ClinicalTrials.gov record NCT06162338
A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
A Prospective, Single-center, Open-arm, Single-arm Study of the Safety and Preliminary Efficacy of Single Intravenous Infusion Administration of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label, single-arm study gives a single intravenous infusion of LY-M001 to adults with Gaucher disease type I. Up to three adults are planned in preset dose cohorts; the first cohort uses a sentinel approach with a 28-day safety (dose‑limiting toxicity) observation before the next subject. Short-term follow-up continues to about 38 weeks, and main plus long-term follow-up lasts up to five years.
What it is studying. The study is testing the safety and initial (preliminary) efficacy of one IV dose of LY-M001 in adults with Gaucher disease type I. It also measures pharmacodynamic effects, immunogenicity (immune response to the product), biodistribution (where the product goes in the body), viral shedding, and changes in GCase protein/activity and plasma Lyso-GL1.
What the researchers hope to learn. To evaluate safety (adverse events and dose-limiting toxicity) and gather preliminary efficacy data. To characterize pharmacodynamic effects, immunogenicity, biodistribution, and viral shedding. To measure changes in plasma GCase protein/activity and Lyso-GL1 levels.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- LY-M001 Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Age ≥ 18 years and ≤ 60 years, male or female.
- The subjects should fully understand the purpose, nature and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily.
- Patients with GD1 who have confirmed double mutations in the Gba1 allele by laboratory testing and meet the standard for clinical diagnosis of Gaucher disease (i.e., reduced GCase enzyme activity to less than 30% of normal values).
- The subjects were type I patients with Gaucher disease. Patients with type I Gaucher disease who had received specific treatment in the past required 5 half-lives of elution
- Negative pregnancy test for female subjects of childbearing potential 6.6.The subject and his/her partner have no plans to have children during the screening period and within 6 months after the end of the study, and voluntarily take effective contraceptive measures (such as abstinence, condom, etc.); and the subject had no plans to donate sperm or eggs.
- Subjects are not to donate blood during the study and for at least 1 year after the end of the study.
Exclusion criteria
- AAV8 neutralizing antibody is strongly positive.
- Patients with clinically suspected Gaucher disease type II (GD2) or Gaucher disease type III (GD3).
- Active and progressive bone disease that is expected to require surgical treatment within the next 6 months.
- Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and/or osteoporosis unrelated to Gaucher disease as judged by the investigator.
- Treatment or disposition of an investigational drug or investigational device for another clinical investigation within 28 days or 5 half-lives (only for drugs), whichever is longer, prior to Screening.
- Evidence of clinically significant liver disease, fragile liver, or history of hepatotoxin exposure, meeting at screening, but not limited to, any of the following:
- Progressive hepatomegaly and greater than 3 times normal volume.
- History of stage 2 or greater liver fibrosis.
- AST, ALT or TBIL greater than 1.5 times the upper limit of normal (ULN).
- History of alcohol or drug abuse within the previous 2 years.
- Hepatitis B surface antigen (HBsAg) positive, and hepatitis B virus DNA positive (HBV-DNA > 103 copies/mL); or taking hepatitis B virus drugs (such as interferon, lamivudine, adefovir and entecavir); or hepatitis C virus (HCV) antibody positive.
- Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
- Severe hyperlipidemia (triglycerides > 1000 mg/dL).
- Uncontrolled concomitant disease or infectious disease (need to be judged by the investigator based on clinical practice).
- Subject had undergone splenectomy and were scheduled to undergo splenectomy during the study period.
- Karnofsky score (KPS) < 70.
- The subject has received or plans to receive bone marrow transplantation, hematopoietic stem cell transplantation and/or major organ transplantation, including but not limited to liver transplantation, kidney transplantation, etc.
- Subject has received erythropoietin, transfusion, or red blood cell transfusion within 3 months prior to screening ;or platelet transfusion within 1 month prior to screening.
- Clinically diagnosed or significant cardiovascular disease as judged by the investigator (e.g., New York Heart Association [NYHA] class ≥ 3 heart failure).
- Hypersensitivity to any component of LY-M001 Injection.
- Previous treatment with any type of gene therapy or cell therapy.
- Use of systemic immunosuppressive agents or steroid therapy other than those required by the protocol for prophylactic administration within 3 months prior to dosing.
- History of cancer within 5 years of screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer, and completely treated ductal carcinoma in situ.
- Has received a live attenuated vaccine within 4 months prior to screening or plans to receive a live attenuated vaccine during the clinical trial.
- Other conditions that, in the opinion of the investigator, make the subject unsuitable for the study.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This study included the screening period (weeks -8 to days -2), the baseline period (days -1), the treatment and safety observation period (days 0 to 28), and the short-term follow-up period (weeks 5 [from day 29] to week 38). Participants eligible for the screening period will be admitted to the study center for a single LY-M001 treatment and a short-term follow-up period after the end of the treatment and safety observation period. Participants who complete all follow-up during the main study phase or who withdraw early from the study are required to complete all assessments required for the End of Study (EOS) visit.The study included up to three adult Gaucher disease type I subjects at preset dose group levels.With 5.0 × 1012 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity [DLT] observation period) to enroll the next subject. Participants enrolled in the long-term follow-up study were those who completed the main study or withdrew early, and the duration of the long-term follow-up study and the main study lasted for a total of 5 years.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After screening and a baseline visit, participants receive a single weight-based intravenous infusion of LY‑M001 given at about 1 mL per minute; the first person in a dose group is observed for at least 28 days before the next is dosed. Safety observation is intensive in the first 28 days, with additional short-term follow-up visits through about 38 weeks and long-term follow-up so total follow-up can last up to 5 years. Participants will have repeated safety checks and laboratory assessments (including measures of GCase protein and activity, Lyso‑GL1, immune response and viral shedding) and will be asked about adverse events. Participants must follow pregnancy testing and contraception rules and must not donate blood for at least one year after the study.
How the study is structured. Interventional treatment study: open-label, single-group (single-arm). Participants receive a single weight-based IV infusion of LY-M001 and are observed in a safety period (days 0–28), followed by short-term follow-up to about 38 weeks and long-term follow-up so that the total follow-up can reach five years. The first dose cohort enrolls 1–2 subjects by a sentinel method; dosing may be adjusted after safety review.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Gaucher disease
Important dates
- Study start
- Nov 15, 2023
- Primary completion
- Dec 17, 2026 (estimated)
- Study completion
- Dec 17, 2031 (estimated)
- Target enrollment
- 3 participants (estimated)
- Last updated on registry
- Feb 14, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- He Huang
- Collaborators
- Lingyi Biotech Co., Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- He Huang, PhD 📞 0571-87236703 ✉ hehuangyu@126.com
Participating sites (1)
- The First Affiliated Hospital of Zhejiang University School of MedicineRecruiting
Hanzhou, Zhejiang, 310003 · China
- He Huang, PhD · 0571-87236703 · hehuangyu@126.com
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- pharmacodynamic
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- long-term follow-up
- Observation that continues for years after the study treatment, often required for gene and cell therapies.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- screening period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- investigational drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- Karnofsky
- A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- DLT
- A side effect, defined in advance, that is severe enough to limit how far the dose can be raised in a dose-escalation study. How many such events stop escalation is set by the study's rules.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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