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New treatment option for SCID, X-Linked
Official title SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
ClinicalTrials.gov ID: NCT03217617
What this study is testing
What is Direct intravenous injection of ivlv-X1 lentiviral vector?
Direct intravenous injection of ivlv-X1 lentiviral vector is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for scid, 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 gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 1, men only
You may be able to join if
- Diagnosis of SCID-X1 based on:
- A proven mutation in the common interleukin-2 receptor gamma chain gene as defined by direct sequencing of patient DNA.
- T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro...
- No available HLA identical related donor.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes...
You likely can't join if
- No available molecular diagnosis confirming SCID-X1.
- Existence of an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
- Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
- Patients with evidence of infection with HIV-1 or 2.
- Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure...
See the full eligibility criteria
- Diagnosis of SCID-X1 based on:
- A proven mutation in the common interleukin-2 receptor gamma chain gene as defined by direct sequencing of patient DNA.
- T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigen...
- No available HLA identical related donor.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
- No prior allogeneic stem cell transplantation.
- Life expectancy ≥ 3 months.
- Documented to be negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
- No available molecular diagnosis confirming SCID-X1.
- Existence of an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
- Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
- Patients with evidence of infection with HIV-1 or 2.
- Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive...
- Current treatment with any immunosuppressive agent, excluding corticosteroids.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
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, 1 month to 1 year. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. 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.