Rollover Study From EXG-US-01

Launched by ELIXIRGEN THERAPEUTICS, INC. · May 18, 2023

Keywords

ClinConnect Summary

This is a long-term follow-up study for people with telomere biology disorders and bone marrow failure who previously participated in the EXG-US-01 trial and received EXG34217. The study will monitor these participants for up to six years after treatment, focusing on safety and overall health. There is no new treatment given in this study; after the initial treatment, participants will have follow-up visits about every six months for the first three years, then once a year for the next two years. Safety and health checks will include adverse events, vital signs, physical exams, ECGs, lab tests, and immune responses.

Who may be eligible: adults 18 years or older who completed at least the 12-month visit in the prior EXG-US-01 study and who give informed consent. The study is enrolling by invitation at Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio, sponsored by Elixirgen Therapeutics. Participants can expect ongoing safety monitoring and health assessments, including checks on blood counts and telomere length over time. An independent data safety monitoring committee oversees safety, and this study involves a FDA-regulated drug.

Gender

ALL

Eligibility 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:
• • -