Rollover Study From EXG-US-01

Phase 1

• Conditions: Telomere Biology Disorders With Bone Marrow Failure
• Interventions: Biological: EXG34217

• Registration Number: NCT05868499
• Lead Sponsor: Elixirgen Therapeutics, Inc.

Brief 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.

Study Timeline

• Study First Submitted: 2023-05-08
• Study First Submitted QC: 2023-05-18
• Study First Posted: 2023-05-22
• Study Start: 2024-01-01
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-10
• Last Update Posted: 2024-07-12
• Primary Completion: 2027-01-30
• Study Completion: 2027-01-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
EXG34217	EXG34217	single autologous CD34+ cells contacted ex vivo with EXG-001

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Number of participants with a change in clinical laboratory evaluations	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change in Electrocardiography (ECG)	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	ECG (standard digital 12-lead in singlicate)
Number of participants with a change of Immunogenicity	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Change in Antibody against virus vector and transgene
umber of participants with a change in in physical examination	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)

Secondary Outcome Measures

Name	Time	Method
Number of participants with a change in telomere length	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Change in telomere length in any peripheral blood cells
Number of participants with improvement of blood counts.	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Blood counts: neutrophils,platelets, or hemoglobin

Trial Locations

Locations (1)

Cincinnati Children's Hospital; 🇺🇸 Cincinnati, Ohio, United States