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NCT05868499 | ENROLLING BY INVITATION | Telomere Biology Disorders With Bone Marrow Failure

Rollover Study From EXG-US-01
Sponsor:

Elixirgen Therapeutics, Inc.

Brief Summary:

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

Condition or disease

Telomere Biology Disorders With Bone Marrow Failure

Intervention/treatment

EXG34217

Phase

PHASE1

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.

Study Type : INTERVENTIONAL
Estimated Enrollment : 12 participants
Masking : NONE
Primary Purpose : SUPPORTIVE_CARE
Official Title : Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
Actual Study Start Date : 2024-01-01
Estimated Primary Completion Date : 2027-01-30
Estimated Study Completion Date : 2027-01-30

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • 1. Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures.
  • 2. Have completed the 12-month visit of Study EXG-US-01.
Exclusion Criteria
  • -
Rollover Study From EXG-US-01

Location Details

NCT05868499

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, Ohio

Cincinnati Children's Hospital

Cincinnati, Ohio, United States, 45229