Actively Recruiting
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Led by Lingyi Biotech Co., Ltd. · Updated on 2026-01-26
12
Participants Needed
3
Research Sites
368 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.
CONDITIONS
Official Title
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 60 years, male or female.
- Fully understand the study and voluntarily sign the informed consent form.
- Confirmed double mutations in the GBA1 gene with glucocerebrosidase activity less than 30% of normal.
- Either previously treated GD1 patients stable on enzyme replacement or substrate reduction therapy, or newly treated/untreated GD1 patients meeting criteria such as low hemoglobin, low platelets, hepatomegaly, or splenomegaly.
- Negative pregnancy test for women of childbearing potential.
- Subjects and partners agree to use effective contraception from screening to 6 months after study end and have no plans to donate sperm or eggs.
- No blood donation during the study and for at least 1 year after study completion.
You will not qualify if you...
- Positive for AAV8 neutralizing antibodies (titer > 1:40).
- Diagnosed with Gaucher disease type II or III.
- Active, progressive bone disease expected to require surgery within 6 months.
- Idiopathic thrombocytopenic purpura, thrombotic thrombocytopenic purpura, thrombocytopenia, anemia, hepatomegaly, splenomegaly, or osteoporosis unrelated to GD.
- Use of investigational drugs or devices within 28 days or 5 half-lives before screening.
- Significant liver disease or hepatotoxin exposure including large hepatomegaly, advanced fibrosis, elevated liver enzymes, recent alcohol or drug abuse, or active hepatitis B or C infection.
- Positive HIV or Treponema pallidum antibodies.
- Severe hyperlipidemia (triglycerides > 11.29 mmol/L).
- Uncontrolled infections or diseases.
- History or plans for major organ or bone marrow transplantation.
- Recent erythropoietin, red blood cell or platelet transfusions.
- Serious cardiovascular disease.
- Hypersensitivity to LY-M001 components.
- Previous gene or cell therapy.
- Recent or planned live attenuated vaccine administration.
- Other conditions judged unsuitable by the investigator.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Guangzhou First People's Hospital
Guangzhou, Guangdong, China, 510000
Actively Recruiting
2
Shanxi Bethune Hospital
Taiyuan, Shanxi, China, 030000
Actively Recruiting
3
Hematology Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China, 300011
Actively Recruiting
Research Team
Q
Qing Lin, PhD
CONTACT
Y
Yixiong Chen, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
4
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