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NCT Number: NCT07371338

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

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.

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Key information

Conditions

Parkinson Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson's Disease Parkinsonian Disorders Synucleinopathies

Age range

50 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Severance Hospital

Seoul, South Korea

Location status: Recruiting

Location contact

Professor, MD, PhD

CONTACT

[email protected]

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

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.

Primary outcomes

  1. Safety Evaluation: dose-limiting toxicity (DLT)

    Time frame: Baseline to Week 8

    Frequency and proportion of subjects who developed dose-limiting toxicity (DLT)

  2. Safety Evaluation: Severity and frequency of reported adverse events

    Time frame: Baseline to Week 52

    Assess severity and frequency of reported adverse events

  3. Safety Evaluation: clinically-relevant changes in laboratory testing assessed by medical personnel

    Time frame: Baseline to Week 52

    The clinical significance of laboratory test results after administration of a clinical trial drug is confirmed by comparing them with those before administration.

  4. Safety Evaluation: clinically-relevant changes in physical exams assessed by medical personnel

    Time frame: Baseline to Week 52

    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.

Secondary outcomes

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

    Time frame: Baseline to Weeks 4, 12, 24, 36, and 52.

    Each symptom is rated on a scale of 0 to 4, with higher scores indicating more severe Parkinson's disease.

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

    Time frame: Weeks 1, 4, 12, 24, 36, and 52.

    This scale is classified from 0 to 5, with a higher score indicating a more severe degree of Parkinson's disease.

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

    Time frame: Weeks 4, 12, 24, 36, and 52.

    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.

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

    Time frame: Weeks 4, 12, 24, 36, and 52

    The scale consists of 800 points, and a higher score indicates a lower quality of life.

  5. Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-CSF

    Time frame: Baseline to Week 52

    After IP administration, the detection of AAV genome was measured using q-PCR.

  6. Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-blood

    Time frame: Baseline to Week 52

    After IP administration, the detection of AAV genome was measured using q-PCR.

  7. Evaluation of humoral (anti-AAV9 antibodies) and cellular (IFN-γ activity) immune responses to AAV9

    Time frame: Baseline to Week 52

    After IP administration, the detection of AAV genome was measured using ELISA

  8. Assessment of AAV9 shedding in biological specimens-urine

    Time frame: Baseline to Week 52

    After IP administration, the detection of AAV genome was measured using q-PCR.

Study contacts

Contact information is provided by the study sponsor or research team.

ChoLong Park

CONTACT

[email protected]

+82-2-3499-4266

Tae-gyun Kim

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Innopeutics Corporation

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Jan 27, 2026
Registry last updated
Apr 21, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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