Actively Recruiting
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Led by Leiden University Medical Center · Updated on 2024-04-18
10
Participants Needed
7
Research Sites
440 weeks
Total Duration
On this page
Sponsors
L
Leiden University Medical Center
Lead Sponsor
Z
ZonMw: The Netherlands Organisation for Health Research and Development
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID.
CONDITIONS
Official Title
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of RAG1-deficient severe combined immunodeficiency (SCID) by genetic testing
- Peripheral blood T cells fewer than 300/µL and/or naive T cells fewer than 1/µL
- Age less than 2 years
- At least 8 weeks old at the time of busulfan and fludarabine treatment
- No available HLA-matched donor (sibling or 10/10 allele-matched related or unrelated donor)
- Signed informed consent from parent or guardian
- Ability to return to the study center for follow-up visits during the 2-year study and 15-year long-term review
You will not qualify if you...
- Availability of an HLA-matched donor (sibling or 10/10 allele-matched related or unrelated donor)
- Peripheral blood T cells more than 300/µL and/or naive T cells more than 1/µL
- Diagnosis of Omenn syndrome
- Previous allogeneic hematopoietic stem cell transplant
- Significant organ problems such as needing mechanical ventilation, heart shortening fraction below 25%, dialysis-dependent kidney failure, or uncontrolled seizures
- Any condition preventing safe collection or infusion of gene-corrected cells or inability to follow the study protocol, including major birth defects or inability to receive anesthesia
- Infection with HIV or Human T-cell Leukemia Virus (HTLV)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
The Royal Childrens Hospital
Melbourne, Australia, 3052
Not Yet Recruiting
2
Ospedale Pediatrico Bambino Gesù
Roma, Italy
Not Yet Recruiting
3
Leiden University Medical Center
Leiden, Netherlands, 2300RC
Actively Recruiting
4
Wroclaw Medical University
Wroclaw, Poland, 50-556
Actively Recruiting
5
Hospital Universitari Vall d'Hebron
Barcelona, Spain, 08035
Actively Recruiting
6
Erciyes Üniversitesi TIP Fakültesi
Kayseri, Turkey (Türkiye)
Actively Recruiting
7
University College London Great Ormond Street
London, United Kingdom
Not Yet Recruiting
Research Team
A
Arjan C Lankester, Prof. Dr.
CONTACT
E
Estefania Laney, MSc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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