Idiopathic Parkinson Disease Clinical Trial
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 Parkinson&Apos;s Disease
NCT number | NCT03652363 |
Other study ID # | 2553 |
Secondary ID | |
Status | Completed |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | October 25, 2012 |
Est. completion date | April 1, 2016 |
Verified date | August 2018 |
Source | North Bristol NHS Trust |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 42 |
Est. completion date | April 1, 2016 |
Est. primary completion date | March 13, 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 35 Years to 75 Years |
Eligibility |
Inclusion Criteria: In order to qualify for entry into the presurgery and surgery and healing periods of the study, subjects MUST meet all of the following criteria: 1. Subjects diagnosed with idiopathic PD according to the United Kingdom (UK) Brain Bank Criteria. Bilateral findings must be present at study entry. 2. Duration of PD = 5 years. 3. Age 35-75 years. 4. Presence of motor fluctuations. Subjects must have an average of at least 2.5 hours of OFF-time per day on 3-day fluctuation diaries completed during screening. 5. Ability to reliably distinguish motor states (ON without dyskinesias, ON with non-troublesome dyskinesias, ON with troublesome dyskinesias and OFF) and accurately complete fluctuation diaries. 6. UPDRS motor score (part III) in a practically defined OFF-state between 25-45. 7. Hoehn and Yahr = stage III in the OFF-state. 8. Responsiveness to levodopa (> 40% improvement in motor UPDRS [part III] following a levodopa challenge). 9. No change in anti-parkinsonian medication for 6 weeks before screening. 10. 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. 11. Provision of informed consent. - Exclusion Criteria: Subjects who meet any of the following criteria will NOT be eligible for entry into presurgery and surgery and healing periods of the study: 1. Diagnosed with atypical parkinsonism or any known secondary parkinsonian syndrome including but not limited to medication induced, toxic, vascular, post-traumatic or post-infectious parkinsonism, progressive supranuclear palsy, multiple systems atrophy, or other neurodegenerative disorder associated with parkinsonism. 2. Signs or symptoms suggestive of atypical parkinsonian syndrome including supranuclear gaze palsy, early postural instability and falls (within 3 years of disease onset), cerebellar signs, myoclonus, disproportionate antecollis, extensor plantar responses, cortical sensory loss, emotional incontinence (pseudobulbar affect), severe bulbar dysfunction (dysarthria, dysphonia or dysphagia) or respiratory symptoms such as stridor or inspiratory sighs. 3. Family history of more than 1 first-degree relative with PD. 4. Severe dyskinesias or severe tremor which could interfere with GDNF infusion. 5. Prior neurosurgical treatment for PD, including previous treatment with GDNF or deep brain stimulation. 6. Significant neurological disorder other than PD including clinically significant head trauma, cerebrovascular disease, CSF shunt or other implanted CNS device. 7. Presence of significant depression as defined as a Beck Depression Inventory (BDI) score = 14. 8. Current or past history of psychosis requiring therapy. The presence of benign hallucinosis is not exclusionary. 9. Presence or history of clinically significant impulse control disorder or presence or history of dopamine dysregulation syndrome. 10. MoCA score < 24. 11. Use within 3 months of planned catheter insertion of concomitant medications known to affect PD symptoms other than prescribed PD therapy including but not limited to neuroleptics or other central dopamine receptor blockers. 12. Any medical condition which might impair outcome measure assessments or safety measures including ability to undergo MRI scanning. 13. Screening MRI demonstrating any abnormality which would suggest an alternative cause for subject's parkinsonism. 14. Any medical condition that would put the subject at undue risk from surgical treatment or chronic implants including but not limited to bleeding disorders, chronic infections, or immunosuppressive illness. 15. History within the last 5 years of cancer with the exception of basal cell carcinoma of the skin. 16. History of drug or alcohol abuse within 2 years of planned catheter insertion. 17. Use of any investigational drug or device within 90 days of planned catheter insertion. 18. Active breastfeeding. 5.3.4 Post-Surgery Randomisation Criteria In order to be eligible for entry into the double-blind period of the study, subjects must meet the following criteria after undergoing surgery: 1. No relevant sequelae from catheter implantation such as clinically significant intracerebral trauma, haemorrhage, or infection. 2. Total distribution volume providing at least 50% volume coverage of a predefined volume of interest in each putamen (approximately 25% volume coverage of total putamen), as assessed by an independent review of an MRI scan taken within 2 hours post-infusion of diluent at the end of the healing period. |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
North Bristol NHS Trust |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | UPDRS in the off state | The primary outcome for this study will be the percentage change in motor UPDRS in the practically defined OFF state between baseline and Week 40. | 40 weeks |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT04202757 -
Intravenous Plasma Treatment for Parkinson's Disease
|
Early Phase 1 | |
Not yet recruiting |
NCT05103618 -
Effect of Meditation and Controls and Subjects With Parkinson's Disease on Brain Activity Measured by fMRI With FDOPA
|
Phase 2 | |
Completed |
NCT03700684 -
Voice Treatment for Parkinson's Disease
|
N/A | |
Completed |
NCT05027620 -
Feasibility of Motor-cognitive Home Training for Parkinson's Disease Using eHealth Technology
|
N/A | |
Recruiting |
NCT02960464 -
tDCS for Treatment of Depression in Parkinson's Disease
|
N/A | |
Completed |
NCT05699161 -
Adipose-derived Stromal Vascular Fraction Cells to Treat Parkinson
|
Phase 1/Phase 2 | |
Completed |
NCT03944785 -
Clinical Outcome Assessment of Parkinson's Disease Patients Treated With XADAGO (Safinamide)
|
||
Completed |
NCT01227265 -
Placebo Controlled Study of Preladenant in Participants With Moderate to Severe Parkinson's Disease (P07037)
|
Phase 3 | |
Terminated |
NCT01215227 -
An Active-Controlled Extension Study to NCT01155466 [P04938] and NCT01227265 [P07037] (P06153)
|
Phase 3 | |
Withdrawn |
NCT05832775 -
Study to Assess the Safety of MRx0029 or MRx0005 Compared to Placebo, in People With Parkinson's
|
Phase 1 | |
Active, not recruiting |
NCT02780895 -
Parkinsonian Brain Repair Using Human Stem Cells
|
Phase 1 | |
Recruiting |
NCT01860794 -
Evaluation of Safety and Tolerability of Fetal Mesencephalic Dopamine Neuronal Precursor Cells for Parkinson's Disease
|
Phase 1/Phase 2 | |
Completed |
NCT02373072 -
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of PF-06649751 in Subjects With Idiopathic Parkinson's Disease
|
Phase 1 | |
Terminated |
NCT02393027 -
Quantification of Dopamine Active Transporter (DAT) in Humans: Validation of a New Radiophamaceutical, the [18F] LBT-999
|
Early Phase 1 | |
Completed |
NCT02445651 -
Physiological Effects of Nutritional Support in Patients With Parkinson's Disease
|
N/A | |
Completed |
NCT00437125 -
Study on the Tolerability of Duloxetine in Depressed Patients With Parkinson's Disease
|
Phase 4 | |
Completed |
NCT02723396 -
Sleep, Awake & Move - Part I
|
||
Completed |
NCT00599339 -
Transdermal Rotigotine User Surveillance Study
|
||
Suspended |
NCT05471609 -
Sustained Release Oral Formulation for Treatment of Parkinson's Disease
|
Early Phase 1 | |
Completed |
NCT00160576 -
Levetiracetam Treatment in Adult Subjects With Parkinson's Disease Experiencing Troublesome Dyskinesias
|
Phase 2 |