Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease

NCT01856439 — Phase 1 / Phase 2, Terminated, 15 participants, Axovant Sciences Ltd.

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A Multicentre, Open-label Study to Determine the Long Term Safety, Tolerability and Efficacy of ProSavin in Patients With Bilateral, Idiopathic Parkinson's Disease.

Registry	NCT01856439
Status	Terminated - 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.
Phase	Phase 1 / Phase 2
Study type	Interventional; na, single group, masking: none, other
Enrollment	15 (Actual)
Sponsor	Axovant Sciences Ltd. (Industry); collaborators: Oxford BioMedica
Conditions	Parkinson's Disease
Interventions	ProSavin (Drug)
Dates	start 2011-05-01, primary completion 2021-11-01, completion 2022-04-01, first posted 2013-05-17, last updated 2022-05-03
Locations	2 sites in France, United Kingdom

-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 .

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+Arms and interventions

Arm	Type	Interventions	Description
Long term follow up	Other	Drug: ProSavin	Long term follow up of patient's who received ProSavin in previous study

ProSavin (Drug): Long term follow up of patients who received ProSavin in a previous study

+Outcome measures

Primary outcomes

To assess the long term safety and tolerability of ProSavin [10 years]: The number and severity of any adverse event

Secondary outcomes

Patients' responses to ProSavin administration [10 years]: 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.

+Eligibility

Sex: All

Inclusion Criteria:

* 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* willing to have L-DOPA dosage reduced/withdrawn at the discretion of the investigator at regular intervals to allow assessment of ProSavin in the absence of concomitant antiparkinsonian medication.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

Exclusion Criteria:

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+Locations (2)

Facility	City	Country	Status
Henri Mondor Hospital	Paris	France
Addenbrookes Hospital	Cambridge	United Kingdom

+Publications

Results publication: 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. PMID 24412048

+Investigators

Stephane Palfi, Professor, Principal investigator, Henri Mondor University Hospital
Roger Barker, Dr, Principal investigator, Cambridge University Hospitals NHS Foundation Trust

+Funding

NIH grants: 2009