Ignite Creation Date:
2024-05-06 @ 12:00 PM
Last Modification Date:
2024-10-26 @ 12:53 PM
Study NCT ID:
NCT03652363
Status:
COMPLETED
Last Update Posted:
2018-08-29
First Post:
2018-08-21
Brief Title:
GDNF in ideopathicParkinsons Disease
Sponsor:
North Bristol NHS Trust
Organization:
North Bristol NHS Trust
Study Overview
Official Title:
A Placebo-Controlled Randomised Double-Blind Trial to Assess the Safety and Efficacy of Intermittent Bilateral Intraputamenal GDNF Infusions Administered Via Convection Enhanced Delivery CED in Subjects With ParkinsonAposs Disease
Status:
COMPLETED
Status Verified Date:
2018-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
A Placebo Controlled Randomised Trial of GDNF vs placebo
The study will require patients to undergo surgery to implant microcatheters precisely into the brain Patients will then attend clinic on a 2 weekly basis for infusions of a nerve growth factor called GDNF or placebo Specific tests will also be carried out at regular intervals to assess your symptoms All participants will undergo radio-isotope brain imaging at the beginning and end of the study Periodically patients will also be required to undergo an MRI scan to assess the delivery of the study drug or placebo
The study will require patients to undergo surgery to implant microcatheters precisely into the brain Patients will then attend clinic on a 2 weekly basis for infusions of a nerve growth factor called GDNF or placebo Specific tests will also be carried out at regular intervals to assess your symptoms All participants will undergo radio-isotope brain imaging at the beginning and end of the study Periodically patients will also be required to undergo an MRI scan to assess the delivery of the study drug or placebo
Detailed Description:
Patients who are considered likely to meet certain suitability criteria for this trial and who provide fully informed consent to participate will have screening blood tests and clinical assessments As part of these assessments patients will be asked to stop their PD medication overnight to allow us to assess the severity of their PD symptoms without the benefit of medication on board These assessments will answer if patients are eligible to enter the trial Some patients will not continue on with the trial following these assessments However we will discuss with those patients alternative clinical care research options
Patients that are felt to be eligible for the trial following these assessments will undergo brain surgery performed under general anaesthetic During the surgery 4 tiny plastic infusion tubes will be inserted into the putamen area of the brain 2 on either side of the brain two in each putamen These tubes connect to a single metal port that will come out exit from under the skin behind the ear This port can be accessed as needed from the outside and will allow GDNF or placebo infusions to be given every 4 weeks directly into the putamen part of the brain without requiring additional surgery or piercing of the skin see Figure 1
Approximately 4 weeks after surgery patients will have an infusion of placebo dummy drug and an MRI to check the tubes and port system are working well enough to allow infusions of either GDNF or placebo further details below
Patients will be required to attend NBT in order to have either GDNF or placebo infusions given through the port and down the tiny plastic tubes into the putamen every 4 weeks for a total of 9 months 10
infusions in total Neither the patient nor the research team will know whether patients are receiving GDNF or placebo dummy infusions
For some of these visits patients will again be asked to stop their PD medication the night before By doing this it will be possible to assess potential reversal of symptoms compared with before the start of brain infusion treatmentsDuring the course of this trial patients will undergo positron emission tomography PET brain scans at another hospital on two or three occasions to look for evidence of damaged brain cell recovery This trial is therefore time-consuming and this will be made clear to you before seeking your consent to participate
We hope that this trial will help clarify whether GDNF does indeed have the potential to reverse Parkinsons disease and restore parts of the brain It must be understood however that even if the trial yields positive results further testing will be required in a larger number of patients to meet the requirements of the health authorities prior to GDNF being available over the long term or routinely
Participation in this trial will involve some risks to patients These risks are described in detail later in this document and will be discussed with you before you give informed consent When the trial ends the information collected from participants will be used to produce reports for scientific journals and will be presented at scientific meetings so that doctors and scientists around the world will better understand the potential brain restoring and disease reversing effects of GDNF in PD
Patients that are felt to be eligible for the trial following these assessments will undergo brain surgery performed under general anaesthetic During the surgery 4 tiny plastic infusion tubes will be inserted into the putamen area of the brain 2 on either side of the brain two in each putamen These tubes connect to a single metal port that will come out exit from under the skin behind the ear This port can be accessed as needed from the outside and will allow GDNF or placebo infusions to be given every 4 weeks directly into the putamen part of the brain without requiring additional surgery or piercing of the skin see Figure 1
Approximately 4 weeks after surgery patients will have an infusion of placebo dummy drug and an MRI to check the tubes and port system are working well enough to allow infusions of either GDNF or placebo further details below
Patients will be required to attend NBT in order to have either GDNF or placebo infusions given through the port and down the tiny plastic tubes into the putamen every 4 weeks for a total of 9 months 10
infusions in total Neither the patient nor the research team will know whether patients are receiving GDNF or placebo dummy infusions
For some of these visits patients will again be asked to stop their PD medication the night before By doing this it will be possible to assess potential reversal of symptoms compared with before the start of brain infusion treatmentsDuring the course of this trial patients will undergo positron emission tomography PET brain scans at another hospital on two or three occasions to look for evidence of damaged brain cell recovery This trial is therefore time-consuming and this will be made clear to you before seeking your consent to participate
We hope that this trial will help clarify whether GDNF does indeed have the potential to reverse Parkinsons disease and restore parts of the brain It must be understood however that even if the trial yields positive results further testing will be required in a larger number of patients to meet the requirements of the health authorities prior to GDNF being available over the long term or routinely
Participation in this trial will involve some risks to patients These risks are described in detail later in this document and will be discussed with you before you give informed consent When the trial ends the information collected from participants will be used to produce reports for scientific journals and will be presented at scientific meetings so that doctors and scientists around the world will better understand the potential brain restoring and disease reversing effects of GDNF in PD
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None