Trials / Unknown
UnknownNCT01175239
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
Gene Therapy for SCID-X1 Using a Self-inactivating (SIN) Gammaretroviral Vector
- Status
- Unknown
- Phase
- N/A
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust · Academic / Other
- Sex
- Male
- Age
- 16 Years
- Healthy volunteers
- Not accepted
Summary
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys. This trial aims to treat SCID-X1 patients using gene therapy to replace the defective gene.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre | Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre |
Timeline
- Start date
- 2011-04-01
- Primary completion
- 2018-12-01
- Completion
- 2018-12-01
- First posted
- 2010-08-04
- Last updated
- 2017-08-01
Locations
1 site across 1 country: United Kingdom
Source: ClinicalTrials.gov record NCT01175239. Inclusion in this directory is not an endorsement.