search
Back to results

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

Primary Purpose

Parkinson's Disease

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
ProSavin
Sponsored by
Axovant Sciences Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Parkinson's Disease focused on measuring ProSavin, Gene therapy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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  • willing to have L-DOPA dosage reduced/withdrawn at the discretion of the investigator at regular intervals to allow assessment of ProSavin in the absence of concomitant antiparkinsonian medication.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

-

Sites / Locations

  • Henri Mondor Hospital
  • Addenbrookes Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Long term follow up

Arm Description

Long term follow up of patient's who received ProSavin in previous study

Outcomes

Primary Outcome Measures

To assess the long term safety and tolerability of ProSavin
The number and severity of any adverse event

Secondary Outcome Measures

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.

Full Information

First Posted
May 14, 2013
Last Updated
April 27, 2022
Sponsor
Axovant Sciences Ltd.
Collaborators
Oxford BioMedica
search

1. Study Identification

Unique Protocol Identification Number
NCT01856439
Brief Title
Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease
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.
Study Type
Interventional

2. Study Status

Record Verification Date
April 2022
Overall Recruitment Status
Terminated
Why 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.
Study Start Date
May 2011 (undefined)
Primary Completion Date
November 2021 (Actual)
Study Completion Date
April 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Axovant Sciences Ltd.
Collaborators
Oxford BioMedica

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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 .

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
Keywords
ProSavin, Gene therapy

7. Study Design

Primary Purpose
Other
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Long term follow up
Arm Type
Other
Arm Description
Long term follow up of patient's who received ProSavin in previous study
Intervention Type
Drug
Intervention Name(s)
ProSavin
Intervention Description
Long term follow up of patients who received ProSavin in a previous study
Primary Outcome Measure Information:
Title
To assess the long term safety and tolerability of ProSavin
Description
The number and severity of any adverse event
Time Frame
10 years
Secondary Outcome Measure Information:
Title
Patients' responses to ProSavin administration
Description
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.
Time Frame
10 years

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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: -
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stephane Palfi, Professor
Organizational Affiliation
Henri Mondor University Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Roger Barker, Dr
Organizational Affiliation
Addenbrookes Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Henri Mondor Hospital
City
Paris
Country
France
Facility Name
Addenbrookes Hospital
City
Cambridge
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
24412048
Citation
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.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs