NCT05868499

Rollover Study From EXG-US-01

Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01

• Status: Enrolling By Invitation
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Elixirgen Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).

Detailed description

Patients who received EXG34217 treatment and had at least one follow-up visit (Month 1, 3, 6, or 12) in Study EXG-US-01 will be eligible for this rollover study. Patient will sign a consent form prior to any study related procedure. This study is to add additional follow-up assessments up to 6 years after EXG34217 treatment. This study does not have additional intervention. Additional visits will be every 6 months in the first 3 years and once a year for two years.

Conditions

• Telomere Biology Disorders With Bone Marrow Failure

Interventions

Type	Name	Description
BIOLOGICAL	EXG34217	Single infusion

Timeline

• Start date: 2024-01-01
• Primary completion: 2027-01-30
• Completion: 2027-01-30
• First posted: 2023-05-22
• Last updated: 2024-07-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study