Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)

NCT02101957 | Unknown Phase 2

• 1 other identifier: PHRC2004-03bis
• Study Type: interventional
• Target: 96
• Locations: 1 country
• Sites: 1

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).

Conditions

Huntington's Disease

Outcome Measures

Primary Outcomes (1)

• Unified Huntington's Disease Rating Scale motor

  at 18 months

Study Arms (2)

RP103

EXPERIMENTAL

RP103 capsule, 16 capsules per day

Drug: RP103

placebo

PLACEBO COMPARATOR

placebo capsule, 16 capsules per day

Drug: Placebo

Interventions

RP103 DRUG

RP103

Placebo DRUG

placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Angers: lead

Study Sites (1)

CHU Angers

Angers, 49000, France

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2014
• First Posted: April 2, 2014
• Study Start: October 1, 2010
• Primary Completion: July 1, 2015
• Last Updated: April 2, 2014

Record last verified: 2013-07

Locations

FR (1)