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New treatment option for Severe Combined Immunodeficiency, X-Linked
Official title Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
ClinicalTrials.gov ID: NCT03601286
What this study is testing
What is Lentiviral vector transduced CD34+ cells?
Lentiviral vector transduced CD34+ cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for severe combined immunodeficiency, x-linked.
Also referred to as G2SCID lentiviral vector transduced CD34+ cells.
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
- Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. SCID arises from a variety of molecular defects which affect lymphocyte development and function.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 8 to 5, men only
You may be able to join if
- 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...
- Lack of an HLA identical (A, B, C, DR, DQ) related donor
- Age \<5 years
- Signed informed consent
- Documentation of willingness to follow up for 15 years post-infusion
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
- 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
- Lack of an HLA identical (A, B, C, DR, DQ) related donor
- Age \<5 years
- Signed informed consent
- Documentation of willingness to follow up for 15 years post-infusion
- If the patient has previously undergone allogeneic transplant or gene therapy, insufficiency of graft-derived T cell engraftment must be documented.
- Age at least 8 weeks of age 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
- Previous allogeneic transplant with cytoreductive chemotherapy
- 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
- London, Greater London, United Kingdom
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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 8 weeks to 5 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include London, Greater London, United Kingdom. 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.