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

NCT07371338 | Recruiting Phase 1 DMC

• 1 other identifier: IPS101A-10
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Parkinson Disease; Parkinson's Disease

Keywords

PD; AAV; Adeno-associated Virus; Gene therapy

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (8)

• Change from Baseline in Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores (defined on/off)

  Baseline to Weeks 4, 12, 24, 36, and 52.

• Change from Baseline in Hoehn & Yahr stage (defined on/off)

  Weeks 1, 4, 12, 24, 36, and 52.

• Change from Baseline in Non-Motor Symptoms Scale for Parkinson's Disease (NMSS)

  Weeks 4, 12, 24, 36, and 52.

• Change from Baseline in Parkinson's Disease Questionnaire (PDQ-39) scores

  Weeks 4, 12, 24, 36, and 52

• Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-CSF

  Baseline to Week 52

Study Arms (2)

Low dose (1.0 x 10^10 vg/patient)

EXPERIMENTAL

IPS101A

Drug: IPS101A

High dose(2.0 x 10^10 vg/patient)

EXPERIMENTAL

IPS101A

Drug: IPS101A

Interventions

IPS101A DRUG

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.

High dose(2.0 x 10^10 vg/patient); Low dose (1.0 x 10^10 vg/patient)

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• 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.
• 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.
• Subjects with a Hoehn \& Yahr stage of 4 or 5 in the off state at the Screening Visit.

You may not qualify if:

• 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.
• Subjects with a Korean Mini-Mental State Examination (K-MMSE) score ≤ 24 at the Screening assessment.
• 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.
• Subjects who do not meet the diagnostic criteria for Parkinson's disease dementia but present with major visual hallucinations, as judged by the Investigator.
• Subjects with drug-induced parkinsonism, confirmed by clinical history and/or medical records, and determined by the Investigator.
• Subjects who are judged by the investigator to be unsuitable for participation in this clinical trial.

Sponsors & Collaborators

• Innopeutics Corporation: lead

Study Sites (1)

Severance Hospital

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

ChoLong Park

CONTACT

+82-2-3499-4266; clpark@innopeutics.com

Tae-gyun Kim

CONTACT

contact@innopeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: January 27, 2026
• Study Start (Estimated): May 30, 2026
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)