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New treatment option for Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Official title Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
ClinicalTrials.gov ID: NCT03645460
What this study is testing
What is Direct intravenous injection of ivlv-ADA lentiviral vector?
Direct intravenous injection of ivlv-ADA lentiviral vector is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for adenosine deaminase severe combined immunodeficiency (ada-scid).
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 trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous (iv) LV gene therapy protocol.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 and older
You may be able to join if
- Diagnosis of classical ADA-SCID based on:
- A proven defective adenosine deaminase (ADA) 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...
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes...
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children.
You likely can't join if
- None
See the full eligibility criteria
- Diagnosis of classical ADA-SCID based on:
- A proven defective adenosine deaminase (ADA) 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 antigens.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children.
- No prior allogeneic stem cell transplantation.
- Life expectancy ≥ 2 months.
- Negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
- None
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 all sexes, 1 month and older. 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.