| Protocol No: | ECCT/25/07/01 | Date of Protocol: | 04-04-2025 |
| Study Title: | A Phase 1, Randomized, Placebo-Controlled, Double Blind, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CLY-124 in Healthy Volunteers and Participants with Sickle Cell Disease |
| Study Objectives: | Primary: • To evaluate the safety and tolerability of a single ascending dose (SAD) and multiple ascending doses (MAD) of CLY-124 in HVs and participants with SCD • To evaluate the pharmacokinetics (PK) profile of CLY-124 and metabolites in HVs and participants with SCD Secondary: • To evaluate the relationship between CLY-124 exposure and fetal hemoglobin (HbF) induction in HVs and participants with SCD • To explore the effects of food on the PK of CLY-124 in HVs Exploratory: • To evaluate the pharmacodynamics (PD) of CLY-124 in HVs and participants with SCD • To explore the association of CLY-124 exposure and response variables |
| Laymans Summary: | The purpose of this FIH study is to assess the safety and tolerability of CLY-124 in HVs and participants with SCD after oral administration of a SAD and MAD of CLY-124 and to identify the range of doses that are safe and pharmacologically active in participants with SCD |
| Abstract of Study: | This is a placebo-controlled, randomized, double-blind study to evaluate the safety, tolerability, PK, and PD of CLY-124 in HVs and participants with SCD after a single dose and after repeat dosing with food. The study will consist of 4 parts with either adult HVs (18 to 65 years of age) or participants with SCD (18 to 55 years of age). A food effect (FE) cohort will be included in Part 1 (SAD – HV), in which HVs will be administered a single dose of CLY-124 under high fat-high calorie, low fat-low calorie, or fasted conditions to obtain a preliminary assessment regarding the effect of food on the PK of CLY-124. Approximately 163 participants are planned to be enrolled across the 4 parts of the study. |