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Long-Term Follow-Up of EXG34217 in Telomere Biology Disorders
NCT05868499Phase 1Enrolling by invitation
- Sponsor
- Elixirgen Therapeutics, Inc.
- Start
- Jan 2024
- Primary completion
- Jan 2027
- Modality
- Gene Editing, Adoptive Cell Transfer, Gene Silencing
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of EXG-34217 in telomere biology disorders (TBD) and bone marrow failure, sponsored by Elixirgen Therapeutics, Inc. Primary endpoints: Number of participants with adverse events at Month 72, Number of participants with a change in physical examination at Month 72 and 3 more. Started Jan 2024; status: enrolling by invitation.
02
Drugs
EXG-34217
03
Conditions
telomere biology disorders (TBD)bone marrow failure
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