Rollover Study From EXG-US-01
Study Details
Study Description
Brief Summary
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Detailed Description
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: EXG34217 single autologous CD34+ cells contacted ex vivo with EXG-001 |
Biological: EXG34217
Single infusion
|
Outcome Measures
Primary Outcome Measures
- 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
Secondary Outcome Measures
- Number of participants with a change in telomere length [Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72]
Change in telomere length in any peripheral blood cells
- Number of participants with improvement of blood counts. [Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72]
Blood counts: neutrophils,platelets, or hemoglobin
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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.
-
Have completed the 12-month visit of Study EXG-US-01.
Exclusion Criteria:
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Cincinnati Children's Hospital | Cincinnati | Ohio | United States | 45229 |
Sponsors and Collaborators
- Elixirgen Therapeutics, Inc.
Investigators
- Principal Investigator: Kasiani Myers, MD, Cincinnati Children Hospital Medical Center
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- EXG-US-02