Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (NCT04211714) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
United States12 participantsStarted 2021-04-08
Plain-language 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.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
2
Number of participants with a change in in physical examination
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
3
Number of participants with a change in Electrocardiography (ECG)
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
4
Number of participants with a change in clinical laboratory evaluations
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
5
Number of participants with a change of Immunogenicity
Timeframe: Multiple times for the duration of the study (baseline through Month 12)