Trial Profile

A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 03 Mar 2025

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At a glance

Drugs EXG 34217 (Primary)
Indications Bone marrow disorders; Chromosome disorders
Focus Adverse reactions; Pharmacodynamics
Sponsors Elixirgen

Most Recent Events

25 Feb 2025 According to an Elixirgen media release, data from this trial published in NEJM
25 Feb 2025 According to an Elixirgen media release, enrollment is currently open for participants aged 12 and older, regardless of gender or ethnicity, in the Phase 1/2 study (NCT04211714) evaluating EXG-34217 in patients with TBDs with bone marrow failure.
25 Feb 2025 Results published in the Media Release

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