A study to improve the delivery of AB-1005 gene therapy into the brain

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Askbio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-04-03

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

AskBio Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

Assess the safety and tolerability of PIA-based intraputaminal delivery of AB-1005 up to Month 6 post-surgery

Secondary objectives 3

1. Stage 0: Investigate prefrontal surgical approach for intraputaminal administration of AB-1005 via CED under iMRI monitoring
2. Stage 1: Investigate the use of PIA-based infusion for consistent intraputaminal administration of AB-1005 via CED under iMRI monitoring
3. Stage 2: Test and confirm the success of PIA-based infusion without iMRI monitoring using an OR-based workflow in conjunction with a standard neuronavigational platform

Conditions and MedDRA coding

Parkinson's Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Participants must be 45 to 75 years of age inclusive, at the time of signing the informed consent
2. >10 years since the diagnosis of Parkinson's Disease (PD)
3. Presence of bradykinesia plus any of the following: rigidity, resting tremor, postural instability
4. Modified Hoehn and Yahr stage III in the practically defined OFF state
5. Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III score >40 in the practically defined OFF state
6. Stable anti-PD medication regimen for ≤4 weeks
7. Responsiveness to levodopa as determined by ≥30% improvement in MDS-UPDRS Part III score from the practically defined OFF state to ON state after taking typical first-daily dose of PD-medications
8. Compliance with standard contraceptive requirements
9. Signed informed consent to participante both in PIA and the Long-Term Follow-Up

Exclusion criteria 12

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence and severity of AEs including: serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), TEAEs related to device and/or surgical procedure, TEAEs related to study drug

Secondary endpoints 5

1. Absolute values and changes from baseline in: MoCA score, BDI-II score, C-SSRS , vital sign parameters, physical examinations, clinical laboratory measures, and neuroimaging findings
2. Immunological responses to AAV2 and/or GDNF
3. End-of-infusion volumetric coverage of the putamen as assessed by MRI
4. Duration of surgical procedure
5. Accuracy of cannula tip placement at targeted first and last infusion points in the putamen

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Askbio Inc.

Sponsor organisation

Askbio Inc.

Address

20 Tw Alexander Drive Suite 110

City

Durham

Postcode

27713-2928

Country

United States

Scientific contact point

Organisation

Askbio Inc.

Contact name

Amber Van Laar

Public contact point

Organisation

Askbio Inc.

Contact name

Matthias Luz

Third parties 3

Sponsor responsibilities

Article 77 compliance

Askbio Inc.

Article 77 implementation

Askbio Inc.

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications