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GDNF Gene Therapy for Parkinson's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04167540
Recruitment Status : Recruiting
First Posted : November 19, 2019
Last Update Posted : January 23, 2020
Sponsor:
Information provided by (Responsible Party):
Brain Neurotherapy Bio, Inc.

Tracking Information
First Submitted Date  ICMJE November 7, 2019
First Posted Date  ICMJE November 19, 2019
Last Update Posted Date January 23, 2020
Estimated Study Start Date  ICMJE February 2020
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 14, 2019)
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations [ Time Frame: 5 years ]
Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 14, 2019)
  • Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) [ Time Frame: 18 months ]
    Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores.
  • Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS) [ Time Frame: 18 months ]
    Change from baseline in the NMSS. The NMSS evaluates 9 domains of non-motor of severity and frequency of PD symptoms associated with cardiovascular health, sleep and fatigue, mood and cognition, perceptual problems and hallucinations, attention and memory, gastrointestinal tract, urinary, sexual function, and a miscellaneous domain for other common non-motor conditions. Severity x frequency scores range 0-108, with 0 being less severe and less frequent.
  • Brain dopaminergic cell integrity as measured by DaTscan [ Time Frame: 18 months ]
    Percentage and absolute changes in Ioflupane retention as a marker for dopamine transporter protein expressed by dopamine producing cells within the brain. Measured by quantitative analysis of DaTscan SPECT imaging.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE GDNF Gene Therapy for Parkinson's Disease
Official Title  ICMJE Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
Brief Summary The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
A total of 12 study participants will be administered the investigational product in this Phase 1b trial. Participants will be enrolled into cohorts, based upon the duration and stage of their PD. Six (6) participants will be dosed in each cohort.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Parkinson's Disease
Intervention  ICMJE Biological: AAV2-GDNF
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Study Arms  ICMJE
  • Experimental: Earlier stage PD
    Intervention: Biological: AAV2-GDNF
  • Experimental: Later stage PD
    Intervention: Biological: AAV2-GDNF
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 14, 2019)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2026
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Male and female adults 35-75 years of age (inclusive)
  • Diagnosed with Parkinson's disease
  • Modified Hoehn and Yahr stage I-III OFF medication
  • Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following:

    1. EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score
    2. OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score
  • Responsiveness to levodopa

Key Exclusion Criteria:

  • Atypical parkinsonism
  • Severe dyskinesia
  • Presence of dementia, psychosis, substance abuse or qualify as "severe depression"
  • Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities
  • Receiving an investigational drug
  • History of cancer or poorly controlled medical conditions that would increase surgical risk
  • Inability to tolerate laying flat in an MRI or allergy to gadolinium
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 35 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Amber Van Laar, MD 510-358-8508 avanlaar@brainneubio.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04167540
Other Study ID Numbers  ICMJE GDNF-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Brain Neurotherapy Bio, Inc.
Study Sponsor  ICMJE Brain Neurotherapy Bio, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Brain Neurotherapy Bio, Inc.
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP