Early Phase 1 Study of Single-Dose YOLT-101 Injection to Evaluate Safety and Dosage in Adults with Familial Hypercholesterolemia

What this Trial Is About

Researchers are evaluating YOLT-101, a drug given by injection, in patients with familial hypercholesterolemia to assess its safety and tolerability. This early phase trial aims to find the optimal biological dose OBD of YOLT-101, defined as the dose that reduces plasma PCSK9 protein levels by 60% to 95% from baseline by day 28 after administration. Additionally, the study will preliminarily examine how a single dose of YOLT-101 affects plasma lipid and lipoprotein levels. Participants receive a single intravenous injection of YOLT-101 at one of several doses 0.2 mgkg, 0.4 mgkg, 0.6 mgkg, 40 mg, or 50 mg. The study includes a screening period up to 42 days before treatment day Day 1 and a safety follow-up lasting up to 52 weeks after the dose. When the OBD is identified, more participants may be added at that dose level for confirmation. Participants in the first dose group may choose to receive a second dose at the OBD level. After the main study, long-term follow-up will continue for up to 15 years as per gene therapy product guidelines. Throughout the study, participants will be monitored for safety through various assessments including laboratory tests and observation for adverse effects. Researchers will evaluate the safety of YOLT-101 through week 52 and study its pharmacokinetics and pharmacodynamics up to day 28 and week 52, respectively. This extensive follow-up aims to capture both short-term and long-term effects of the drug. The total participation may span over 15 years to ensure comprehensive evaluation.

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)