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

Description

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.

Conditions

Telomere Shortening, Bone Marrow Failure

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Telomere Shortening
Intervention / Treatment

-

Contacts and Locations

Cincinnati

Cincinnati Children's Hospital, Cincinnati, Ohio, United States, 45229

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

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

Ages Eligible for Study

12 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Elixirgen Therapeutics, Inc.,

Kasiani Myers, MD, PRINCIPAL_INVESTIGATOR, Cincinnati Children Hospital Medical Center

Study Record Dates

2027-04-08