Clinical trials: SCID
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.
ADA-SCID
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Background: Severe combined immunodeficiency (SCID) is a rare inherited disorder in which certain white blood cells have impaired function and are unable to properly fight infections.
- Trial name
- NCT01182857
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adenosine Deaminase Deficiency, ADA-SCID
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector
This is a clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase (ADA) gene into the hematopoietic progenitors of patients…
- Trial name
- NCT02022696
- Testing
- Lentiviral Gene Transfer
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Human Genome Research Institute (NHGRI)