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

Brief Title

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

Official Title

A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Brief Summary

      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.

Detailed Description

      This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders
      with bone marrow failure. The study is open to all participants regardless of gender or
      ethnicity. Subjects who are enrolled but not evaluable will be replaced.

      Subjects will sign a consent form prior to any study related procedure and will complete
      baseline screening assessments. Subjects for this study will not require any preparative
      regimen such as chemotherapy or radiation.

      The study will be conducted in three parts

        -  Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis,

        -  Ex vivo cell processing

        -  Processed cell infusion and post-infusion safety monitoring,

        -  Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12)

Study Phase

Phase 1

Study Type


Primary Outcome

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

Secondary Outcome

 Number of participants with a change in telomere length


Telomere Shortening



Study Arms / Comparison Groups

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


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

April 8, 2021

Completion Date

April 8, 2024

Primary Completion Date

April 8, 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Age > 18 years.

          -  Mild or moderate bone marrow failure defined by satisfying specific conditions.

          -  Diagnosis of telomere biology disorders

        Exclusion Criteria:

          -  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

          -  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.




18 Years - N/A

Accepts Healthy Volunteers



Kasiani Myers, MD, 301-343-8894, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Elixirgen Therapeutics, Inc.

Study Sponsor

Kasiani Myers, MD, Principal Investigator, Cincinnati Children Hospital Medical Center

Verification Date

April 2021