Parkinson's Disease (PD)Early Phase 1NCT07685444
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Injection of LY-N001 Injection for the Treatment of Moderate to Advanced Parkinson's Disease With GBA1 Mutations
A Early Phase 1 study testing LY-N001 Injection in people with Parkinson's Disease (PD), run by Lingyi Biotech Co., Ltd. at 1 site in China.
Trial summary
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. Read more about this study
- Who is it for?
- Ages 30 to 70 years
- Conditions the study is for
- Parkinson's Disease (PD)
- What is being tested?
- LY-N001 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 7 Jul 2026to 30 Dec 2032
- Phase
- Early 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 30 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 30 to 70 years inclusive; both males and females are eligible.
- The participant fully understands the purpose, nature, procedures of the study and potential adverse events, voluntarily agrees to take part in the study and signs the informed consent form (ICF).
- Participants with clinically diagnosed idiopathic Parkinson's disease (PD) meeting the 2015 Movement Disorder Society (MDS) diagnostic criteria for idiopathic PD.
- Participants carrying at least one pathogenic GBA1 variant.
- Duration of PD disease ≥ 3 years.
- Off-state Hoehn-Yahr stage ranges from 2.5 to 4.
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- Neutralizing antibody titer against AAV ≤ 1:200.
- The off-state MDS-UPDRS Part III (see Appendix 6 for MDS-UPDRS Part III motor examination) score is greater than 25, with either a ≥30% improvement rate on the acute levodopa challenge test, or an average daily off time of ≥2.5 hours over 3 consecutive days.
- Investigators determined that patients must receive a stable dose of dopaminergic medications, including levodopa, for a minimum of 4 weeks prior to surgery.
- Acceptable laboratory test values shall be obtained during the screening period and prior to administration (Day -3)：a).Hemoglobin ≥ 100 g/L; b).Platelets ≥ 100 × 10⁹/L; c). Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) ≤ 2 × upper limit of normal (ULN); d).Total bilirubin ≤ 2 × upper limit of normal (ULN); e).Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min.
- Demonstrates good treatment adherence and ability to attend scheduled follow-up visits; the participant shall be capable of accurately completing the Parkinson's disease (PD) diary during follow-up, or the participant's family member, legal guardian or caregiver may assist with diary completion.
- The participant agrees to postpone any other planned elective neurosurgical procedures (excluding emergent life-threatening neurosurgical operations occurring during the study), including Deep Brain Stimulation (DBS), until completion of the 52-week follow-up period, unless DBS treatment is recommended by a specialist in the participant's best interest.
- The participant agrees not to participate in any other therapeutic intervention studies during the study period and shall not take any other medications that may interfere with treatment other than those specified in the protocol.
- The participant agrees to refrain from receiving any vaccinations within 30 days after surgery.
- The participant must be willing to refrain from donating blood, organs, tissues or cells at any time following treatment administration.
- Female participants of childbearing potential (WOCBP) must have a negative pregnancy test.
- The participant and their partner have no plans to conceive from screening through 6 months after study completion, and will voluntarily use effective contraceptive measures (such as abstinence, condoms, etc.); the participant has no intention to donate sperm or ova.
Who cannot join
- Atypical or secondary parkinsonism (including Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
- Patients with clinically confirmed LRRK2 mutation.
- Previous history of pallidotomy, Deep Brain Stimulation (DBS) surgery, striatal surgery, extrapyramidal surgery, stereotactic brain surgery or other brain surgeries; or any other surgical history judged by the investigators to interfere with the subject's participation in this study.
- Participants with previous head Computed Tomography (CT) / Magnetic Resonance Imaging (MRI) showing neurological diseases such as brain trauma, vascular malformations, hydrocephalus, brain tumors; subjects with hyperintense white matter signals on MRI indicating risk of intracranial hemorrhage; subjects with cerebrovascular lesions or microbleeds on SWI (Susceptibility-Weighted Imaging); subjects with vulnerable plaques or intraplaque hemorrhage of craniocervical arteries on HR-MRI (High-Resolution Magnetic Resonance Imaging); or subjects with imaging abnormalities in the striatum or other brain regions that substantially increase surgical risks.
