Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Running, not enrolling · Phase 1

Conditions studied: Telomere Shortening, Bone Marrow Failure

In brief

This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.

Key facts

Study ID

NCT04211714

Run by

Elixirgen Therapeutics, Inc.

People needed

12

Starts

2021-04-08

Expected to finish

2027-10-08

Last updated by the study team

2026-02-06

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 12 years and above
• Mild or moderate bone marrow failure defined by satisfying specific conditions.
• Diagnosis of telomere biology disorders

You may not qualify if…

• Women of child bearing potential or breastfeeding.
• Patients with cancer who are on active chemotherapeutic treatment.
• Patients with severe bone marrow failure.
• Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination.
• Uncontrolled bacterial, viral or fungal infections.
• Prior allogeneic marrow or stem cell transplantation.
• Patients who are not eligible for G-CSF and plerixafor dosing.
• Patients who are not eligible for the apheresis.
• Patients currently taking or have taken danazol and androgens within 60 days prior to Day 1.
• Patients with any other clinically relevant acute or chronic diseases which could interfere with the patients' safety during the trial, expose them to undue risk, or which could interfere with study objectives.
• Patients who have participated in another clinical trial with an investigational drug within the previous 30 days.

Where it is running

• Cincinnati Children's Hospital — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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