Huntington DiseaseCAPIT-HD BetaNCT03119246
Beta Testing of a New Assessment in Huntington's Disease (HD)
A study testing CAPIT-HD beta in people with Huntington Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Huntington Disease
- What is being tested?
- CAPIT-HD beta
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2016to Mar 2018
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- The inability to approve consent
- Any comorbid condition that has the potential to confound the results of the study
- Controls Inclusion criteria - Must be 18 years or above
- The inability to approve consent
- Any comorbid condition that has the potential to confound the results of the study
Trial locations and contacts
1 site in 1 country.
Anne-Catherine BACHOUD-LEVI, MD, PhD contact
About this study
Official title: 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.
Read more
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.
- Conditions
- Huntington Disease
- Being tested
- CAPIT-HD beta
- Keywords
- beta testing, assessment battery, HD
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAPIT-HD betaOther
New assessment protocol for assessment of complex therapies in Huntington's disease for both groups
| Study group | Role | What they receive |
|---|---|---|
| HD patients | Experimental | Not described Receives: Other: CAPIT-HD beta |
| Controls | Experimental | Not described Receives: Other: CAPIT-HD beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 1 year
Secondary outcomes (5)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 1 year
Number of new assessment battery performed correctly
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2017
- Study startJun 2016
- Primary completionMar 2018 (estimated)
- Study completionMar 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- 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
- Responsible party
- Sponsor
- Investigators
- Anne-Catherine BACHOUD-LEVI, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de ParisAnne ROSSER, MD, PhD, Principal investigator, School of Biosciences - Cardiff UniversityDavid CRAUFURD, MD, PhD, Principal investigator, Manchester Centre for Genomic Medicine - St. Mary's HospitalRalf REILMANN, MD, PhD, Principal investigator, George-Huntington-Institut GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03119246
- Sponsor's protocol ID
- P150201
- Acronym
- CAPIT-HD Beta
- Status last verified
- Aug 2016
- First submitted
- 25 Aug 2016
- Last updated
- 18 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 38795650
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beta Testing of a New Assessment Protocol for Assessment of Complex Therapies in Huntington's Disease (HD)
- ClinicalTrials.gov ID
- NCT03119246
- Acronym / sponsor ID
- CAPIT-HD Beta / P150201
- Conditions
- Huntington Disease
- Keywords
- beta testing, assessment battery, HD
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- 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
- Study start
- Jun 2016
- Primary completion
- Mar 2018 (estimated)
- Study completion
- Mar 2018 (estimated)
- First posted
- 18 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HD patients | Experimental | Other: CAPIT-HD beta |
| Controls | Experimental | Other: CAPIT-HD beta |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (5 listed)
- Controls Inclusion criteria - Must be 18 years or above
- The inability to approve consent
- Any comorbid condition that has the potential to confound the results of the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CAPIT-HD betaNew assessment protocol for assessment of complex therapies in Huntington's disease for both groups | Other | Controls, HD patients |
Primary outcome
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)
5 secondary outcomes
Difference on motor score between patient and healthy volontary measured by motor tests of the revised Core Assessment Protocol
Time frame: 1 year
Difference on cognitive score between patient and healthy volontary measured by cognitive tests of the revised Core Assessment Protocol
Time frame: 1 year
Difference on psychiatric score between patient and healthy volontary measured by psychiatric evaluation of the revised Core Assessment Protocol
Time frame: 1 year
Difference on functional scale between patient and healthy volontary measured by functional evaluation of the revised Core Assessment Protocol
Time frame: 1 year
Number of new assessment battery performed correctly
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2017.
- Derived from this study: PubMed 38795650Chenain 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne-Catherine BACHOUD-LEVI, MD, PhD contact
Anne-Catherine BACHOUD-LEVI, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
Anne ROSSER, MD, PhD principal investigator
School of Biosciences - Cardiff University
David CRAUFURD, MD, PhD principal investigator
Manchester Centre for Genomic Medicine - St. Mary's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CAPIT-HD beta in people with Huntington Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anne-Catherine BACHOUD-LEVI, MD, PhD ((0)1.49.81.43.01 ext. +33, anne-catherine.bachoud-levi@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
All dysfunction or disorder of huntington patient measured by means of a new battery assessments (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03119246, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2017.