IPS101A AAV gene therapy for advanced Parkinson's disease

Dose-Escalation, Single-Center, Open-label Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Adeno-associated Virus(AAV) Gene Therapy Product IPS101A in Parkinson's Disease Patients With Hoehn-Yahr Stage 4-5, Diagnosed in More Than 10 Years and Uncontrolled by All Available Monotherapy or Combination Therapy

This Phase 1 test will see if IPS101A, an AAV gene therapy, is safe and helps people aged 50–80 with advanced Parkinson's (Hoehn & Yahr 4–5) whose symptoms are not controlled by standard treatments.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 50 Years to 80 Years
• Sex: All
• Sponsor: Innopeutics Corporation (Industry-sponsored)
• Locations: 1 site (Seoul)
• Trial ID: NCT07371338 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, interventional, dose-escalation trial testing IPS101A, an adeno-associated virus (AAV) gene therapy, in patients with advanced Parkinson's disease. The primary focus is on dose-related safety and tolerability and determining a maximum tolerated dose, with secondary collection of preliminary efficacy signals and pharmacokinetic data. Eligible participants are 50–80 years old with at least 10 years of Parkinson's disease and Hoehn & Yahr stage 4–5 in the off state who remain inadequately controlled on standard therapies. The study is sponsored by Innopeutics Corporation and conducted at Severance Hospital in Seoul.

Who should consider this trial

Good fit: Ideal candidates are adults 50–80 years old with a UK Brain Bank–confirmed Parkinson's diagnosis of at least 10 years' duration, Hoehn & Yahr stage 4–5 off state, symptoms not controlled by standard therapies, and without Parkinson's disease dementia.

Not a fit: Patients with Parkinson's disease dementia, earlier-stage disease, or symptoms already well controlled by medication are unlikely to receive benefit from this intervention.

Why it matters

Potential benefit: If successful, IPS101A could reduce disabling symptoms or improve function for people with advanced, treatment-refractory Parkinson's disease.

How similar studies have performed: Other AAV-based gene therapy programs for Parkinson's have completed early-phase testing and in some cases shown acceptable safety, but they have produced limited and inconsistent evidence of clear clinical benefit so far.

Eligibility criteria

Inclusion Criteria:

1. Subjects who have a diagnosis of Parkinson's disease that meets the UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria at the time of the Screening Visit.
2. Male or female subjects aged 50 to 80 years (inclusive) at the time of providing written informed consent, with a documented diagnosis of Parkinson's disease.
3. Subjects with a duration of Parkinson's disease of at least 10 years prior to the Screening Visit, based on medical history and/or medical records.
4. Subjects with Parkinson's disease that is inadequately controlled despite all available standard-of-care treatments, including monotherapy or combination therapy, as determined by the Investigator.
5. Subjects with a Hoehn \& Yahr stage of 4 or 5 in the off state at the Screening Visit.

Exclusion Criteria:

1. Subjects with Parkinson's disease dementia (PDD) who meet the diagnostic criteria established by the Movement Disorder Society (MDS) Task Force, as determined by the Investigator at Screening.
2. Subjects with a Korean Mini-Mental State Examination (K-MMSE) score ≤ 24 at the Screening assessment.
3. Subjects in whom imaging findings suggestive of Parkinsonism-plus syndrome are observed on PET and MRI performed at the Screening Visit, as assessed by the Investigator and/or a qualified imaging specialist.
4. Subjects who do not meet the diagnostic criteria for Parkinson's disease dementia but present with major visual hallucinations, as judged by the Investigator.
5. Subjects with drug-induced parkinsonism, confirmed by clinical history and/or medical records, and determined by the Investigator.
6. Subjects who are judged by the investigator to be unsuitable for participation in this clinical trial.

Where this trial is running

Seoul

• Severance Hospital — Seoul, South Korea (Recruiting)

Study contacts

• Study coordinator: ChoLong Park

Conditions Parkinson Disease; Parkinson's Disease; PD; AAV; Adeno-associated Virus; Gene therapy