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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low dose (1.0 x 10^10 vg/patient) | Experimental | IPS101A |
|
| High dose(2.0 x 10^10 vg/patient) | Experimental | IPS101A |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| 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 |
| 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| ChoLong Park | Contact | +82-2-3499-4266 | clpark@innopeutics.com | |
| Tae-gyun Kim | Contact | contact@innopeutics.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Severance Hospital | Recruiting | Seoul | South Korea |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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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 |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |