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

Launched by ELIXIRGEN THERAPEUTICS, INC. · Dec 23, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called EXG34217 for patients who have bone marrow failure related to telomere biology disorders. Telomere biology disorders are conditions where the protective ends of chromosomes, called telomeres, are shorter than normal, which can lead to problems with blood cell production in the bone marrow. This study is currently looking for participants aged 12 and older who have mild to moderate bone marrow failure and a confirmed diagnosis of these disorders.

If you or someone you know is interested in participating, it's important to know that the trial is open to all genders, but there are some exclusions. For example, individuals with severe bone marrow failure, ongoing cancer treatment, or certain infections cannot join. Participants will receive the study drug and help researchers learn about its safety and how well it works. This is a great opportunity for those who meet the criteria and are looking for new treatment options, but it's essential to discuss any concerns with a healthcare provider before participating.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Age 12 years and above
• • 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.