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ACTIVE NOT RECRUITING
NCT04211714
PHASE1
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
Sponsor: Elixirgen Therapeutics, Inc.
View on ClinicalTrials.gov
Summary
This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
Official title: A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
Key Details
Gender
All
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2021-04-08
Completion Date
2027-10-08
Last Updated
2026-02-06
Healthy Volunteers
No
Conditions
Interventions
BIOLOGICAL
EXG34217
Single infusion
Locations (1)
Cincinnati Children's Hospital
Cincinnati, Ohio, United States