
Official Title  ICMJE Beta Testing of a New Assessment Protocol for Assessment of Complex Therapies in Huntington's Disease (HD)
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Huntington Disease
Intervention  ICMJE Other: CAPIT-HD beta
New assessment protocol for assessment of complex therapies in Huntington's disease for both groups
Study Arms  ICMJE
  • Experimental: HD patients
    Intervention: Other: CAPIT-HD beta
  • Experimental: Controls
    Intervention: Other: CAPIT-HD beta
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: April 13, 2017)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2018
Estimated Primary Completion Date March 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03119246
Other Study ID Numbers  ICMJE P150201
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Assistance Publique - Hôpitaux de Paris
Study Sponsor  ICMJE Assistance Publique - Hôpitaux de Paris
Collaborators  ICMJE
  • 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-Institut GmbH
Investigators  ICMJE
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
PRS Account Assistance Publique - Hôpitaux de Paris
Verification Date August 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP