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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events — Multiple times for the duration of the study (baseline through Month 12) Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Number of participants with a change in in physical examination — Multiple times for the duration of the study (baseline through Month 12) Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Number of participants with a change in Electrocardiography (ECG) — Multiple times for the duration of the study (baseline through Month 12) ECG (standard digital 12-lead in singlicate)
Number of participants with a change in clinical laboratory evaluations — Multiple times for the duration of the study (baseline through Month 12) Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity — Multiple times for the duration of the study (baseline through Month 12) Change in Antibody against virus vector and transgene
Trial sites (1)
Facility
City
Region
Status
Cincinnati Children's Hospital
Cincinnati
Ohio
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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