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Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Bone Marrow Failure Clinical Trial

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

Clinical Trial 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.

Clinical Trial Description

This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders with bone marrow failure. The study is open to all participants regardless of gender or ethnicity. Subjects who are enrolled but not evaluable will be replaced. Subjects will sign a consent form prior to any study related procedure and will complete baseline screening assessments. Subjects for this study will not require any preparative regimen such as chemotherapy or radiation. The study will be conducted in three parts - Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis, - Ex vivo cell processing - Processed cell infusion and post-infusion safety monitoring, - Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12) ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04211714
Study type Interventional
Source Elixirgen Therapeutics, Inc.
Contact Martine Francis
Phone 301-343-8894
Email martine@mafinc.com
Status Recruiting
Phase Phase 1
Start date April 8, 2021
Completion date April 8, 2027
View Details
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