ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 2 ENCODING GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR for Parkinson's Disease

Inclusion criteria

• {"criterion_text":"- Participants must be 45 to 75 years of age inclusive, at the time of signing the informed consent"}
• {"criterion_text":"- >10 years since the diagnosis of Parkinson's Disease (PD)"}
• {"criterion_text":"- Presence of bradykinesia plus any of the following: rigidity, resting tremor, postural instability"}
• {"criterion_text":"- Modified Hoehn and Yahr stage III-IV in the practically defined OFF state"}
• {"criterion_text":"- Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III score >40 in the practically defined OFF state"}
• {"criterion_text":"- Stable anti-PD medication regimen for ≤4 weeks"}
• {"criterion_text":"- ≥30% reduction in MDS-UPDRS Part III following a levodopa challenge"}
• {"criterion_text":"- Compliance with standard contraceptive requirements"}
• {"criterion_text":"- Signed informed consent to participante both in PIA and the Long-Term Follow-Up"}

Exclusion criteria

• {"criterion_text":"- Evidence of secondary or atypical parkinsonism"}
• {"criterion_text":"- Chronic immunosuppressive therapy"}
• {"criterion_text":"- Prior cell or gene therapy"}
• {"criterion_text":"- Relevant laboratory values abnormalities"}
• {"criterion_text":"- Presence or history of psychosis or impulse control disorder"}
• {"criterion_text":"- Presence of untreated or suboptimally treated depression (Beck Depression Inventory [BDI]-II score ≥20)"}
• {"criterion_text":"- Suicidal ideation (Columbia-Suicide Severity Rating Scale [C-SSRS] items 4 and/or 5 positive)"}
• {"criterion_text":"- Clinically significant cognitive impairment (Montreal Cognitive Assessment [MoCA] score <25)"}
• {"criterion_text":"- Presence or history of malignancy (except cutaneous squamous or basal cell carcinomas)"}
• {"criterion_text":"- Contraindications to MRI, surgery, and/or anesthesia (in prone position)"}
• {"criterion_text":"- Use of PD pump therapies (intestinal gel, subcutaneous apomorphine)"}
• {"criterion_text":"- Prior brain surgery (including deep brain stimulation [DBS])"}

Primary endpoints

• {"endpoint_text":"- End-of-infusion volumetric putaminal coverage as assessed by MRI (Stage 1 and 2)","definition_or_measurement_approach":"Volumetric putaminal coverage measured by MRI at end of infusion (Stage 1 and 2)"}

Secondary endpoints

• {"endpoint_text":"- Duration of surgical procedure (Stage 1 and 2)","definition_or_measurement_approach":"Measured duration of the surgical procedure (Stage 1 and 2)"}
• {"endpoint_text":"- Change in total volumetric putaminal coverage within approximately 60-120 min from end of infusion (Stage 1)","definition_or_measurement_approach":"Change in total volumetric putaminal coverage measured by MRI approximately 60–120 minutes after end of infusion (Stage 1)"}
• {"endpoint_text":"- Accuracy of cannula tip placement at targeted first and last infusion stops in putamen (Stage 1 and 2)","definition_or_measurement_approach":"Assessment of cannula tip placement accuracy at first and last targeted infusion stops (Stage 1 and 2)"}
• {"endpoint_text":"- Incidence and severity of AEs including: SAES, TEAEs, TEAEs related to device and/or surgical procedure, TEAEs related to study drug (Stage 1 and 2)","definition_or_measurement_approach":"Adverse events recorded and categorised including SAE, TEAE and relationship to device/procedure/drug (Stage 1 and 2)"}
• {"endpoint_text":"- MoCA score (Stage 1 and 2)","definition_or_measurement_approach":"Montreal Cognitive Assessment score measured at specified visits (Stage 1 and 2)"}
• {"endpoint_text":"- BDI-II score (Stage 1 and 2)","definition_or_measurement_approach":"Beck Depression Inventory-II score measured at specified visits (Stage 1 and 2)"}
• {"endpoint_text":"- C-SSRS (Stage 1 and 2)","definition_or_measurement_approach":"Columbia-Suicide Severity Rating Scale assessments (Stage 1 and 2)"}
• {"endpoint_text":"- Vital sign parameters (Stage 1 and 2)","definition_or_measurement_approach":"Standard vital sign measurements (Stage 1 and 2)"}
• {"endpoint_text":"- Neurological and physical examinations (Stage 1 and 2)","definition_or_measurement_approach":"Clinical neurological and physical examinations (Stage 1 and 2)"}
• {"endpoint_text":"- Clinical laboratory measures (Stage 1 and 2)","definition_or_measurement_approach":"Laboratory tests evaluated per protocol (Stage 1 and 2)"}
• {"endpoint_text":"- Neuroimaging findings (Stage 1 and 2)","definition_or_measurement_approach":"Neuroimaging assessments (MRI) as specified (Stage 1 and 2)"}
• {"endpoint_text":"- Immunological responses to AAV2 and/or GDNF (Stage 1 and 2)","definition_or_measurement_approach":"Immunologic assays measuring responses to AAV2 and/or GDNF (Stage 1 and 2)"}

Poland

Earliest CTIS Part Ii Submission Date

03-03-2026

Latest Decision Or Authorization Date

03-04-2026

Processing Time Days

31

Number Of Sites

3

Number Of Participants

12

Primary sponsor

Full Name

Askbio Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Third parties

• {"country":"Germany","full_name":"Bayer AG","duties_or_roles":"Sponsor duties codes listed in submission: 1, 10, 11, 12, 13, 15 (Central Imaging Service), 5, 6, 7, 8, 9; contact email: clinical-trials-contact@bayer.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Sample management (sponsor duty code 15); contact email: jean-paul.lewest@labcorp.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Concierge services (sponsor duty code 15); contact email: ctis_inquiry@greenphire.com","organisation_type":"Non-Pharmaceutical company"}

Co-sponsors

• AskBio France