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Parkinson DiseasePhase 2REGENERATE-PDNCT06285643

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

A Phase 2 study testing AAV2-GDNF gene therapy in people with Parkinson Disease, run by AskBio Inc at 46 sites in 4 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 45 to 75 years
Conditions the study is for
Parkinson Disease
What is being tested?
AAV2-GDNF gene therapy
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
59 participants
Where
46 sitesin 4 countries
Study dates
11 Jun 2024to 31 Aug 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 45 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age
  • Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics
  • Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:
  • Presence of bradykinesia PLUS any of the following:
  • Rigidity
  • Rest tremor
Show all 10Show fewer
  • Postural instability
  • Presence of motor fluctuations as measured by the PD Motor Diary
  • Stable anti-parkinsonian medication regimen for >/= 4 weeks prior to screening
  • Must demonstrate responsiveness to levodopa therapy

Who cannot join

  • 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
Show all 9Show fewer
  • 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

Trial locations and contacts

46 sites in 4 countries.

46 sites in 4 countries

United States34 sites

  • Emory University (Surgical)

    Atlanta, Georgia, 30322

  • Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)

    Boca Raton, Florida, 33486

  • Massachusetts General Hospital (Neurology)

    Boston, Massachusetts, 02114

  • Massachusetts General Hospital (Surgical)

    Boston, Massachusetts, 02114

  • University of North Carolina (Neurology)

    Chapel Hill, North Carolina, 27514

  • Rush University Medical Center (Neurology)

    Chicago, Illinois, 60612

  • Ohio State University (Surgical)

    Columbus, Ohio, 43210

  • Duke Neurosurgery (Surgical)

    Durham, North Carolina, 27705

  • Duke University (Neurology)

    Durham, North Carolina, 27710

  • Michigan State University (Neurology)

    East Lansing, Michigan, 48824

  • CenExel Rocky Mountain Clinical Research (Neurology)

    Englewood, Colorado, 80113

  • Quest Research Institute (Neurology)

    Farmington Hills, Michigan, 48334

  • University of Florida

    Gainesville, Florida, 32608

  • Texas Movement Disorder Specialists

    Georgetown, Texas, 78628

  • Houston Methodist (Neurology)

    Houston, Texas, 77030

  • Houston Methodist (Surgical)

    Houston, Texas, 77030

  • UCI Alpha Clinic (Neurology)

    Irvine, California, 92697

  • University of Kansas Medical Center (Neurology)

    Kansas City, Kansas, 66103

  • EvergreenHealth Research Department (Neurology)

    Kirkland, Washington, 98034

  • Cleveland Clinic Lou Ruvo (Neurology)

    Las Vegas, Nevada, 89106

  • Loma Linda University (Neurology)

    Loma Linda, California, 92354

  • Loma Linda University (Surgical)

    Loma Linda, California, 92354

  • University of Wisconsin (Neurology)

    Madison, Wisconsin, 53705

  • University of Miami

    Miami, Florida, 33125

  • Vanderbilt University (Neurology)

    Nashville, Tennessee, 37232

  • Columbia University

    New York, New York, 10032

  • Orlando Health Neuroscience Institute

    Orlando, Florida, 32806

  • University of Pennsylvania (Neurology)

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinic

    Phoenix, Arizona, 85054

  • Parkinson's Disease Treatment Center of Southwest Florida (Neurology)

    Port Charlotte, Florida, 33980

  • University of Utah

    Salt Lake City, Utah, 84132

  • University of California San Francisco (Neurology)

    San Francisco, California, 94143

  • University of San Francisco (Surgical)

    San Francisco, California, 94143

  • Georgetown University (Neurology)

    Washington D.C., District of Columbia, 20057

Other trials in United States

Germany5 sites

  • Charité - Universitätsmedizin Berlin (Surgical)

    Berlin

  • Philipps-Universität Marburg (Neurology)

    Marburg

  • Universitätsklinikum Tübingen (Neurology)

    Tübingen

  • Universitätsklinikum Tübingen (Surgical)

    Tübingen

  • Universitätsklinikum Würzburg (Neurology)

    Würzburg

Other trials in Germany

Poland5 sites

  • Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)

    Gdansk

  • Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)

    Katowice

  • Mazowiecki Szpital Bródnowski (Neurology)

    Warsaw

  • Mazowiecki Szpital Bródnowski (Surgical)

    Warsaw

  • Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)

    Warsaw

Other trials in Poland

United Kingdom2 sites

  • North Bristol NHS Trust - Southmead Hospital (Neurology)

    Bristol

  • University Hospital of Wales (Surgical)

    Cardiff

Other trials in United Kingdom

About this study

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

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.

Being tested
AAV2-GDNF gene therapy, control surgery
Keywords
Gene therapy
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AAV2-GDNF gene therapyDrug

    Also called Biological

    Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

  • control surgeryProcedure or surgery

    Bilateral partial burr/twist holes without dural penetration

Study groups in NCT06285643
Study groupRoleWhat they receive
AAV2-GDNFExperimentalNot described
Receives: Drug: AAV2-GDNF gene therapy
Control SurgerySham comparatorNot described
Receives: Procedure: control surgery

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline to Month 18 on PD Motor Diary

    Normalized Good ON time per Hauser Diary

    Measured: 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Feb 2024
  2. Study start11 Jun 2024
  3. Primary completion31 Aug 2028 (estimated)
  4. Study completion31 Aug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
59 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06285643
Sponsor's protocol ID
ASK-PD5-CS201
Acronym
REGENERATE-PD
Status last verified
Nov 2026
First submitted
14 Feb 2024
Last updated
5 Nov 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AAV2-GDNF gene therapy in people with Parkinson Disease, run by AskBio Inc at 46 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 46 sites in United States, Germany, Poland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline to Month 18 on PD Motor Diary (time frame: 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06285643.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.

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