Extension study to Assess the Benefit and Safety of Administering Intermittent GDNF Infusions in Parkinson's Disease (PD)

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2013-001881-40-GB
• Date of registration: 05/08/2013
• Prospective Registration: Yes
• Primary sponsor: North Bristol NHS Trust (NBT)
• Public title: Extension study to Assess the Benefit and Safety of Administering Intermittent GDNF Infusions in Parkinson's Disease (PD)
• Scientific title: An Extension Study to Assess the Safety and Efficacy of Intermittent Bilateral Intraputamenal Glial Cell Line-Derived Neurotrophic Factor (GDNF) Infusions Administered via onvection Enhanced Delivery (CED) in Subjects with Parkinson’s Disease - Intermittent Bilateral GDNF for Parkinson’s Disease
• Date of first enrolment: 16/09/2013
• Target sample size: 42
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-001881-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: yes; Other specify the comparator: All patients previously completed study 2553; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

United Kingdom

Contacts

• Name: Clinical Trials Manager Helen Lewis
• Affiliation: North Bristol NHS Trust (NBT)

• Name: Clinical Trials Manager Helen Lewis
• Affiliation: North Bristol NHS Trust (NBT)

Key inclusion & exclusion criteria

Inclusion criteria:
In order to qualify for entry into the study, subjects MUST meet all of the following criteria:
1. Enrolled and completed treatment in the Pilot or Primary Study Stages of Study 2553.
2. Females of childbearing potential must have a negative pregnancy test at study entry and be willing to use an approved (by the PI or designee) form of contraception until the end of the study.
3. Males with female partners of childbearing potential must be willing to use condoms for contraception until the end of the study.
4. Provision of informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12

Exclusion criteria:
Subjects who meet any of the following criteria will NOT be eligible for inclusion in the study:
1. Discontinued treatment early in Study 2553.
2. Had any significant (in the opinion of the PI or designee) protocol deviation in Study 2553; this includes receipt of any disallowed anti-parkinsonian treatment or any investigational treatment.
3. Presence of clinically significant (in the opinion of the PI) depression.
4. MoCA score < 24 at the final assessment in Study 2553.
5. Any new medical condition which might impair outcome measure assessments or safety measures including ability to undergo MRI scanning.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Parkinson's disease
MedDRA version: 18.1 Level: LLT Classification code 10034008 Term: Parkinson's syndrome System Organ Class: 100000004852

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: Glial Cell Line-Derived Neurotrophic Factor (GDNF)
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Liatermin
Other descriptive name: r-metHuGDNF
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 9.0-11.0

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 9 months of treatment in extension study (18 months treatment overall including treatment in study 2553)

Secondary Objective: To compare the effects of intermittent bilateral intraputamenal GDNF infusions on ON-state motor function, motor complications, & ON- & OFF-state activities of daily living after 18m treatment with the effects after 9m treatment in subjects who completed Study 2553.

• To assess the safety of intermittent bilateral intraputamenal GDNF infusions at 18m in subjects who received GDNF or placebo for 9m in Study 2553.

• To explore the effects of intermittent bilateral intraputamenal GDNF infusions on other motor & non-motor functions, quality of life assessments, & imaging endpoints at 18 months in subjects who completed Study 2553.

• To compare the results for various motor outcomes between the subjects who started GDNF early (i.e. were randomized to GDNF in Study 2553) & those who started GDNF late (i.e. were randomized to placebo in Study 2553).

• Pilot Extension: To generate long-term safety data & provide continued access to GDNF until results of Study 2553 are available

Main Objective: To compare the effects of intermittent bilateral intraputamenal GDNF infusions on OFF-state motor function after 18 months of treatment with the effects after 9 months of treatment in subjects who completed in Study 2553.

Primary end point(s): The primary endpoint of the study is the percentage change from baseline to the end of treatment in the practically defined OFF-state UPDRS motor score (part III).

Secondary Outcome(s)

Secondary end point(s): • Percentage change from baseline to the end of treatment in UPDRS motor score (part III) in the ON-state (following a levodopa challenge)
• Percentage change from baseline to the end of treatment in UPDRS ADL (part II) .
• Change from baseline to the end of treatment in PD diary ratings; i.e., total OFFtime per day, total good quality ON-time (ON without dyskinesias or ON with non-troublesome dyskinesias) and ON-time with troublesome dyskinesias

Timepoint(s) of evaluation of this end point: 9 months of treatment in extension study (18 months treatment overall including treatment in study 2553)

Pilot subjects only will be eligible for up to an additional 80 weeks of Treatment (Pilot Extension).

Secondary ID(s)

2797

Source(s) of Monetary Support

Parkinson's UK

MedGenesis Therapeutix Inc.

The Cure Parkinson's Trust

Ethics review

Status: Approved
Approval date:
Contact: