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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT02101957
• Date of registration: 25/03/2014
• Prospective Registration: No
• Primary sponsor: University Hospital, Angers
• Public title: Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
• Date of first enrolment: October 2010
• Target sample size: 96
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT02101957
• Study type: Interventional
• Study design: Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Huntington's Disease

Intervention(s)

Drug: Placebo

Drug: RP103

Primary Outcome(s)

Unified Huntington's Disease Rating Scale motor [Time Frame: at 18 months]

Secondary ID(s)

PHRC2004-03bis

Source(s) of Monetary Support

Please refer to primary and secondary sponsors