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

NCT01856439 | Terminated Phase 1 DMC

• 2 other identifiers: PS1/001/092009-017253-35
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 2

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 .

Conditions

Parkinson's Disease

Keywords

ProSavin; Gene therapy

Outcome Measures

Primary Outcomes (1)

• To assess the long term safety and tolerability of ProSavin

  The number and severity of any adverse event

  10 years

Secondary Outcomes (1)

• Patients' responses to ProSavin administration

  10 years

Study Arms (1)

Long term follow up

OTHER

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

Drug: ProSavin

Interventions

ProSavin DRUG

Long term follow up of patients who received ProSavin in a previous study

Long term follow up

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• willing to have L-DOPA dosage reduced/withdrawn at the discretion of the investigator at regular intervals to allow assessment of ProSavin in the absence of concomitant antiparkinsonian medication.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

Sponsors & Collaborators

• Axovant Sciences Ltd.: lead
• Oxford BioMedica: collaborator

Study Sites (2)

Henri Mondor Hospital

Paris, France

Location

Addenbrookes Hospital

Cambridge, United Kingdom

Location

Related Publications (1)

• 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 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Stephane Palfi, Professor

  Henri Mondor University Hospital

  PRINCIPAL INVESTIGATOR

• Roger Barker, Dr

  Cambridge University Hospitals NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2013
• First Posted: May 17, 2013
• Study Start: May 1, 2011
• Primary Completion: November 1, 2021
• Study Completion: April 1, 2022
• Last Updated: May 3, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); GB (1)