- The participant has a Mini-Mental State Examination (MMSE) score of less than 20.
- A Patient Health Questionnaire (PHQ) score of ≥16 indicates severe depression.
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- Any severe psychiatric illnesses including epilepsy, severe anxiety, schizophrenia that are deemed unsuitable for participation in this study by the investigators, excluding mild hallucinations and similar symptoms induced by anti-Parkinsonian medications.
- Patients taking anticoagulants or antiplatelet drugs whose coagulation function has not returned to normal 10 days after drug discontinuation.
- Patients who have previously received gene therapy or cell therapy.
- Use of systemic glucocorticoids or immunosuppressive drugs within 12 weeks prior to administration, except for prophylactic immunosuppressive agents required by the protocol (excluding prophylactic immunosuppressive therapy specified in the protocol).
- Participants with unstable cardiovascular and cerebrovascular diseases, defined as clinical cardiovascular and cerebrovascular events occurring within 3 months prior to screening (e.g., unstable angina, myocardial infarction, stroke, etc.); uncontrolled diabetes mellitus with glycated hemoglobin >8.0%; or other unstable acute or chronic diseases including infectious diseases, severe uncontrolled hypertension (systolic blood pressure ≥160 mmHg and/or diastolic blood pressure ≥100 mmHg), or severe orthostatic hypotension (a drop in systolic blood pressure ≥20 mmHg or diastolic blood pressure ≥10 mmHg within 3 minutes after the participant changes from supine/squatting position to standing position).
- Positive Hepatitis B surface antigen (HBsAg) or Hepatitis B Virus Deoxyribonucleic Acid (HBV-DNA); positive Hepatitis C Virus antibody (HCV-Ab) or Hepatitis C Virus Ribonucleic Acid (HCV-RNA).For participants with a prior history of hepatitis B or hepatitis C, two samples collected at an interval of at least 3 months must test negative for all above indicators to be considered negative. Participants with spontaneously cleared or antivirally treated cleared hepatitis B or C are eligible for this study.Participants with positive Human Immunodeficiency Virus (HIV) test or positive syphilis serology.
- Participants with surgical contraindications, those who have received other surgical procedures deemed to interfere with this study by the investigators within six months, or those with other contraindications to neurosurgery.
- Participants with an average weekly alcohol intake exceeding 21 units for males or 14 units for females within 30 days prior to screening; 1 alcohol unit equals one 5 oz (150 mL) glass of red wine, 12 oz (360 mL) beer, or 1.5 oz (45 mL) distilled spirits; or participants with a history of drug dependence.
- Participants with contrast medium allergy who cannot undergo MRI, or those unable to tolerate surgical anesthesia.
- Participants with hypersensitivity to LY-N001 Injection.
- History of cancer within 5 years prior to screening, excluding completely resected non-melanoma skin cancer, non-metastatic prostate cancer, fully cured carcinoma in situ, and papillary thyroid carcinoma cured after radical surgical resection.
- Pregnant women, women planning pregnancy, or breastfeeding women.
- Participants who are currently enrolled in another clinical trial, or those who participated in another clinical trial and received investigational products within one month.
- Participants who are deemed ineligible for enrollment upon investigator's assessment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022
Yi Wang, PhD, +86 15056000767, yfy1082230@fy.ahmu.edu.cn
Yi Wang, PhD
About this study
Official title: A Prospective, Single-Arm, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Administration of LY-N001 Injection in Subjects With Moderate to Advanced Parkinson's Disease Carrying GBA1 Mutations
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Read the detailed description
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Three dose cohorts are pre-specified in this study, including a de-escalation dose cohort (0.5 × 10¹¹ vg/g brain weight), a low-dose cohort (1.0 × 10¹¹ vg/g brain weight), and a high-dose cohort (2.0 × 10¹¹ vg/g brain weight). The low-dose cohort serves as the starting dose of this study, and the dose design is presented in Table 1. The first participant will be enrolled at the starting dose of 1.0 × 10¹¹ vg/g brain weight. Subsequent participants will be enrolled only after safety confirmation following the DLT observation period.
