Parkinson DiseasePhase 1NCT07371338
Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of IPS101A in Parkinson's Disease Patients
A Phase 1 study testing IPS101A in people with Parkinson Disease and Parkinson's Disease, run by Innopeutics Corporation at 1 site in South Korea.
Trial 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… Read more about this study
- Who is it for?
- Ages 50 to 80 years
- Conditions the study is for
- Parkinson Disease, Parkinson's Disease
- What is being tested?
- IPS101A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteSouth Korea
- Study dates
- 13 May 2026to 31 Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Tae-gyun Kim contact
South Korea1 site, 1 recruiting
- Recruiting
Severance Hospital
Seoul
Professor, MD, PhD, phlee@yuhs.ac
About this study
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
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
- Being tested
- IPS101A
- Keywords
- PD, AAV, Adeno-associated Virus, Gene therapy
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IPS101ADrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Low dose (1.0 x 10^10 vg/patient) | Experimental | IPS101A Receives: Drug: IPS101A |
| High dose(2.0 x 10^10 vg/patient) | Experimental | IPS101A Receives: Drug: IPS101A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Evaluation: dose-limiting toxicity (DLT)
Frequency and proportion of subjects who developed dose-limiting toxicity (DLT)
Measured: Baseline to Week 8
Safety Evaluation: Severity and frequency of reported adverse events
Assess severity and frequency of reported adverse events
Measured: 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.
Measured: 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.
Measured: 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)
Each symptom is rated on a scale of 0 to 4, with higher scores indicating more severe Parkinson's disease.
Measured: Baseline to Weeks 4, 12, 24, 36, and 52.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2026
- Study start13 May 2026
- Primary completion30 Jun 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Innopeutics Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07371338
- Sponsor's protocol ID
- IPS101A-10
- Status last verified
- Nov 2026
- First submitted
- 17 Nov 2025
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07371338
- Acronym / sponsor ID
- IPS101A-10
- Conditions
- Parkinson Disease, Parkinson's Disease
- Keywords
- PD, AAV, Adeno-associated Virus, Gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Innopeutics CorporationIndustry (pharma, biotech, devices)
- Study start
- 13 May 2026 (actual)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 27 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low dose (1.0 x 10^10 vg/patient) | Experimental | Drug: IPS101AIPS101A |
| High dose(2.0 x 10^10 vg/patient) | Experimental | Drug: IPS101AIPS101A |
Who may qualify
- Ages
- 50 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IPS101AThe 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. | Drug | High dose(2.0 x 10^10 vg/patient), Low dose (1.0 x 10^10 vg/patient) |
Primary outcome
Safety Evaluation: dose-limiting toxicity (DLT)
Time frame: Baseline to Week 8
Frequency and proportion of subjects who developed dose-limiting toxicity (DLT)
Safety Evaluation: Severity and frequency of reported adverse events
Time frame: Baseline to Week 52
Assess severity and frequency of reported adverse events
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.
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.
8 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Tae-gyun Kim contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IPS101A in people with Parkinson Disease and Parkinson's Disease, run by Innopeutics Corporation at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact ChoLong Park (+82-2-3499-4266, clpark@innopeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety Evaluation: dose-limiting toxicity (DLT) (time frame: Baseline to Week 8). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.