Rollover Study From EXG-US-01

Phase 1Enrolling By InvitationNCT05868499

Elixirgen Therapeutics, Inc.12 enrolled

Overview

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

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

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Enrollment

Conditions

Telomere Biology Disorders With Bone Marrow Failure

Interventions

EXG34217BIOLOGICAL

Single infusion

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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: \-

Locations (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Number of participants with adverse events -Safety by Incidence of Treatment-Emergent

Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.