- Conditions
- Parkinson's Disease (PD)
- Being tested
- LY-N001 Injection
- Keywords
- gene therapy, GBA1-mutant Parkinson's disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY-N001 InjectionGenetic
LY-N001 Injection shall be administered as a single intracerebroventricular (ICV) injection, with one administration only
| Study group | Role | What they receive |
|---|---|---|
| LY-N001 Dose Cohort 1 | Experimental | LY-N001 is administered via a single intracerebral injection at Dose Cohort 1. Receives: Genetic: LY-N001 Injection |
| LY-M003 Dose Cohort 2 | Experimental | LY-N001 is administered via a single intracerebral injection at Dose Cohort 2. Receives: Genetic: LY-N001 Injection |
| LY-N001 Dose De-escalation Cohort | Experimental | LY-N001 is administered via a single intracerebral injection at Dose De-escalation Cohort Receives: Genetic: LY-N001 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicity (DLT) events occurring within at least 28 days following a single intracranial administration of LY-N001
DLT events will be assessed per CTCAE Version 6.0.
Measured: Within 28 days post-administration
Incidence of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) occurring during the treatment period
All AEs and SAEs will be graded per CTCAE Version 6.0 and adjudicated in accordance with SAE criteria.
Measured: Within 52 weeks post-administration
Secondary outcomes (14)
Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part III (MDS-UPDRS-III) motor examination scores during OFF and ON medication states
Efficacy was assessed via the change from baseline in the MDS-UPDRS Part III total score. The maximum possible total score of the scale is 132, and higher scores correspond to greater disease severity.
Measured: Within 52 weeks post-administration
Parkinson's disease medication use: changes in daily oral levodopa (L-DOPA) dose or levodopa equivalent dose (LED)
Assessment was based on the change from baseline in daily Parkinson's disease medication usage.
Measured: within 52 weeks post-administration
Motor fluctuations assessed via patient diaries: changes in "ON" time without dyskinesia or without troublesome dyskinesia, and changes in "OFF" time
Assessment was conducted based on changes in ON/OFF time recorded in patient diaries.
Measured: Within 52 weeks post-administration
Changes in scores of the MDS Unified Parkinson's Disease Rating Scale (MDS-UPDRS Part I, MDS-UPDRS Part II and MDS-UPDRS Part IV)
Assessment was performed based on the scores of MDS-UPDRS Part I, MDS-UPDRS Part II and MDS-UPDRS Part IV.The maximum total scores of MDS-UPDRS Part I, Part II and Part IV are 52, 52 and 24 respectively. Higher scores indicate more severe disease.
Measured: Within 52 weeks post-administration
Change from baseline in Mini-mental State Examination (MMSE) score
Assessment will be performed based on the change from baseline in MMSE score. The total maximum score of MMSE is 30, and lower scores indicate more severe cognitive impairment.
Measured: Within 52 weeks post-administration
Change from baseline in Montreal Cognitive Assessment (MoCA) score
Assessment will be evaluated based on the change from baseline in MoCA score. The total maximum score of MoCA is 30, with lower scores indicating more severe cognitive impairment.
Measured: Within 52 weeks post-administration
Change from baseline in Geriatric Depression Scale (GDS) score
Assessment will be conducted based on the change from baseline in total GDS score. The maximum total score of GDS is 15, and higher scores represent more severe depressive symptoms.
Measured: Within 52 weeks post-administration
Change from baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2) score
Assessment will be performed based on the change from baseline in total PDSS-2 score. The maximum total score of PDSS-2 is 150, and higher scores indicate more severe Parkinson's disease-related sleep disturbances.
Measured: Within 52 weeks post-administration
Change from baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) score
Assessment will be evaluated by the change from baseline in total PDQ-39 score. The maximum total score of PDQ-39 is 156, and higher scores mean more severe impairment of Parkinson's disease-related quality of life.
Measured: Within 52 weeks post-administration
Change from baseline in Dopamine Transporter Positron Emission Tomography (DAT-PET) and Fluorodeoxyglucose Positron Emission Tomography (FDG-PET) parameters.
