drugset / Trial / NCT03315078
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the safety and effectiveness of lentiviral gene transfer treatment at restoring immune function to participants with X-linked severe combined immunodeficiency (XSCID) who are 2 to 40 years of age, and have significant impairment of immunity.
Timeline
- Start
- 2012-04
- Primary completion
- 2022-12
- Completion
- 2022-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT | Cell therapy | — | Intravenous |
| Background | Busulfan | Small molecule | 3 mg/kg | Intravenous |
| Background | Palifermin | Protein / enzyme biologic | 60 mg/kg | Intravenous |