A Study of AB-1005 Gene Therapy in Japanese Adults With Moderate Parkinson's Disease (J-REGENERATE-PD)

NCT07566429 — Phase 2, Not yet recruiting, 8 participants, Bayer

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A Phase 2, Open-label, Single Group, Fixed-dose Study to Evaluate the Safety and Efficacy of a Single Bilateral Intraputaminal Administration of AB-1005 (AAV2-GDNF Gene Therapy) in Japanese Participants With Moderate Stage Parkinson's Disease

Registry	NCT07566429
Status	Not yet recruiting
Phase	Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	8 (Estimated)
Sponsor	Bayer (Industry); collaborators: AskBio Inc
Conditions	Parkinson Disease
Interventions	AB-1005 (Drug)
Dates	start 2027-01-25 (estimated), primary completion 2029-06-15 (estimated), completion 2032-12-15 (estimated), first posted 2026-05-05, last updated 2026-07-29
Locations	6 sites in Japan

-Summary

The objective of this Phase 2, open-label, single group, one-time, fixed-dose study is to assess the safety and efficacy of bilateral intraputaminal infusion of AB-1005 (AAV2-GDNF gene therapy) in Japanese patients with moderate Parkinson's Disease (PD).

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+Arms and interventions

Arm	Type	Interventions	Description
AB-1005 infusion	Experimental	Drug: AB-1005

AB-1005 (Drug): Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

+Outcome measures

Primary outcomes

Incidence of adverse events up to Month 18 after study intervention [Up to 18 months]: Number and proportion of participants with at least one adverse event
Change from Baseline to Month 18 in normalized Good ON time measured with PD Motor Diary [Up to 18 months]

+Eligibility

Sex: All, ages: 45 Years to 75 Years

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) and currently meeting all the following criteria:

  1. Presence of bradykinesia PLUS any of the following: rigidity, rest tremor, postural instability
  2. Presence of motor fluctuations as measured by the PD Motor Diary
  3. Stable anti-parkinsonian medication regimen for 4 weeks or more, prior to screening
  4. Responsiveness to levodopa therapy

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 clinically significant cognitive impairment
* 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

+Locations (6)

Facility	City	Country
Nagoya Central Hospital - Neurosurgery	Nagoya, Aichi-ken	Japan
Nagoya University Hospital - Neurology	Nagoya, Aichi-ken	Japan
Yokohama City University Medical Center - Neurology	Yokohama, Kanagawa	Japan
National Hospital Organization Osaka Toneyama Medical Center - Neurology	Toyonaka, Osaka	Japan
Juntendo University Hospital - Neurology	Bunkyo-ku, Tokyo	Japan
National Hospital Organization Utano National Hospital - Neurology	Kyoto	Japan