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New treatment option for Severe Combined Immunodeficiency, X Linked
Official title Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
ClinicalTrials.gov ID: NCT03311503
What this study is testing
What is autologous CD34+ cell transduced with G2SCID vector?
autologous CD34+ cell transduced with G2SCID vector is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for severe combined immunodeficiency, x linked.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 3 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 5, men only
You may be able to join if
- \- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \<10% of the lower limit of normal for the...
- 7\. Age at least 8 weeks by the time of busulfan administration
You likely can't join if
- Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and...
- Mechanical ventilation including continuous positive airway pressure
- Abnormal liver function defined by AST and ALT \>10 times the upper range of normal OR Bilirubin \>2 mg/dL
- Shortening fraction on echocardiogram \<25% or ejection fraction \<50%
- Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence
- Uncontrolled seizure disorder
See the full eligibility criteria
- \- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \<10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG 2. Lack of an HLA...
- 7\. Age at least 8 weeks by the time of busulfan administration
- Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite...
- Mechanical ventilation including continuous positive airway pressure
- Abnormal liver function defined by AST and ALT \>10 times the upper range of normal OR Bilirubin \>2 mg/dL
- Shortening fraction on echocardiogram \<25% or ejection fraction \<50%
- Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence
- Uncontrolled seizure disorder
- Encephalopathy
- Documented coexistence of any disorder known to affect DNA repair
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease
- Patients with evidence of infection with HIV-1
- Major (life-threatening) congenital anomalies. Examples of "major (life-threatening) congenital anomalies" include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other...
- Other conditions which in the opinion of the P.I. or co-investigators, contra-indicate collection and/or infusion of transduced cells or indicate patient's inability to follow the protocol. These may include for example...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Atlanta, Georgia, United States
- Boston, Massachusetts, United States
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 3 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling male, 0 years to 5 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Atlanta, Georgia, United States; Boston, Massachusetts, United States; Cincinnati, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.