At a glance
ClinicalIndex Comparison Record- ✓Aged 18 to 75 years inclusive
- ✓Meets diagnostic criteria for familial hypercholesterolemia
- ✓Mutation in PCSK9 and/or ApoB and/or LDLR gene at screening
- ✓Weight ≥40kg and BMI >18 kg/m²
- ✕Prescription drugs affecting lipid metabolism (other than statins) within 14 days or 5 half-lives or 2 months for PCSK9 inhibitors or 1 year for PCSK9 small nucleic acid drugs
- ✕Non-prescription drugs affecting lipid metabolism within 14 days (vitamins, fish oil >1000mg/day, red yeast rice, etc.)
- ✕Currently participating in other clinical studies of lipid-lowering drugs
- ✕Poorly controlled hypertension (SBP ≥160 mmHg and/or DBP ≥100 mmHg)
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
NCT06458010Early Ph 1RecruitingMonitorUpdated 14mo ago · Completion was 5mo agoClinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)
In Brief
A Early Phase 1 clinical trial evaluating YOLT-101 for Familial Hypercholesterolemia. Currently recruiting, targeting 20 participants across 1 site.
Signals
Detailed Summary
This study is a single arm, open, single dose escalation trial aimed at evaluating the safety and tolerability of YOLT-101 administration in patients with familial hypercholesterolemia; Determination of YOLT-101 OBD; Preliminary evaluation of the effects of single administration of YOLT-101 on plasma lipid and lipoprotein levels. Note: OBD is defined as the dosage at which plasma PCSK9 protein levels decrease between 60% and 95% from baseline on the 28th day after YOLT-101 administration. OBD ≤ Maximum Tolerable Dose (MTD). In this study, the longest screening period for the main study was 42 days, the treatment day was Day 1 (D1), and the safe follow-up period was up to 52 weeks after medication. In the main study, when OBD occurs, additional subjects will be added to the dose group (specific number of cases will be negotiated between the cooperating organization and investigators) for further validation. In addition, subjects in the first dose group can voluntarily receive a second drug administration of OBD level. After the completion of the main study, participants will undergo long-term follow-up. According to the Technical Guidelines for Long term Follow up Clinical Research of Gene Therapy Products (Trial) released by CDE, a long-term follow-up until 15 years after the medicine administration is required .
Study Details
Timeline
Interventions
The IP is administered intravenously at the predetermined dose.