Actively Recruiting
A Multicenter, Open, Single-Arm, Single-Dose, Dose-Escalation and Expanded Phase I/II Study Evaluating Safety, Tolerability, and Efficacy of LY-M001 Injection in Adult Type I Gaucher Disease
Led by Lingyi Biotech Co., Ltd. · Updated on 2026-01-26
12
Participants Needed
3
Research Sites
208 weeks
Total Duration
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AI-Summary
What this Trial Is About
Gaucher disease type 1 (GD1) is caused by mutations in the GBA1 gene, leading to a deficiency in the enzyme glucocerebrosidase (GCase). This deficiency results in the buildup of glucosylceramide (GlcCer) in various organs, including the liver, spleen, kidney, bone, lung, and brain, causing cells to change and leading to tissue and organ problems. Researchers are evaluating LY-M001, a gene therapy using an rAAV8 vector, which aims to deliver the GCase protein to liver cells after a single intravenous infusion to address this enzyme deficiency. This clinical trial includes two phases: Phase I is a dose escalation study with three dose groups, starting at a lower dose and increasing to higher doses to assess safety. Participants receive a single intravenous infusion of LY-M001 at one of the specified doses. Phase II is a dose expansion study where the recommended dose from Phase I is given to more participants to further evaluate safety and efficacy. The study has a main period of 52 weeks following infusion and a long-term follow-up phase lasting from 53 weeks up to 5 years. Participants will undergo regular assessments including monitoring for adverse events, liver function tests, blood enzyme activity, blood counts, imaging of liver and spleen volumes, bone mineral density, and bone marrow evaluation. Safety evaluations also include electrocardiograms, vital signs, and laboratory tests. The study aims to track the effects of LY-M001 over time, with extended follow-up to gather long-term data on safety and treatment impact throughout the study duration.
CONDITIONS
Brief 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 18 years to 60 years, male or female.
- Understands the study purpose and procedures and voluntarily signs informed consent.
- Confirmed double mutations in GBA1 allele with glucocerebrosidase activity less than 30% of normal.
- Either stable treated patients on enzyme replacement or substrate reduction therapy, or newly treated/untreated patients meeting criteria such as low hemoglobin, low platelets, hepatomegaly, or splenomegaly.
- Negative pregnancy test for women of childbearing potential.
- No plans for pregnancy or sperm/egg donation during the study and 6 months after.
- No blood donation during the study and for at least 1 year after study end.
You will not qualify if you...
- Positive for AAV8 neutralizing antibody (titer > 1:40).
- Diagnosis of Gaucher disease type II or III.
- Active progressive bone disease likely needing surgery within 6 months.
- Certain blood, liver, or bone conditions unrelated to Gaucher disease.
- Recent participation in other clinical trials or investigational treatments within specified timeframes.
- Significant liver disease or history of liver damage.
- Positive tests for HIV, hepatitis B or C, or history of substance abuse.
- Severe hyperlipidemia.
- Uncontrolled infections or diseases.
- History or plans for bone marrow, stem cell, or major organ transplant.
- Recent blood transfusions.
- Significant cardiovascular disease.
- Allergy to LY-M001 components.
- Prior gene or cell therapy.
- Recent use of systemic immunosuppressants or steroids.
- Recent or planned live attenuated vaccination.
- Other conditions making participation unsuitable as judged by the investigator.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks after LY-M001 infusion
Participants receive a single intravenous infusion of LY-M001 at assigned dose levels during Phase I dose escalation and Phase II dose expansion to evaluate safety, tolerability, and efficacy.
Multiple visits for safety and efficacy assessments over 52 weeks
Duration - 53 weeks to 5 years after LY-M001 infusion
Participants who complete or withdraw early from the 52-week treatment phase enter long-term follow-up for extended safety and efficacy assessments.
Periodic visits during long-term follow-up
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
Y
Yixiong Chen, PhD
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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