Treatment of X-linked Chronic Granulomatous Disease
Conditions
Brief summary
Safety as measured by the incidence of adverse events due either to the conditioning regimen, the IMP or to the procedure itself., Efficacy as measured by the restoration and stability over time of the NADPH functioning granulocytes assessed by a DHR test (≥ 5% of expressing cells at 12 months).
Detailed description
Normalisation of nutritional status, growth, development (as measured by clinical evaluation), clearing of the pre-existing severe infection and/or chronic inflammatory lesions which recommended patient’s inclusion, Percentage of transduced CD34+ haematopoietic stem cell at one year and percentage of transduced mature blood cells over time, Immunological reconstitution as measured by evidence of restored neutrophil functionality and immunity against bacterial and fungal infections over time.
Interventions
DRUGG1XCGD (lentiviral vector transduced CD34+ cells)
Sponsors
Genethon
Eligibility
Sex/Gender
Male
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety as measured by the incidence of adverse events due either to the conditioning regimen, the IMP or to the procedure itself., Efficacy as measured by the restoration and stability over time of the NADPH functioning granulocytes assessed by a DHR test (≥ 5% of expressing cells at 12 months). | — |
Secondary
| Measure | Time frame |
|---|---|
| Normalisation of nutritional status, growth, development (as measured by clinical evaluation), clearing of the pre-existing severe infection and/or chronic inflammatory lesions which recommended patient’s inclusion, Percentage of transduced CD34+ haematopoietic stem cell at one year and percentage of transduced mature blood cells over time, Immunological reconstitution as measured by evidence of restored neutrophil functionality and immunity against bacterial and fungal infections over time. | — |
Countries
France
Outcome results
None listed