Assessment will be performed via DAT-PET and FDG-PET examinations.
Measured: Within 52 weeks post-administration
Change from baseline in blood glucocerebrosidase (GCase) activity level
Assessment will be conducted based on the change from baseline in blood GCase test results.
Measured: Within 52 weeks post-administration
Change from baseline in blood lyso-glucosylsphingosine (Lyso-GL1) levels
Assessment will be conducted based on the change from baseline in Lyso-GL1 test results.
Measured: Within 52 weeks post-administration
Change from baseline in cerebrospinal fluid glucocerebrosidase (GCase) activity levels
Assessment will be performed based on the change from baseline in cerebrospinal fluid GCase activity levels.
Measured: Within 52 weeks post-administration
Change from baseline in cerebrospinal fluid lyso-glucosylsphingosine (Lyso-GL1) levels
Assessment will be conducted based on the change from baseline in cerebrospinal fluid Lyso-GL1 test results.
Measured: Within 52 weeks post-administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2026
- Study start7 Jul 2026 (estimated)
- Primary completion7 Jul 2028 (estimated)
- Study completion30 Dec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's Disease (PD)Studies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lingyi Biotech Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yi Wang, PhD, Principal investigator, The First Affiliated Hospital of Anhui Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07685444
- Sponsor's protocol ID
- LY-N001-PD-IIT-001-1
- Status last verified
- Jun 2026
- First submitted
- 29 Jun 2026
- Last updated
- 6 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-Arm, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Administration of LY-N001 Injection in Subjects With Moderate to Advanced Parkinson's Disease Carrying GBA1 Mutations
- ClinicalTrials.gov ID
- NCT07685444
- Acronym / sponsor ID
- LY-N001-PD-IIT-001-1
- Conditions
- Parkinson's Disease (PD)
- Keywords
- gene therapy, GBA1-mutant Parkinson's disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Lingyi Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 7 Jul 2026 (estimated)
- Primary completion
- 7 Jul 2028 (estimated)
- Study completion
- 30 Dec 2032 (estimated)
- First posted
- 6 Jul 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.
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Detailed description
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Three dose cohorts are pre-specified in this study, including a de-escalation dose cohort (0.5 × 10¹¹ vg/g brain weight), a low-dose cohort (1.0 × 10¹¹ vg/g brain weight), and a high-dose cohort (2.0 × 10¹¹ vg/g brain weight). The low-dose cohort serves as the starting dose of this study, and the dose design is presented in Table 1. The first participant will be enrolled at the starting dose of 1.0 × 10¹¹ vg/g brain weight. Subsequent participants will be enrolled only after safety confirmation following the DLT observation period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| LY-N001 Dose Cohort 1 | Experimental | Genetic: LY-N001 InjectionLY-N001 is administered via a single intracerebral injection at Dose Cohort 1. |
| LY-M003 Dose Cohort 2 | Experimental | Genetic: LY-N001 InjectionLY-N001 is administered via a single intracerebral injection at Dose Cohort 2. |
| LY-N001 Dose De-escalation Cohort | Experimental | Genetic: LY-N001 InjectionLY-N001 is administered via a single intracerebral injection at Dose De-escalation Cohort |
Who may qualify
- Ages
- 30 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Aged 30 to 70 years inclusive; both males and females are eligible.
- The participant fully understands the purpose, nature, procedures of the study and potential adverse events, voluntarily agrees to take part in the study and signs the informed consent form (ICF).
- Participants with clinically diagnosed idiopathic Parkinson's disease (PD) meeting the 2015 Movement Disorder Society (MDS) diagnostic criteria for idiopathic PD.
- Participants carrying at least one pathogenic GBA1 variant.
- Duration of PD disease ≥ 3 years.
Key exclusion criteria (20 listed)
- Atypical or secondary parkinsonism (including Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
- Patients with clinically confirmed LRRK2 mutation.
- Previous history of pallidotomy, Deep Brain Stimulation (DBS) surgery, striatal surgery, extrapyramidal surgery, stereotactic brain surgery or other brain surgeries; or any other surgical history judged by the investigators to interfere with the subject's participation in this study.
