Autologous hematopoietic stem cell gene therapy in RAG-deficient severe combined immunodeficiency: pre-phase study

Autologous hematopoietic stem cell gene therapy in RAG-deficient severe combined immunodeficiency: pre-phase study - RAG gene therapy pre-phase

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON55430

Enrollment

Registered

2018-09-03

Start date

2018-12-21

Completion date

Unknown

Last updated

2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

severe combined immunodeficiency

Interventions

None listed

Eligibility

Age

16 Years to 99 Years

Inclusion criteria

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

Exclusion criteria: Any condition precluding stem cell donation Pregnancy

Design outcomes

Primary

Measure	Time frame
Successful transduction of fresh CD34+ hematopoietic stem cells with a lentiviral SIN vector encoding codon-optimized human RAG1 or RAG2 cDNA (SIN-LV-RAG1 or RAG2 vector), defined as a vector copy number higher than 0.5.	—

Secondary

Measure	Time frame
Total number and percentage of viable CD34+ HSC after thawing of cryopreserved transduced cells. Failure to meet release criteria as defined in IMPD. in vitro and/or in vivo functionality assays of transduced CD34+ HSC after thawing.	—

Countries

Netherlands

Contacts

Public ContactCS Ootjers

Leids Universitair Medisch Centrum

stafsecretariaathematologie@lumc.nl0715262267

Outcome results