Parkinson's DiseasePhase 1/2NCT01856439
Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease
A Phase 1/2 study testing ProSavin in people with Parkinson's Disease, run by Axovant Sciences Ltd. at 2 sites in 2 countries.
Trial summary
The study is designed to assess the long term tolerability of ProSavin and whether it is safe and efficacious in patients administered ProSavin from the PS1/001/07 study . Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- ProSavin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- May 2011to Apr 2022
- Phase
- Phase 1/2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- must have met all inclusion criteria for study PS1/001/07
- willing to have L-DOPA dosage reduced/withdrawn at the discretion of the investigator at regular intervals to allow assessment of ProSavin in the absence of concomitant antiparkinsonian medication.
- affiliated with the French social security health care system (Patients enrolled in France only)
- signed and dated written informed consent obtained from the patient and/or the patient's legally acceptable representative, if applicable, in accordance with the local regulations
- must have been treated with ProSavin/Sham
Who cannot join
- -
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Multicentre, Open-label Study to Determine the Long Term Safety, Tolerability and Efficacy of ProSavin in Patients With Bilateral, Idiopathic Parkinson's Disease.
The study is designed to assess the long term tolerability of ProSavin and whether it is safe and efficacious in patients administered ProSavin from the PS1/001/07 study .
- Conditions
- Parkinson's Disease
- Being tested
- ProSavin
- Keywords
- ProSavin, Gene therapy
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProSavinDrug
Long term follow up of patients who received ProSavin in a previous study
| Study group | Role | What they receive |
|---|---|---|
| Long term follow up | Other | Long term follow up of patient's who received ProSavin in previous study Receives: Drug: ProSavin |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the long term safety and tolerability of ProSavin
The number and severity of any adverse event
Measured: 10 years
Secondary outcomes (1)
Patients' responses to ProSavin administration
Assessed using Unified Parkinson's Disease Rating Score(UPDRS) Part III, evaluating percentage of time during waking day that patient is in the "off" state through patient diaries and activities of daily living using PDQ-39 at 36 months post administration.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2013 (estimated)
- Study startMay 2011
- Primary completionNov 2021
- Study completionApr 2022
Why it stopped: As a result of termination of development of Prosavin, it is no longer possible for Sio to either commence or to continue any related clinical trials.
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 Parkinson's DiseaseStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Axovant Sciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Oxford BioMedica
- Responsible party
- Sponsor
- Investigators
- Stephane Palfi, Professor, Principal investigator, Henri Mondor University HospitalRoger Barker, Dr, Principal investigator, Cambridge University Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01856439
- Sponsor's protocol ID
- PS1/001/09
- Other IDs
- 2009-017253-35, 2009
- Status last verified
- Apr 2022
- First submitted
- 14 May 2013
- Last updated
- 3 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, Watts C, Miskin J, Kelleher M, Deeley S, Iwamuro H, Lefaucheur JP, Thiriez C, Fenelon G, Lucas C, Brugieres P, Gabriel I, Abhay K, Drouot X, Tani N, Kas A, Ghaleh B, Le Corvoisier P, Dolphin P, Breen DP, Mason S, Guzman NV, Mazarakis ND, Radcliffe PA, Harrop R, Kingsman SM, Rascol O, Naylor S, Barker RA, Hantraye P, Remy P, Cesaro P, Mitrophanous KA. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet. 2014 Mar 29;383(9923):1138-46. doi: 10.1016/S0140-6736(13)61939-X. Epub 2014 Jan 10.
PubMed 24412048
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicentre, Open-label Study to Determine the Long Term Safety, Tolerability and Efficacy of ProSavin in Patients With Bilateral, Idiopathic Parkinson's Disease.
- ClinicalTrials.gov ID
- NCT01856439
- Acronym / sponsor ID
- PS1/001/09
- Conditions
- Parkinson's Disease
- Keywords
- ProSavin, Gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: As a result of termination of development of Prosavin, it is no longer possible for Sio to either commence or to continue any related clinical trials.
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Axovant Sciences Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Oxford BioMedica
- Study start
- May 2011
- Primary completion
- Nov 2021 (actual)
- Study completion
- Apr 2022 (actual)
- First posted
- 17 May 2013 (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 study is designed to assess the long term tolerability of ProSavin and whether it is safe and efficacious in patients administered ProSavin from the PS1/001/07 study .
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Long term follow up | Other | Drug: ProSavinLong term follow up of patient's who received ProSavin in previous study |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- must have met all inclusion criteria for study PS1/001/07
- willing to have L-DOPA dosage reduced/withdrawn at the discretion of the investigator at regular intervals to allow assessment of ProSavin in the absence of concomitant antiparkinsonian medication.
- affiliated with the French social security health care system (Patients enrolled in France only)
- signed and dated written informed consent obtained from the patient and/or the patient's legally acceptable representative, if applicable, in accordance with the local regulations
- must have been treated with ProSavin/Sham
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| ProSavinLong term follow up of patients who received ProSavin in a previous study | Drug | Long term follow up |
Primary outcome
To assess the long term safety and tolerability of ProSavin
Time frame: 10 years
The number and severity of any adverse event
1 secondary outcome
Patients' responses to ProSavin administration
Time frame: 10 years
Results and safety
The study was stopped early (reason given: As a result of termination of development of Prosavin, it is no longer possible for Sio to either commence or to continue any related clinical trials.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2022.
- Derived from this study: PubMed 24412048Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, Watts C, Miskin J, Kelleher M, Deeley S, Iwamuro H, Lefaucheur JP, Thiriez C, Fenelon G, Lucas C, Brugieres P, Gabriel I, Abhay K, Drouot X, Tani N, Kas A, Ghaleh B, Le Corvoisier P, Dolphin P, Breen DP, Mason S, Guzman NV, Mazarakis ND, Radcliffe PA, Harrop R, Kingsman SM, Rascol O, Naylor S, Barker RA, Hantraye P, Remy P, Cesaro P, Mitrophanous KA. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet. 2014 Mar 29;383(9923):1138-46. doi: 10.1016/S0140-6736(13)61939-X. Epub 2014 Jan 10.
- Individual participant dataSharing planned: No
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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.
Stephane Palfi, Professor principal investigator
Henri Mondor University Hospital
Roger Barker, Dr principal investigator
Cambridge University Hospitals NHS Foundation Trust
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ProSavin in people with Parkinson's Disease, run by Axovant Sciences Ltd. at 2 sites in 2 countries.
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 5 inclusion and 1 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: stopped early, as of the sponsor's last update on 3 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France, United Kingdom. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: As a result of termination of development of Prosavin, it is no longer possible for Sio to either commence or to continue any related clinical trials.). 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?
To assess the long term safety and tolerability of ProSavin (time frame: 10 years). 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 NCT01856439, 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 3 May 2022.