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Trial record 1 of 9 for:    gdnf therapy | Parkinson Disease
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A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (REGENERATE-PD)

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ClinicalTrials.gov Identifier: NCT06285643
Recruitment Status : Not yet recruiting
First Posted : February 29, 2024
Last Update Posted : February 29, 2024
Sponsor:
Collaborator:
Bayer
Information provided by (Responsible Party):
Asklepios Biopharmaceutical, Inc.

Study Description
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Brief Summary:
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.

Condition or disease Intervention/treatment Phase
Parkinson Disease Drug: AAV2-GDNF Gene therapy Procedure: Control Surgery Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 87 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
Estimated Study Start Date : April 1, 2024
Estimated Primary Completion Date : November 30, 2027
Estimated Study Completion Date : November 30, 2027

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Arms and Interventions
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Arm Intervention/treatment
Experimental: AAV2-GDNF Drug: AAV2-GDNF Gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Other Name: Biological
Sham Comparator: Control Surgery Procedure: Control Surgery
Bilateral partial burr/twist holes without dural penetration


Outcome Measures
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Primary Outcome Measures :
  1. Motor symptoms-MDS-UPDRS parts 2 and 3 [ Time Frame: 18 months ]
  2. PD Motor Diary Non-Motor symptoms-MDS-UPDRS part1 [ Time Frame: 18 months ]
  3. PDQ39 Levodopa Equivalent Daily Dose [ Time Frame: 18 months ]

Secondary Outcome Measures :
  1. Change from baseline in the MDS-UPDRS parts 2 and 3, and the PD Motor Diary [ Time Frame: 18 months ]
  2. Change from baseline in the MDS-UPDRS part 1 and PDQ-39 [ Time Frame: 18 months ]
  3. Change in Levodopa equivalent daily dose [ Time Frame: 18 months ]

Eligibility Criteria
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Ages Eligible for Study:   45 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent
  • Diagnosed with Parkinson's Disease in the past 4-10 years inclusive defined by the following:

Presence of bradykinesia PLUS any of the following:

Rigidity Rest Tremor Postural instability

  • Modified Hoehn and Yahr stage II-III in the practically defined OFF medication state (>/= 12 hours from last dose of anti-parkinsonian medications)
  • MDS-UPDRS Part III score in the practically defined OFF state of 33 to 60 (inclusive)
  • Presence of motor fluctuations with >/= 2.5 hours of absolute time in the OFF state averaged over 3 days as measured by the PD Motor Diary, and assessed at screening, and baseline visit 1 and 2
  • Stable anti-parkinsonian medication regimen for >/= 4 weeks prior to Screening and held stable throughout the baseline period o Responsiveness to levodopa as determined by change in modified H&Y stage or MDS-UPDRS Part III score from the practically defined OFF state to ON state after taking first daily dose of PD medication

Exclusion Criteria:

  • Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
  • Presence or history of significant vascular and/or cardiovascular disease
  • Presence of significant cognitive impairment, poorly controlled depression/anxiety
  • Presence or history of psychosis or impulse control disorder
  • History of malignancy other than treated cutaneous squamous or basal cell carcinomas
  • Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
  • Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
  • Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
  • Chronic immunosuppressive therapy
Contacts and Locations
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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): NCT06285643


Contacts
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Contact: Nisha Chhabria, MD 919-388-1040 askfirst@askbio.com
Contact: Christian Urrea, MD 919-388-1040 askfirst@askbio.com

Sponsors and Collaborators
Asklepios Biopharmaceutical, Inc.
Bayer
More Information
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Responsible Party: Asklepios Biopharmaceutical, Inc.
ClinicalTrials.gov Identifier: NCT06285643    
Other Study ID Numbers: ASK-PD5-CS201
First Posted: February 29, 2024    Key Record Dates
Last Update Posted: February 29, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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 Asklepios Biopharmaceutical, Inc.:
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
Synucleinopathies
Neurodegenerative Diseases