Autologous hematopoietic stem cell gene therapy in RAG-deficient severe combined immunodeficiency: pre-phase study
Recruiting
- Conditions
- severe combined immunodeficiency10021460
- Registration Number
- NL-OMON55430
- Lead Sponsor
- eids Universitair Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 8
Inclusion Criteria
Selection as stem cell donor for a family member;
- In case of stem cells from mobilized peripheral blood: completion of G-CSF
mobilization procedure for collection of CD34+ HSC from G-CSF mobilized
peripheral blood;
Age > 18 years;
Able to comprehend and give signed informed consent.
Exclusion Criteria
Any condition precluding stem cell donation
Pregnancy
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Successful transduction of fresh CD34+ hematopoietic stem cells with a<br /><br>lentiviral SIN vector encoding codon-optimized human RAG1 or RAG2 cDNA<br /><br>(SIN-LV-RAG1 or RAG2 vector), defined as a vector copy number higher than 0.5.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Total number and percentage of viable CD34+ HSC after thawing of cryopreserved<br /><br>transduced cells.<br /><br>Failure to meet release criteria as defined in IMPD.<br /><br>in vitro and/or in vivo functionality assays of transduced CD34+ HSC after<br /><br>thawing.</p><br>