Gene therapy for treating ADA-SCID
Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)
NA · Shenzhen Geno-Immune Medical Institute · NCT03645460
This study is testing a new gene therapy to see if it can help people with ADA-SCID recover their immune system and fight off severe infections.
Quick facts
| Phase | NA |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 1 Month and up |
| Sex | All |
| Sponsor | Shenzhen Geno-Immune Medical Institute (other) |
| Locations | 2 sites (Shenzhen, Guangdong and 1 other locations) |
| Trial ID | NCT03645460 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of an improved lentiviral vector gene therapy for patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID). The approach involves direct intravenous injection of a self-inactivating lentiviral vector carrying a functional ADA gene to correct the genetic defect responsible for the condition. The study aims to assess immune recovery and the long-term effects of this gene transfer procedure, particularly in patients suffering from severe infections due to their immunodeficiency.
Who should consider this trial
Good fit: Ideal candidates include patients diagnosed with classical ADA-SCID who have a proven defective ADA gene and are experiencing severe infections.
Not a fit: Patients who have undergone prior allogeneic stem cell transplantation or have cytogenetic abnormalities may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a safer alternative to bone marrow transplants for ADA-SCID patients, potentially leading to improved immune function and reduced infection rates.
How similar studies have performed: Other studies using lentiviral gene therapy for similar conditions have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Shenzhen, Guangdong and 1 other locations
- Shenzhen Geno-immune Medical Institute — Shenzhen, Guangdong, China (RECRUITING)
- Guilin Hospital of Chinese Traditional and Western Medicine — Guilin, Guangxi, China (RECRUITING)
Study contacts
- Principal investigator: Lung-Ji Chang, Ph.D — Shenzhen Geno-Immune Medical Institute
- Study coordinator: Lung-Ji Chang, Ph.D
- Email: c@szgimi.org
- Phone: 86-0755-86725195
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Adenosine DeAminase Severe Combined ImmunoDeficiency, Adenosine deaminase severe combined immunodeficiency lentiviral vector, in vivo gene therapy