drugset / Trial / NCT07577817
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.
RandomizedSequentialQuadruple-blindTreatment
Summary
This study is testing CTX001 for certain conditions where the body does not have enough available iron or has difficulty storing or moving iron properly. The purpose of this study is to investigate any side effects that may happen with CTX001, how CTX001 is absorbed by and processed in the body, and how CTX001 affects iron levels in the blood when administered with or without iron and/or food.
Timeline
- Start
- 2026-06
- Primary completion
- 2026-10
- Completion
- 2026-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | EXAGAMGLOGENE AUTOTEMCEL | CRISPR / gene editing | — | Oral |
| Background | Iron Sulfate | Small molecule | — | — |