NCT03119246

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
40

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Jun 2016

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2016

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

August 25, 2016

Completed
8 months until next milestone

First Posted

Study publicly available on registry

April 18, 2017

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2018

Completed
Last Updated

April 18, 2017

Status Verified

August 1, 2016

Enrollment Period

1.7 years

First QC Date

August 25, 2016

Last Update Submit

April 13, 2017

Conditions

Huntington Disease

Keywords

beta testingassessment batteryHD

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (5)

  • Difference on motor score between patient and healthy volontary measured by motor tests of the revised Core Assessment Protocol

    1 year

  • Difference on cognitive score between patient and healthy volontary measured by cognitive tests of the revised Core Assessment Protocol

    1 year

  • Difference on psychiatric score between patient and healthy volontary measured by psychiatric evaluation of the revised Core Assessment Protocol

    1 year

  • Difference on functional scale between patient and healthy volontary measured by functional evaluation of the revised Core Assessment Protocol

    1 year

  • Number of new assessment battery performed correctly

    1 year

Study Arms (2)

HD patients

EXPERIMENTAL
Other: CAPIT-HD beta

Controls

EXPERIMENTAL
Other: CAPIT-HD beta

Interventions

CAPIT-HD betaOTHER

New assessment protocol for assessment of complex therapies in Huntington's disease for both groups

ControlsHD patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

You may not qualify if:

  • The inability to approve consent
  • Any comorbid condition that has the potential to confound the results of the study
  • The inability to approve consent
  • Any comorbid condition that has the potential to confound the results of the study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Henri Mondor Hospital

Créteil, 94010, France

RECRUITING

Related Publications (1)

  • Chenain L, Riad R, Fraisse N, Jubin C, Morgado G, Youssov K, Lunven M, Bachoud-Levi AC. Graph methods to infer spatial disturbances: Application to Huntington's Disease's speech. Cortex. 2024 Jul;176:144-160. doi: 10.1016/j.cortex.2024.04.014. Epub 2024 May 17.

    PMID: 38795650DERIVED

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Anne-Catherine BACHOUD-LEVI, MD, PhD

CONTACT

(0)1.49.81.43.01anne-catherine.bachoud-levi@aphp.fr

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 25, 2016

First Posted

April 18, 2017

Study Start

June 1, 2016

Primary Completion

March 1, 2018

Study Completion

March 1, 2018

Last Updated

April 18, 2017

Record last verified: 2016-08

Locations

FR(1)