Actively Recruiting
A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
Led by He Huang · Updated on 2025-02-14
3
Participants Needed
1
Research Sites
422 weeks
Total Duration
On this page
Sponsors
H
He Huang
Lead Sponsor
L
Lingyi Biotech Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 60 years, male or female
- Understand the study purpose, methods, and possible side effects and voluntarily sign informed consent
- Confirmed diagnosis of Gaucher Disease Type I with double mutations in the Gba1 allele and enzyme activity less than 30% of normal
- Type I Gaucher Disease patients, with previous specific treatments completed at least 5 half-lives prior
- Negative pregnancy test for females of childbearing potential
- Willingness to avoid pregnancy during screening and for 6 months after study, using effective contraception
- No plans to donate sperm or eggs during the study
- Agree not to donate blood during the study and for at least 1 year after study completion
You will not qualify if you...
- Strongly positive for AAV8 neutralizing antibody
- Diagnosed or suspected Gaucher Disease type II or III
- Active, progressive bone disease likely requiring surgery within 6 months
- Presence of blood disorders, hepatomegaly, splenomegaly, or osteoporosis not caused by Gaucher Disease
- Use of investigational drugs or devices within 28 days or 5 half-lives before screening
- Significant liver disease or damage including specific liver volume, fibrosis, abnormal liver tests, recent alcohol or drug abuse, or hepatitis infection
- Positive for HIV or syphilis antibodies
- Severe hyperlipidemia (triglycerides > 1000 mg/dL)
- Uncontrolled or infectious diseases judged by investigator
- History of or planned splenectomy during study
- Karnofsky performance score below 70
- Previous or planned bone marrow, stem cell, or major organ transplantation
- Recent blood transfusions within specified timeframes
- Significant cardiovascular disease (e.g., NYHA class 3 or higher heart failure)
- Allergy to any component of LY-M001 Injection
- Previous gene or cell therapy
- Recent use of systemic immunosuppressive or steroid therapy not required by protocol
- Cancer history within 5 years except certain treated skin or prostate cancers
- Recent or planned live attenuated vaccine within 4 months before or during study
- Any other condition making the subject unsuitable according to investigator judgment
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Zhejiang University School of Medicine
Hanzhou, Zhejiang, China, 310003
Actively Recruiting
Research Team
H
He Huang, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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