Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Recruiting
N/A
Interventional Study
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 1 Month and older
- Sex
- Any
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Shenzhen Geno-Immune Medical Institute · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion 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 …
Contacts