- Participants with previous head Computed Tomography (CT) / Magnetic Resonance Imaging (MRI) showing neurological diseases such as brain trauma, vascular malformations, hydrocephalus, brain tumors; subjects with hyperintense white matter signals on MRI indicating risk of intracranial hemorrhage; subjects with cerebrovascular lesions or microbleeds on SWI (Susceptibility-Weighted Imaging); subjects with vulnerable plaques or intraplaque hemorrhage of craniocervical arteries on HR-MRI (High-Resolution Magnetic Resonance Imaging); or subjects with imaging abnormalities in the striatum or other brain regions that substantially increase surgical risks.
- The participant has a Mini-Mental State Examination (MMSE) score of less than 20.
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 |
|---|---|---|
| LY-N001 InjectionLY-N001 Injection shall be administered as a single intracerebroventricular (ICV) injection, with one administration only | Genetic | LY-M003 Dose Cohort 2, LY-N001 Dose Cohort 1, LY-N001 Dose De-escalation Cohort |
Primary outcome
Incidence of dose-limiting toxicity (DLT) events occurring within at least 28 days following a single intracranial administration of LY-N001
Time frame: Within 28 days post-administration
DLT events will be assessed per CTCAE Version 6.0.
Incidence of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) occurring during the treatment period
Time frame: Within 52 weeks post-administration
All AEs and SAEs will be graded per CTCAE Version 6.0 and adjudicated in accordance with SAE criteria.
14 secondary outcomes
Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part III (MDS-UPDRS-III) motor examination scores during OFF and ON medication states
Time frame: Within 52 weeks post-administration
Parkinson's disease medication use: changes in daily oral levodopa (L-DOPA) dose or levodopa equivalent dose (LED)
Time frame: within 52 weeks post-administration
Motor fluctuations assessed via patient diaries: changes in "ON" time without dyskinesia or without troublesome dyskinesia, and changes in "OFF" time
Time frame: Within 52 weeks post-administration
Changes in scores of the MDS Unified Parkinson's Disease Rating Scale (MDS-UPDRS Part I, MDS-UPDRS Part II and MDS-UPDRS Part IV)
Time frame: Within 52 weeks post-administration
Change from baseline in Mini-mental State Examination (MMSE) score
Time frame: Within 52 weeks post-administration
Change from baseline in Montreal Cognitive Assessment (MoCA) score
Time frame: Within 52 weeks post-administration
Change from baseline in Geriatric Depression Scale (GDS) score
Time frame: Within 52 weeks post-administration
Change from baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2) score
Time frame: Within 52 weeks post-administration
Change from baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) score
Time frame: Within 52 weeks post-administration
Change from baseline in Dopamine Transporter Positron Emission Tomography (DAT-PET) and Fluorodeoxyglucose Positron Emission Tomography (FDG-PET) parameters.
Time frame: Within 52 weeks post-administration
Change from baseline in blood glucocerebrosidase (GCase) activity level
Time frame: Within 52 weeks post-administration
Change from baseline in blood lyso-glucosylsphingosine (Lyso-GL1) levels
Time frame: Within 52 weeks post-administration
Change from baseline in cerebrospinal fluid glucocerebrosidase (GCase) activity levels
Time frame: Within 52 weeks post-administration
Change from baseline in cerebrospinal fluid lyso-glucosylsphingosine (Lyso-GL1) levels
Time frame: Within 52 weeks post-administration
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 6 Jul 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.
Yi Wang, PhD principal investigator
The First Affiliated Hospital of Anhui Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing LY-N001 Injection in people with Parkinson's Disease (PD), run by Lingyi Biotech Co., Ltd. at 1 site in China.
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 17 inclusion and 20 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: opening soon, as of the sponsor's last update on 6 Jul 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 China. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Rihong He, BD (+8619121572057, rihong.he@lingyimed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose-limiting toxicity (DLT) events occurring within at least 28 days following a single intracranial administration of LY-N001 (time frame: Within 28 days post-administration). The study lists 2 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 6 Jul 2026.