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New treatment option for Gaucher Disease Type 1
Official title A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
ClinicalTrials.gov ID: NCT06818838
What this study is testing
What is LY-M001?
LY-M001 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for gaucher disease type 1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 60
You may be able to join if
- Age ≥ 18 years and ≤ 60 years, male or female.
- The people should fully understand the purpose, nature, and method of this study as well as possible adverse reactions, and sign the informed consent...
- Patients with confirmed double mutations in the GBA1 allele through laboratory testing, and the glucocerebrosidase activity was reduced to less than...
- Patients who meet a) or b) below:
- Treated patients with Gaucher disease type I who had previously received enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) with...
You likely can't join if
- AAV8 neutralizing antibody positive (Antibody titer \> 1:40).
- Patients with clinically diagnosed Gaucher disease type II or III (GD2 or 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...
- Treatment or disposal of investigational drugs or investigational devices received in other clinical studies within 28 days prior to screening or...
- Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins that meets, but is not limited to, any of the...
See the full eligibility criteria
Who can join
- Age ≥ 18 years and ≤ 60 years, male or female.
- The people 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 confirmed double mutations in the GBA1 allele through laboratory testing, and the glucocerebrosidase activity was reduced to less than 30% of the normal value(For example, the result of the dried blood...
- Patients who meet a) or b) below:
- Treated patients with Gaucher disease type I who had previously received enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) with GD, were on stable medication, eluted 5 drugs for a half-life or more...
- Newly treated or untreated GD1 patients who meet one or more of the following criteria at screening:
- Hemoglobin ≥80g/L and less than the lower limit of normal;
- Platelets ≥40×10\^9/L and less than the lower limit of normal;
- Hepatomegaly;
- Splenomegaly.
- Negative pregnancy test for female people of childbearing potential (WOCBP). Notes: WOCBP is defined as the absence of postmenopausal status (continuous amenorrhea of at least 12 months with no identifiable cause other...
- people and their partners have no childbearing plans from the screening period to 6 months after the end of the study, and voluntarily adopt effective contraceptive measures (e.g., abstinence, condoms, etc.); people...
- people are not to donate blood during the study and for at least 1 year after the end of the study.
What rules you out
- AAV8 neutralizing antibody positive (Antibody titer \> 1:40).
- Patients with clinically diagnosed Gaucher disease type II or III (GD2 or 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 GD as judged by the Investigator.
- Treatment or disposal of investigational drugs or investigational devices received in other clinical studies within 28 days prior to screening or within 5 half-lives (drugs only), whichever is older.
- Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins that meets, but is not limited to, any of the following at the time of screening:
- Progressive hepatomegaly larger than 3 times the normal volume.
- History of stage 2 or above liver fibrosis.
- AST, ALT, or TBIL are 1.5 times higher than ULN.
- A history of alcohol or drug abuse within the previous 2 years (defined as having consumed more than 14 standard units of alcohol per week [1 standard unit containing 14 g of alcohol, such as 360 mL beer, 45 mL spirits...
- Hepatitis B surface antigen (HBsAg) positive and HBV deoxyribonucleic acid (HBV-DNA) positive (HBV-DNA\>10\^3 copy number /mL); Or take hepatitis B drugs (such as interferon, lamivudine, adefovir and entecavir); Or...
- Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
- Severe hyperlipidemia (triglycerides \> 11.29mol/L).
- Uncontrolled concomitant or infectious diseases (need to be determined by the investigator based on clinical practice).
- 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...
- 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 investigator-determined serious cardiovascular disease (such as heart failure ≥3 from the New York College of Cardiology [NYHA]).
- 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 prior to screening, or currently active neoplastic disease, except for basal or squamous cell carcinoma of the skin or carcinoma in situ that has been definitively treated.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Guangzhou, Guangdong, China
- Taiyuan, Shanxi, China
- Tianjin, Tianjin Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Guangzhou, Guangdong, China; Taiyuan, Shanxi, China; Tianjin, Tianjin Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.