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

Inclusion criteria

• {"criterion_text":"- Male and female adults 45-75 years of age inclusive\n- Diagnosed with Parkinson’s Disease in the past 4 to 10 years (inclusive) as defined by the following: •\tPresence of bradykinesia PLUS any of the following: •\tRigidity •\tRest Tremor •\tPostural Instability\n- Modified Hoehn and Yahr stage II-III in the practically defined OFF medication state (≥ 12 hours from last dose of anti-parkinsonian medications)\n- Responsiveness to levodopa"}

Exclusion criteria

• {"criterion_text":"- Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to an idiopathic PD diagnosis\n- Presence or history of significant vascular and/or cardiovascular disease\n- Presence of significant cognitive impairment, poorly controlled depression/anxietyc\n- Presence or history of psychosis or impulse control disorder\n- History of malignancy other than treated cutaneous squamous or basal cell carcinomas\n- Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety\n- Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents\n- Prior history of brain surgery including, but not limited: DBS, pallidotomy, focused ultrasound thalamotomy, or other experimental neurosurgical procedure\n- Chronic immunosuppressive therapy (e.g., chronic steroids, tumor necrosis factor antagonists, chemotherapy)"}

Primary endpoints

• {"endpoint_text":"- Change from Baseline to Month 18 in normalized Good ON time measured with PD Motor Diary","definition_or_measurement_approach":"Measured as change from baseline to Month 18 in normalized Good ON time as recorded by participant PD Motor Diary (PD Motor Diary used to assess ON/OFF time)."}

Secondary endpoints

• {"endpoint_text":"- Change from Baseline to Month 18 in the following measures: \t-MDS-UPDRS part III score in the practically defined OFF state \t-FDOPA uptake in the putamen as assessed by PET \t-Normalized OFF time measured with PD Motor Diary \t-MDS-UPDRS part III score in the ON state \t-MDS-UPDRS part II score in the OFF state \t-UDysRS total score \t-PDQ-39 summary index \t-NMSS total score\n- Incidence and severity of AEs, TEAEs, and SAEs\n- Vital signs\n- Safety laboratory assessments\n- Neurological and physical examinations\n- Antibody responses to AAV2 and/or GDNF\n- T-cell responses against AAV2 and/or GDNF\n- C-SSRS\n- Shedding of AAV2-GDNF","definition_or_measurement_approach":"Secondary measures include standard clinical scales and assessments: MDS-UPDRS parts II/III in defined ON/OFF states, FDOPA PET uptake in putamen (assessed by PET imaging), normalized OFF/ON time via PD Motor Diary, UDysRS total score, PDQ-39 summary index, NMSS total score. Safety endpoints assessed by incidence/severity of AEs/TEAEs/SAEs, vital signs, safety laboratory tests, neurological/physical exams. Immunogenicity measured by antibody and T-cell responses to AAV2 and/or GDNF. Suicidality assessed by C-SSRS. Shedding assessed for AAV2-GDNF."}

Methods

• myTomorrows digital recruitment materials: Google Ads copy, Clinical Trial Landing Page copy and Landing Page screenshots (documents named myTomorrows_Google Ads copy, Clinical Trial Landing Page Copy, Landing Page Screenshots) — digital advertising and landing page for patient-facing recruitment (documents available for PL and DE).
• myTomorrows Patient Navigator Script — patient navigator outreach via myTomorrows platform (patient support/navigation).
• Patient Database Letter — letter to patient databases (documents: Patient Database Letter for PL and DE).
• Advocacy Pack Letter — outreach to advocacy groups via an advocacy pack letter (documents present for PL and DE).
• Dear Doctor Letter — clinician outreach to referring physicians (documents present for PL and DE).
• Patient Brochure / PD Phase 2 Patient Brochure — informational printed/digital brochure for prospective participants (documents present for PL and DE).
• Third-party-supported recruitment and site support (organisations with duties such as Patient recruitment, Patient Travel, Patient Retention and Recruitment, Patient Navigator, Site Training Platform) as listed in third-party roles.

Poland

Latest Decision Or Authorization Date

26-01-2026

Number Of Sites

5

Number Of Participants

35

Germany

Latest Decision Or Authorization Date

23-01-2026

Number Of Sites

5

Number Of Participants

20

Primary sponsor

Full Name

Askbio Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Gray Consulting Inc.

Responsibilities

Patient Travel

Name

Longboat Clinical Limited

Responsibilities

Site Training Platform

Name

Cti Clinical Trial Services Inc.

Responsibilities

Multiple operational roles (sponsorDuties codes: 1,12,13,2,4,5)

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

Multiple operational roles (sponsorDuties codes: 1,12,2,5)

Name

WCG Clinical Inc.

Responsibilities

Central Rater and ePRO/eCOA

Name

4g Clinical LLC

Responsibilities

sponsorDuties code: 3

Third parties

• {"country":"France","full_name":"Genosafe S.A.S.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient Travel","organisation_type":"Pharmaceutical company"}
• {"country":"Spain","full_name":"CTI Laboratory Services Spain S.L.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Longboat Clinical Limited","duties_or_roles":"Site Training Platform (sponsorDuties code: 15)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Central Rater and ePRO/eCOA (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Cti Clinical Trial Services Inc.","duties_or_roles":"sponsorDuties codes: 1,12,13,2,4,5","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"sponsorDuties codes: 1,12,2,5","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Bayer AG","duties_or_roles":"Central Imaging; sponsorDuties codes: 12,8","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient Retention and Recruitment (sponsorDuties code: 15)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Finland","full_name":"Blueprint Genetics Oy","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Clearpoint Neuro Inc.","duties_or_roles":"Provision and oversight of surgical software (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Impatients N.V.","duties_or_roles":"Patient recruitment (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scarritt Group Inc.","duties_or_roles":"Investigator Meeting (sponsorDuties code: 15)","organisation_type":"Non-Pharmaceutical company"}