Huntington's DiseasePhase 2/3NCT02101957
Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
A Phase 2/3 study testing RP103 in people with Huntington's Disease, run by University Hospital, Angers at 1 site in France.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- RP103
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2010to Jul 2015
- Phase
- Phase 2/3
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
- Being tested
- RP103, Placebo
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RP103Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| RP103 | Experimental | RP103 capsule, 16 capsules per day Receives: Drug: RP103 |
| placebo | Placebo comparator | placebo capsule, 16 capsules per day Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Unified Huntington's Disease Rating Scale motor
Measured: at 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2014 (estimated)
- Study startOct 2010
- Primary completionJul 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
- More trials run by University Hospital, AngersThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Angers
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02101957
- Sponsor's protocol ID
- PHRC2004-03bis
- Status last verified
- Jul 2013
- First submitted
- 25 Mar 2014
- Last updated
- 2 Apr 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
- ClinicalTrials.gov ID
- NCT02101957
- Acronym / sponsor ID
- PHRC2004-03bis
- Conditions
- Huntington's Disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 96 participants (actual)
- Lead sponsor
- University Hospital, AngersOther government
- Study start
- Oct 2010
- Primary completion
- Jul 2015 (estimated)
- First posted
- 2 Apr 2014 (estimated)
- 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.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RP103 | Experimental | Drug: RP103RP103 capsule, 16 capsules per day |
| placebo | Placebo comparator | Drug: Placeboplacebo capsule, 16 capsules per day |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (15 listed)
- 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
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 |
|---|---|---|
| RP103 | Drug | RP103 |
| Placebo | Drug | placebo |
Primary outcome
Unified Huntington's Disease Rating Scale motor
Time frame: at 18 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing RP103 in people with Huntington's Disease, run by University Hospital, Angers 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 7 inclusion and 15 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 2 Apr 2014. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Unified Huntington's Disease Rating Scale motor (time frame: at 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Apr 2014.