6 completed, 3 unknown, 1 enrolling by invitation. 4 Phase 1, 2 Phase 2, 1 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 4 |
| Phase 2 | 2 | Completed 2 |
| Not applicable | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow Adenosine Deaminase Deficiency: new trials by email Literature Review of Adenosine Deaminase Deficiency trials Research Report on Adenosine Deaminase Deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E ADENOSINE DEAMINASE TEST - Adenosine Deaminase Deficiency | Unknown | TRPHARM | 100 | 2021-12-03 | 2022-03-29 | |
| Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA) adenosine deaminase enzyme deficiency test - Bronchiectasis, Adenosine Deaminase Deficiency | Unknown | TRPHARM | 500 | 2021-06-17 | 2022-03-29 | |
| Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach bone marrow aspiration and biopsy - Adenosine Deaminase Deficiency | Not applicable | Completed | Istituto Giannina Gaslini | 29 | 2021-05-31 | 2026-10-20 |
| Investigation of ADA Enzyme Deficiency adenosine deaminase test - Adenosine Deaminase Deficiency | Unknown | TRPHARM | 800 | 2019-10-10 | 2022-03-29 | |
| An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) - Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID) | Enrolling by invitation | University of California, Los Angeles | 70 | 2019-09-26 | 2025-12-18 | |
| EZN-2279 in Patients With ADA-SCID EZN-2279, Adagen - ADA-SCID, Adenosine Deaminase Deficiency | Phase 3 | Completed | Leadiant Biosciences, Inc. | 7 | 2014-01-24 | 2020-04-16 |
| 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 Lentiviral Gene Transfer - Adenosine Deaminase Deficiency, ADA-SCID | Phase 1 | Completed | National Human Genome Research Institute (NHGRI) | 1 | 2013-12-16 | 2017-09-27 |
| Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan - Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID) | Phase 1 / Phase 2 | Completed | Great Ormond Street Hospital for Children NHS Foundation Trust | 36 | 2012-11-15 | 2021-09-16 |
| Gene Therapy ADA Deficiency Intravenous infusion of transduced cells - Adenosine Deaminase Deficiency | Phase 1 / Phase 2 | Completed | Great Ormond Street Hospital for Children NHS Foundation Trust | 8 | 2003-10-01 | 2015-09-14 |
| Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis - Adenosine Deaminase Deficiency, Autosomal Recessive Disorder | Phase 1 | Completed | Fred Hutchinson Cancer Center | 6 | 1997-08-11 | 2019-07-29 |
Severe combined immunodeficiency due to adenosine deaminase deficiency (10) · Severe Combined Immunodeficiency (4) · Lymphopenia (2) · Bronchiectasis (1) · Immune System Diseases (1) · Lung Diseases (1) · Purine Nucleoside Phosphorylase Deficiency (1) · X-Linked Combined Immunodeficiency Diseases (1)
Immunologic Deficiency Syndromes (6) · Immune System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · DNA Repair-Deficiency Disorders (4) · Genetic Diseases, Inborn (4) · Infant, Newborn, Diseases (4) · Metabolic Diseases (4) · Nutritional and Metabolic Diseases (4) · Primary Immunodeficiency Diseases (4) · Cytopenia (2) · Hematologic Diseases (2) · Hemic and Lymphatic Diseases (2) · Leukocyte Disorders (2) · Leukopenia (2) · Bronchial Diseases (1)
pegademase bovine (2) · Biopsy (1) · Busulfan (1) · Cyclosporine (1) · Cyclosporins (1) · Genes (1) · Mycophenolic Acid (1) · Whole-Body Irradiation (1)
TRPHARM (3) · Great Ormond Street Hospital for Children NHS Foundation Trust (2) · Fred Hutchinson Cancer Center (1) · Istituto Giannina Gaslini (1) · Leadiant Biosciences, Inc. (1) · National Human Genome Research Institute (NHGRI) (1) · University of California, Los Angeles (1)
United States (4) · Turkey (Türkiye) (3) · United Kingdom (3) · Italy (1)
Claire Booth, Dr (1) · Donald B. Kohn, M.D. (1) · Elie Haddad, MD, PhD (1) · Elizabeth K Garabedian, R.N. (1) · Lauri Burroughs (1)
P01HL036444 (1), P30CA015704 (1)