A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease (NCT06528080) | Clinical Trial Compass
Active — Not RecruitingEarly Phase 1
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease
China9 participantsStarted 2024-08-01
Plain-language summary
The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 6 years ≤ age \< 18 years. This study mainly includes the main study stage and the long-term follow-up study stage.
Who can participate
Age range6 Years – 18 Years
SexALL
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Inclusion criteria
✓. The subject and/or parent, caregiver, or legal representative must be willing and able to provide written informed consent/consent for the study in accordance with applicable regulations and guidelines and comply with all study access and procedures, including the use of any data collection devices that can be used to directly record participant data.
✓. Gender is not limited, 6 years old ≤ 18 years old.
✓. Patients with double allele mutation of glucocerebrosidase gene (GBA1) and decreased glucocerebrosidase activity were confirmed by laboratory tests and met the clinical manifestations of type I Gaucher disease.
✓. Subjects were newly treated or treated patients with type I Gaucher disease; For patients treated with enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) before screening, 5 drug half-lives are required before administration.
✓. The subject is willing to participate in all study follow-up and comply with all study procedures and evaluations.
✓. The subject must be willing to refrain from donating blood, organs, tissues, or cells at any time after receiving treatment.
✓. Pregnant Women (WOCBP) subjects tested negative for pregnancy.
✕. Patients with type II or III Gaucher disease (GD2 or GD3), or with suspected Gaucher disease as assessed by the investigator (e.g., subjects with Gaucher disease-related central nervous system manifestations or abnormal electroencephalogram \[EEG\] examination).
What they're measuring
1
Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion
Timeframe: Within 52 weeks of infusion
2
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: Within 52 weeks of infusion
3
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: Within 52 weeks of infusion
Trial details
NCT IDNCT06528080
SponsorShanghai Jiao Tong University School of Medicine
✕. Active and progressive bone diseases that are expected to require surgical treatment within the next 6 months.
✕. The subjects were judged by the investigator to have idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomeglia, splenomeglia, and/or osteoporosis unrelated to GD (bone mineral density z-score ±2).
✕. Treatment with an investigational drug in another clinical study within 28 days prior to screening or 5 half-lives, whichever is older.
✕. Evidence of a history of clinically significant liver disease or hepatotoxin exposure that meets, but is not limited to, any of the following at the time of screening:
✕. The subject's blood indicators have any of the following: