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

  • End date
    Apr 8, 2024
  • participants needed
  • sponsor
    Elixirgen Therapeutics, Inc.
Updated on 12 May 2022


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.


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)

Condition Telomere Shortening, Bone Marrow Failure
Treatment EXG34217
Clinical Study IdentifierNCT04211714
SponsorElixirgen Therapeutics, Inc.
Last Modified on12 May 2022


Yes No Not Sure

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 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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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