Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
Primary Purpose
Huntington's Disease
Status
Unknown status
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
RP103
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Huntington's Disease
Eligibility Criteria
Inclusion Criteria:
- Clinically disease-registered for at least one year, leading to consult (abnormal movements, neuropsychiatric disorders, neuropsychological impairment).
- Unified Huntington's Disease Rating Scale motor ≥ 5
- Total Functional Capacity > 10 (≥ 11)
- Huntington Disease diagnosed with abnormal number of CAG repeats: 38 < nucleotide expansion (CAG)
- Age between 18 and 65
- Voluntarily Patient Consent
- Patients willing and able to take oral medications, and comply with the specific procedures of the study
Exclusion Criteria:
- Severe cognitive impairment or neuropsychiatric troubles.
- No drug compliance to previous treatment.
- Patients with contra indication to the realization of imaging studies (including claustrophobia ) .
- Patients who have not given their written and informed consent signed .
- No national health insurance affiliation
- Private patients of their liberty by judicial or administrative decision, or patients under supervision.
- Pregnant women ( pregnancy test will be carried out systematically for women at risk) or lactating .
- Women who could become pregnant during the study period and with no contraception.
- Patients who have developed hypersensitivity to cysteamine or penicillamine ( against indication of cysteamine ) .
- Brain Damage intercurrent MRI. Brain morphological abnormalities , other than those characteristic of the disease .
- Disease - associated with neurological repercussions.
- Affection - visceral serious , scalable , involving life-threatening.
- Mental - disorder may disrupt accession to the Protocol , including a history of spontaneous and / or drug-induced hallucinations history of severe depression that required repeated hospitalizations , history of repeated suicide attempts .
- Participation in progress, or interrupted for less than three months, a therapeutic protocol of Huntington's disease .
- Patients with a history of surgical interventions to improve the symptoms of Huntington 's disease such as graft neuron, deep brain stimulation, infusion of neurotrophic agent
Sites / Locations
- CHU Angers
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
RP103
placebo
Arm Description
RP103 capsule, 16 capsules per day
placebo capsule, 16 capsules per day
Outcomes
Primary Outcome Measures
Unified Huntington's Disease Rating Scale motor
Secondary Outcome Measures
Full Information
NCT ID
NCT02101957
First Posted
March 25, 2014
Last Updated
April 1, 2014
Sponsor
University Hospital, Angers
1. Study Identification
Unique Protocol Identification Number
NCT02101957
Brief Title
Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2013
Overall Recruitment Status
Unknown status
Study Start Date
October 2010 (undefined)
Primary Completion Date
July 2015 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Angers
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to evaluate the effect of cysteamine in patients with symptomatic Huntington's disease by comparing two groups of patients (cysteamine vs placebo) on the results of the Unified Huntington's Disease Rating Scale (UHDRS, Huntington study group 1996).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington's Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
96 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RP103
Arm Type
Experimental
Arm Description
RP103 capsule, 16 capsules per day
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
placebo capsule, 16 capsules per day
Intervention Type
Drug
Intervention Name(s)
RP103
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Unified Huntington's Disease Rating Scale motor
Time Frame
at 18 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Clinically disease-registered for at least one year, leading to consult (abnormal movements, neuropsychiatric disorders, neuropsychological impairment).
Unified Huntington's Disease Rating Scale motor ≥ 5
Total Functional Capacity > 10 (≥ 11)
Huntington Disease diagnosed with abnormal number of CAG repeats: 38 < nucleotide expansion (CAG)
Age between 18 and 65
Voluntarily Patient Consent
Patients willing and able to take oral medications, and comply with the specific procedures of the study
Exclusion Criteria:
Severe cognitive impairment or neuropsychiatric troubles.
No drug compliance to previous treatment.
Patients with contra indication to the realization of imaging studies (including claustrophobia ) .
Patients who have not given their written and informed consent signed .
No national health insurance affiliation
Private patients of their liberty by judicial or administrative decision, or patients under supervision.
Pregnant women ( pregnancy test will be carried out systematically for women at risk) or lactating .
Women who could become pregnant during the study period and with no contraception.
Patients who have developed hypersensitivity to cysteamine or penicillamine ( against indication of cysteamine ) .
Brain Damage intercurrent MRI. Brain morphological abnormalities , other than those characteristic of the disease .
Disease - associated with neurological repercussions.
Affection - visceral serious , scalable , involving life-threatening.
Mental - disorder may disrupt accession to the Protocol , including a history of spontaneous and / or drug-induced hallucinations history of severe depression that required repeated hospitalizations , history of repeated suicide attempts .
Participation in progress, or interrupted for less than three months, a therapeutic protocol of Huntington's disease .
Patients with a history of surgical interventions to improve the symptoms of Huntington 's disease such as graft neuron, deep brain stimulation, infusion of neurotrophic agent
Facility Information:
Facility Name
CHU Angers
City
Angers
ZIP/Postal Code
49000
Country
France
12. IPD Sharing Statement
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Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
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