A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gauc… (NCT06818838) | Clinical Trial Compass
RecruitingPhase 1/2
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
China12 participantsStarted 2024-07-05
Plain-language summary
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.
Who can participate
Age range18 Years – 60 Years
SexALL
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Inclusion criteria
✓. Age ≥ 18 years and ≤ 60 years, male or female.
✓. The subjects 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 spot (DBS) method is \< 1.19 μmol/L/h), and meeting the standard clinical diagnosis criteria for GD1.
✓. 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 before administration, or were comprehensively judged to be stable by the investigator.
✓. Newly treated or untreated GD1 patients who meet one or more of the following criteria at screening:
✓. Negative pregnancy test for female subjects 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 than menopause), and the absence of surgical (i.e., ovarian, salpingectomy, and/or hysterectomy) or Investigator-determined cause of permanent infertility due to other causes (e.g., lenticular hypoplasia) after menarche in female subjects.
What they're measuring
1
Phase I: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion
Timeframe: From enrollment to 52 weeks after administration
2
Phase I: Incidence rate of dose-limiting toxicity (DLT) events determined by the data safety review committee (SRC) within at least 28 days after LY-M001 infusion.
Timeframe: From enrollment to 52 weeks after administration
3
Phase I: Liver function levels (including alanine aminotransferase [ALT], aspartate aminotransferase [AST], total bilirubin [TBIL], alkaline phosphatase [ALP], gamma-glutamyl transferase [GGT]) within 52 weeks after LY-M001 infusion.
Timeframe: From enrollment to 52 weeks after administration
Timeframe: From enrollment to 52 weeks after administration
5
Phase II: The incidence rates of adverse events (AEs) and serious adverse events (SAEs), as well as the occurrence of abnormalities in 12-lead electrocardiogram (ECG) findings, vital signs, laboratory test parameters, and physical examination results.
Timeframe: From enrollment to 52 weeks after administration
✓. Subjects 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.); subjects have no plans to donate sperm or eggs.
✕. 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:
✕. Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
✕. Severe hyperlipidemia (triglycerides \> 11.29mol/L).