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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 : December 20, 2019
Sponsor:
Information provided by (Responsible Party):
Brain Neurotherapy Bio, Inc.

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.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Biological: AAV2-GDNF Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
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
Official Title: Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
Estimated Study Start Date : December 2019
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : June 2026

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Earlier stage PD Biological: AAV2-GDNF
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Experimental: Later stage PD Biological: AAV2-GDNF
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.



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Ages Eligible for Study:   35 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04167540


Contacts
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Contact: Amber Van Laar, MD 510-358-8508 avanlaar@brainneubio.com

Locations
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United States, California
University of California San Francisco Not yet recruiting
San Francisco, California, United States, 94103
Contact: Marin Thompson         
Principal Investigator: Chadwick Christine, MD         
Sub-Investigator: Paul Larson, MD         
United States, Ohio
The Ohio State University Medical Center Recruiting
Columbus, Ohio, United States, 43210
Contact: Katherine Ambrogi, BSN, RN    614-688-6685    Katherine.Ambrogi@osumc.edu   
Principal Investigator: J. Bradley Elder, MD         
Sub-Investigator: Sandra Kostyk, MD         
Sponsors and Collaborators
Brain Neurotherapy Bio, Inc.

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Responsible Party: Brain Neurotherapy Bio, Inc.
ClinicalTrials.gov Identifier: NCT04167540    
Other Study ID Numbers: GDNF-102
First Posted: November 19, 2019    Key Record Dates
Last Update Posted: December 20, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Brain Neurotherapy Bio, Inc.:
PD
Parkinson's disease
Neurotrophic factor
Growth factor
Glial cell line-derived neurotrophic factor
GDNF
AAV
Gene therapy
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases