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This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
Patients who received EXG34217 treatment and had at least one follow-up visit (Month 1, 3, 6, or 12) in Study EXG-US-01 will be eligible for this rollover study. Patient will sign a consent form prior to any study related procedure. This study is to add additional follow-up assessments up to 6 years after EXG34217 treatment. This study does not have additional intervention. Additional visits will be every 6 months in the first 3 years and once a year for two years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| EXG34217 | Experimental | single autologous CD34+ cells contacted ex vivo with EXG-001 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EXG34217 | Biological | Single infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events -Safety by Incidence of Treatment-Emergent | Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight. | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| umber of participants with a change in in physical examination | Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic) | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| Number of participants with a change in Electrocardiography (ECG) | ECG (standard digital 12-lead in singlicate) | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| Number of participants with a change in clinical laboratory evaluations | Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis) | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| Number of participants with a change of Immunogenicity | Change in Antibody against virus vector and transgene | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with a change in telomere length | Change in telomere length in any peripheral blood cells | Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |
| Number of participants with improvement of blood counts. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kasiani Myers, MD | Cincinnati Children Hospital Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
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Sequential Assignment
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Blood counts: neutrophils,platelets, or hemoglobin |
| Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72 |