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EXG34217 in telomere biology disorders with bone marrow failure
NCT04211714Phase 1Active, not recruiting
- Sponsor
- Elixirgen Therapeutics, Inc.
- Start
- Apr 2021
- Primary completion
- Oct 2026
- 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 Disorder (TBD) and bone marrow failure, sponsored by Elixirgen Therapeutics, Inc. Primary endpoints: Incidence of Treatment-Emergent Adverse Events at 12 months, Change in physical examination at 12 months and 3 more. Started Apr 2021; status: active, not recruiting.
02
Drugs
EXG-34217
03
Conditions
Telomere Biology Disorder (TBD)bone marrow failure
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