Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of IPS101A in Parkinson's Disease Patients
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age50-80
SponsorInnopeutics Corporation
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
IPS101A
DrugThe 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.
Treatment groups
6 Participants
are divided into 2 treatment groupsGroup A: Low dose (1.0 x 10^10 vg/patient)Experimental treatment 1 intervention
Group B: High dose(2.0 x 10^10 vg/patient)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety Evaluation: dose-limiting toxicity (DLT)
Frequency and proportion of subjects who developed dose-limiting toxicity (DLT)
Time frame
Baseline to Week 82
Safety Evaluation: Severity and frequency of reported adverse events
Assess severity and frequency of reported adverse events
Time frame
Baseline to Week 523
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.
Time frame
Baseline to Week 524
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.
Time frame
Baseline to Week 52Secondary 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)
Each symptom is rated on a scale of 0 to 4, with higher scores indicating more severe Parkinson's disease.
Time frame
Baseline to Weeks 4, 12, 24, 36, and 52.2
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.
Time frame
Weeks 1, 4, 12, 24, 36, and 52.3
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.
Time frame
Weeks 4, 12, 24, 36, and 52.4
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.
Time frame
Weeks 4, 12, 24, 36, and 52Sponsors and contacts
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Source ClinicalTrials.gov