NCT04211714

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

A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Elixirgen Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Detailed 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)

Conditions

• Telomere Shortening
• Bone Marrow Failure

Interventions

Type	Name	Description
BIOLOGICAL	EXG34217	Single infusion

Timeline

• Start date: 2021-04-08
• Primary completion: 2026-10-08
• Completion: 2027-10-08
• First posted: 2019-12-26
• Last updated: 2026-02-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study