Telomere Biology Disorders With Bone Marrow Failure
Investigational drug(s) / intervention(s)
EXG34217
EXG34217: Single infusion
Study summary
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures.
2. Have completed the 12-month visit of Study EXG-US-01.
Exclusion Criteria:
\-
Primary outcome measure(s)
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent — Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
umber of participants with a change in in physical examination — Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 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) — Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 ECG (standard digital 12-lead in singlicate)
Number of participants with a change in clinical laboratory evaluations — Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity — Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 Change in Antibody against virus vector and transgene
Trial sites (1)
Facility
City
Region
Status
Cincinnati Children's Hospital
Cincinnati
Ohio
More Elixirgen Therapeutics, Inc. trials in the USA
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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