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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

Interventions

BIOLOGICAL

EXG34217

Single infusion

Locations (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, United States