Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of IPS101A in Parkinson's Disease Patients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: IPS101A.
- Who it may be relevant to
- Registry conditions: Parkinson Disease, Parkinson's Disease. Basic parameters: 50 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
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.
Interventions
- 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.
Primary outcome measures
- Safety Evaluation: dose-limiting toxicity (DLT) [Time frame: Baseline to Week 8]
- Safety Evaluation: Severity and frequency of reported adverse events [Time frame: Baseline to Week 52]
- Safety Evaluation: clinically-relevant changes in laboratory testing assessed by medical personnel [Time frame: Baseline to Week 52]
- Safety Evaluation: clinically-relevant changes in physical exams assessed by medical personnel [Time frame: Baseline to Week 52]
Secondary outcome measures (8)
- 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.]
- Change from Baseline in Hoehn & Yahr stage (defined on/off) [Time frame: Weeks 1, 4, 12, 24, 36, and 52.]
- Change from Baseline in Non-Motor Symptoms Scale for Parkinson's Disease (NMSS) [Time frame: Weeks 4, 12, 24, 36, and 52.]
- Change from Baseline in Parkinson's Disease Questionnaire (PDQ-39) scores [Time frame: Weeks 4, 12, 24, 36, and 52]
- Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-CSF [Time frame: Baseline to Week 52]
- Distribution evaluation of IPS101A (AAV9 vector distribution evaluation)-blood [Time frame: Baseline to Week 52]
- Evaluation of humoral (anti-AAV9 antibodies) and cellular (IFN-γ activity) immune responses to AAV9 [Time frame: Baseline to Week 52]
- Assessment of AAV9 shedding in biological specimens-urine [Time frame: Baseline to Week 52]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
South Korea · 1 center
- Severance Hospital — Seoul
Identifiers
NCT: NCT07371338 · IPS101A-10