Trials / Unknown
UnknownNCT04286815
Gene Therapy for X Linked Severe Combined Immunodeficiency
- Status
- Unknown
- Phase
- N/A
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- Sponsor
- Children's Hospital of Chongqing Medical University · Academic / Other
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DEVICE | Lentiviral Vector Gene Therapy | Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells |
Timeline
- Start date
- 2020-05-01
- Primary completion
- 2023-05-01
- Completion
- 2025-05-01
- First posted
- 2020-02-27
- Last updated
- 2020-03-27
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT04286815. Inclusion in this directory is not an endorsement.