Rollover Study From EXG-US-01

Elixirgen Therapeutics

Status and phase: Invitation-only; Phase 1

Conditions: Telomere Biology Disorders With Bone Marrow Failure

Treatments: Biological: EXG34217

Study type: Interventional

Funder types: Industry

Identifiers

NCT05868499

EXG-US-02

Details and patient eligibility

About

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

Full 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.

Enrollment: 12 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Supportive Care

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

EXG34217

Experimental group

Description:

single autologous CD34+ cells contacted ex vivo with EXG-001

Treatment:

Biological: EXG34217