Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of IPS101A in Parkinson's Disease Patients

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

The purpose of this study is to evaluate the dose-related safety and tolerability of IPS101A, an adeno-associated virus (AAV) gene therapy, in patients with Parkinson's disease who exhibit severe functional impairment corresponding to Hoehn & Yahr stages 4-5 and whose symptoms are not adequately controlled despite all available monotherapy and combination therapy options. In addition, the study aims to assess the maximum tolerated dose (MTD) of IPS101A, as well as its preliminary efficacy and pharmacokinetic (PK) characteristics.

Study Overview

• Status: Recruiting
• Conditions: Parkinson's Disease; Parkinson Disease
• Intervention / Treatment: Drug: IPS101A

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: ChoLong Park; Phone Number: +82-2-3499-4266; Email: clpark@innopeutics.com
• Study Contact Backup: Name: Tae-gyun Kim; Email: contact@innopeutics.com

Study Locations

• South Korea
  • Seoul, South Korea
    • Recruiting
    • Severance Hospital
    • Contact: Professor, MD, PhD; Email: phlee@yuhs.ac

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low dose (1.0 x 10^10 vg/patient); IPS101A	Drug: IPS101A; The investigational product (IP) will be administered as a single dose. All subjects will receive stereotactic injections into the left and right substantia nigra of the midbrain, with one administration per side.
Experimental: High dose(2.0 x 10^10 vg/patient); IPS101A	Drug: IPS101A; The investigational product (IP) will be administered as a single dose. All subjects will receive stereotactic injections into the left and right substantia nigra of the midbrain, with one administration per side.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety Evaluation: dose-limiting toxicity (DLT)	Frequency and proportion of subjects who developed dose-limiting toxicity (DLT)	Baseline to Week 8
Safety Evaluation: Severity and frequency of reported adverse events	Assess severity and frequency of reported adverse events	Baseline to Week 52
Safety Evaluation: clinically-relevant changes in laboratory testing assessed by medical personnel	The clinical significance of laboratory test results after administration of a clinical trial drug is confirmed by comparing them with those before administration.	Baseline to Week 52
Safety Evaluation: clinically-relevant changes in physical exams assessed by medical personnel	The results of the physical examination after administration of the clinical trial drug are compared with those before administration to determine whether clinically significant symptoms occur.	Baseline to Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores (defined on/off)	Each symptom is rated on a scale of 0 to 4, with higher scores indicating more severe Parkinson's disease.	Baseline to Weeks 4, 12, 24, 36, and 52.
Change from Baseline in Hoehn & Yahr stage (defined on/off)	This scale is classified from 0 to 5, with a higher score indicating a more severe degree of Parkinson's disease.	Weeks 1, 4, 12, 24, 36, and 52.
Change from Baseline in Non-Motor Symptoms Scale for Parkinson's Disease (NMSS)	This scale is rated on a scale of severity (0-3 points) and frequency (1-4 points). A higher score indicates more severe Parkinson's disease.	Weeks 4, 12, 24, 36, and 52.
Change from Baseline in Parkinson's Disease Questionnaire (PDQ-39) scores	The scale consists of 800 points, and a higher score indicates a lower quality of life.	Weeks 4, 12, 24, 36, and 52
Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-CSF	After IP administration, the detection of AAV genome was measured using q-PCR.	Baseline to Week 52
Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-blood	After IP administration, the detection of AAV genome was measured using q-PCR.	Baseline to Week 52
Evaluation of humoral (anti-AAV9 antibodies) and cellular (IFN-γ activity) immune responses to AAV9	After IP administration, the detection of AAV genome was measured using ELISA	Baseline to Week 52
Assessment of AAV9 shedding in biological specimens-urine	After IP administration, the detection of AAV genome was measured using q-PCR.	Baseline to Week 52

Collaborators and Investigators

• Sponsor: Innopeutics Corporation

Study record dates

Study Major Dates

• Study Start (Estimated): May 30, 2026
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: November 17, 2025
• First Submitted That Met QC Criteria: January 19, 2026
• First Posted (Actual): January 27, 2026

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 19, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: AAV; Gene therapy; PD; Adeno-associated Virus
• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: IPS101A-10

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No