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Clinical Trial Summary

Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.


Clinical Trial Description

n/a


Study Design

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT01279720
Study type Interventional
Source Great Ormond Street Hospital for Children NHS Foundation Trust
Contact
Status Completed
Phase Phase 1/Phase 2
Start date October 2003
Completion date November 2013

See also
  Status Clinical Trial Phase
Enrolling by invitation NCT04049084 - An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Enrolling by invitation NCT05300334 - Investigation of ADA Enzyme Deficiency
Completed NCT01380990 - Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency Phase 1/Phase 2
Completed NCT03878069 - Registry Study of Revcovi Treatment in Patients With ADA-SCID
Active, not recruiting NCT05300347 - Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)
Completed NCT01420627 - EZN-2279 in Patients With ADA-SCID Phase 3
Enrolling by invitation NCT05300373 - Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E
Completed NCT02022696 - 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 Phase 1
Completed NCT00008450 - Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant Phase 1