Gene Therapy ADA Deficiency

Status Completed

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

Clinical Trial Details

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

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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