Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

Launched by SHENZHEN GENO-IMMUNE MEDICAL INSTITUTE · Aug 22, 2018

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy designed to treat a rare genetic disorder called Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID). ADA-SCID affects the immune system, making it difficult for the body to fight off infections. The therapy uses a specially designed virus to deliver a healthy copy of the ADA gene directly into the patient's cells, aiming to correct the underlying genetic defect. The trial will focus on ensuring that this treatment is safe and effective for patients.

To participate in this trial, individuals must have been diagnosed with ADA-SCID and show specific signs of immune deficiency, such as low levels of certain immune cells. They should also be experiencing severe infections and not have had previous stem cell transplants. Participants will receive the gene therapy through an intravenous (IV) infusion and will be closely monitored for their safety and how well the treatment works. This trial is currently recruiting participants of all ages, and anyone interested will need to provide written consent before joining.

Gender

ALL

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 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.
• Exclusion Criteria:
• • None