The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.
Azita Nabizadeh, Bahman Amani, Maliheh Kadivar...
https://pubmed.ncbi.nlm.nih.gov/30622983Actively Recruiting
Phase Not Applicable
Age: 18Years - 60Years
All Genders
ID06162338
A Prospective Single-Center Study of LY-M001 Injection Safety and Preliminary Efficacy in Adults With Gaucher Disease Type I
Led by He Huang · Updated on 2025-02-14
3
Participants Needed
1
Research Sites
260 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 research aims to evaluate the safety, initial efficacy, pharmacodynamics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection in adult patients with Gaucher Disease Type I. The study is prospective, single-center, open-label, single-arm, and involves a single intravenous dose. The study includes a main phase and a long-term follow-up phase, focusing on adults aged 18 to 60 with confirmed Gaucher Disease Type I. Participants receive a single intravenous infusion of LY-M001 at a dose calculated based on their body weight, administered at a rate of 1 mL per minute. The initial dose group uses 5.0 × 10^12 vg/kg, starting with one subject observed for 28 days for safety before enrolling additional subjects. Dose adjustments may be made depending on safety and efficacy data. The study includes a screening period, baseline period, treatment and safety observation period, and a short-term follow-up lasting up to 38 weeks. Those completing the main study or withdrawing early may enter a long-term follow-up lasting up to five years. Throughout the study, participants undergo various assessments including monitoring for treatment-related adverse events and dose-limiting toxicities within 38 weeks post-infusion. Laboratory tests measure glucocerebrosidase protein levels and enzyme activity, as well as glucosylsphingosine in plasma. Participants complete all required evaluations at the end of the study or early withdrawal, and are monitored for safety and efficacy during both short-term and long-term follow-up periods.
CONDITIONS
Brief 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
Age: 18Years - 60Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 to 60 years, male or female
- Fully understand the study and voluntarily sign informed consent
- Confirmed double mutations in the Gba1 allele and clinical diagnosis of Gaucher Disease Type I with reduced GCase enzyme activity below 30% of normal
- Type I Gaucher disease patients, including those previously treated who completed 5 half-lives of treatment
- Negative pregnancy test for women of childbearing potential
- No plans for pregnancy or sperm/egg donation during the study and 6 months after, with effective contraception
- No blood donation during the study and for at least 1 year after study completion
You will not qualify if you...
- Strongly positive AAV8 neutralizing antibody
- Clinically suspected Gaucher Disease Type II or III
- Active, progressive bone disease expected to need surgery within 6 months
- Idiopathic thrombocytopenic purpura, thrombotic thrombocytopenic purpura, thrombocytopenia, anemia, hepatomegaly, splenomegaly, or osteoporosis unrelated to Gaucher Disease
- Recent investigational drug or device use within 28 days or 5 half-lives prior to screening
- Significant liver disease including hepatomegaly >3 times normal, stage 2+ fibrosis, elevated liver enzymes, recent alcohol or drug abuse, hepatitis B or C infection
- HIV or Treponema pallidum antibody positive
- Severe hyperlipidemia (triglycerides >1000 mg/dL)
- Uncontrolled diseases or infections
- History or planned splenectomy during study
- Karnofsky score below 70
- Previous or planned bone marrow, stem cell, or major organ transplantation
- Recent blood transfusions
- Significant cardiovascular disease (NYHA class 3 or higher heart failure)
- Hypersensitivity to LY-M001 components
- Prior gene or cell therapy
- Recent systemic immunosuppressive or steroid use
- Cancer history within 5 years except certain treated types
- Recent or planned live attenuated vaccine
- Other conditions making participation unsuitable as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 1 day with 28 days safety observation
Participants receive a single intravenous infusion of LY-M001 Injection. Dosage is calculated based on body weight and administered at a rate of 1 mL per minute. The first participant in the initial dose group is observed for dose-limiting toxicity for at least 28 days before enrolling the next participant.
1 treatment visit and weekly safety observation visits for 4 weeks
Short-term Follow-up
Duration - Approximately 9 weeks (from week 5 to week 38)
Participants undergo follow-up assessments to monitor safety and preliminary efficacy after treatment.
Periodic follow-up visits during weeks 5 to 38
Long-term Follow-up
Duration - Up to 5 years
Participants who complete or withdraw early from the main study enter a long-term follow-up period to monitor safety and outcomes for up to 5 years.
Visits as scheduled during long-term follow-up
Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Zhejiang University School of Medicine
Hanzhou, Zhejiang, China, 310003
Actively Recruiting
Loading map...
Research Team
H
He Huang, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Position statement: National Gaucher Foundation Medical Advisory Board, January 7, 2014.
John A Barranger, Roscoe O Brady, Gregory A Grabowski...
https://pubmed.ncbi.nlm.nih.gov/24488939Effect of velaglucerase alfa enzyme replacement therapy on red blood cell properties in Gaucher disease.
Melanie Franco, Nelly Reihani, Mickael Marin...
https://pubmed.ncbi.nlm.nih.gov/28621801Open-label, expanded access study of taliglucerase alfa in patients with Gaucher disease requiring enzyme replacement therapy.
David J Kuter, Michael Wajnrajch, Betina Hernandez...
https://pubmed.ncbi.nlm.nih.gov/32146279Pharmacotherapy of Gaucher Disease: Current and Future Options.
Lunawati L Bennett, Chris Fellner
https://pubmed.ncbi.nlm.nih.gov/29719368Allogenic bone marrow transplantation in severe Gaucher disease.
P Tsai, J M Lipton, I Sahdev...
https://pubmed.ncbi.nlm.nih.gov/1603628BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Barbara A Konkle, Christopher E Walsh, Miguel A Escobar...
https://pubmed.ncbi.nlm.nih.gov/33067633Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Amit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham...
https://pubmed.ncbi.nlm.nih.gov/25409372Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease.
You-Hai Xu, Brian Quinn, David Witte...
https://pubmed.ncbi.nlm.nih.gov/14578207Substrate compositional variation with tissue/region and Gba1 mutations in mouse models--implications for Gaucher disease.
Ying Sun, Wujuan Zhang, You-Hai Xu...
https://pubmed.ncbi.nlm.nih.gov/23520473