Evaluating EXG34217 for patients with telomere disorders and bone marrow failure
A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
This study is testing a new treatment called EXG34217 to see if it is safe and helps people with bone marrow failure caused by telomere disorders.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Elixirgen Therapeutics, Inc. Industry-sponsored |
| Drugs / interventions | chemotherapy, radiation |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT04211714 on ClinicalTrials.gov |
What this trial studies
This Phase I/II open-label study aims to assess the safety and tolerability of EXG34217 in patients suffering from bone marrow failure due to telomere biology disorders. The study will enroll up to 12 participants, who will undergo a series of procedures including the collection of peripheral blood mononuclear cells, ex vivo cell processing, and subsequent infusion of processed cells. Participants will be monitored for safety and efficacy over a follow-up period of up to 12 months. The study does not require preparative regimens like chemotherapy or radiation prior to participation.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 12 years and older diagnosed with mild to moderate bone marrow failure due to telomere biology disorders.
Not a fit: Patients with severe bone marrow failure, active cancer treatment, or significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with telomere biology disorders and associated bone marrow failure.
How similar studies have performed: While this approach is novel, similar studies targeting telomere biology disorders have shown promise in preliminary investigations.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Cincinnati, Ohio
- Cincinnati Children's Hospital — Cincinnati, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Kasiani Myers, MD — Cincinnati Children Hospital Medical Center
- Study coordinator: Martine Francis
- Email: martine@mafinc.com
- Phone: 301-343-8894
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.