Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Suspended
Phase 1
Phase 2
Interventional Study
Severe Combined Immunodeficiency Due to RAG1 Deficiency
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 8 Weeks – 24 Months
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Videja B.V. · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
This trial is a prospective, non-randomized, open-label, multicentre single-arm phase I/II intervention trial in children up to 24 months of age with RAG1-deficient SCID and an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The trial involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
1. RAG1-deficient SCID as confirmed by genetic analysis
2. Peripheral blood CD3+T cells \< 300/μL
3. Absence of peripheral blood naïve CD4+ T cells
4. Age \< 2 years
5. Age at least 8 weeks by the time of busulfan and fludarabine administration
6. Lack of an available HLA-identi…
Contacts
No contact information available.