Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
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Phase 2 N=12 ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Phase 2 N=46 Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
ADA-SCID
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 85.71; 100; 92.31 percentage of participants
Phase 3 N=7 EZN-2279 in Patients With ADA-SCID
ADA-SCID · Adenosine Deaminase Deficiency · Severe Combined Immunodeficiency
Primary: Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period — 6; 6; 6; 5 Participants
Phase 2 N=36 Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Adenosine Deaminase Deficiency · Severe Combined Immunodeficiencies (SCID)
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 100; 100; 100 percentage of participants
Phase 2 N=10 Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Severe Combined Immunodeficiency Due to ADA Deficiency
Primary: Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months) — 100; 100; 90 percentage of participants
N/A N=32 Registry Study of Revcovi Treatment in Patients With ADA-SCID
Adenosine Deaminase Severe Combined Immunodeficiency
Primary: Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement — 6; 17; 4 Participants
Phase 2 N=8 Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Severe Combined Immunodeficiency
Primary: CD3 Cell Count Post Infusion — 5; 3 Participants
Early Phase 1 N=48 Donor Stem Cell Transplantation for Congenital Immunodeficiencies
Inherited Immune Deficiencies
Primary: Stem Cell Transplant Engraftment — 7; 31; 3; 0 Participants
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=12 Use of ACTIMMUNE in Patients With ADO2
Autosomal Dominant Osteopetrosis Type 2
Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Phase 2 N=136 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
Phase 2 N=47 Alemtuzumab to Treat Severe Aplastic Anemia
Severe Aplastic Anemia, Refractory · Severe Aplastic Anemia, Relapse
Primary: Number of Participants With Hematological Response at 6 Months — 0; 18 Participants
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Phase 2 N=22 Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Blood Counts and Adverse Event Profile After 6 Months of Treatment. — 4; 5; 12 participants
Phase 2 N=29 Alemtuzumab, Fludarabine Phosphate, and Total-Body Irradiation Followed by a Donor Stem Cell Transplant in Treating Patients With Immunodeficiency or Other Nonmalignant Inherited Disorders
Immunodeficiency Syndrome · Non-Cancer Diagnosis
Primary: Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism — 13 Participants
Phase 3 N=98 TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Primary Immune Deficiency Disorder · Hematopoietic Stem Cell Transplantation
Primary: Overall Survival — 86; 86; 87 percentage of survival probability — p=0.95
N/A N=9 Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Number of Patients With Engraftment Response — 9 Participants
N/A N=29 Thymus Transplantation Safety-Efficacy
Complete DiGeorge Anomaly · DiGeorge Syndrome · DiGeorge Anomaly
Primary: Survival Rate at End of 1 Year — 79.3 percentage of participants
Phase 2 N=8 Gene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Primary: Safety of Reduced Conditioning Regimen — 8 Participants
Phase 2 N=26 Thymus Transplantation in DiGeorge Syndrome #668
DiGeorge Syndrome · Complete Typical DiGeorge Anomaly
Primary: Survival at 1 Year Post-Cultured Thymus Tissue Implantation (CTTI) — 72 % of participants who survive to 1 year
N/A N=4 Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Anemia, Aplastic · Amegakaryocytic Thrombocytopenia · Diamond-Blackfan Anemia
Primary: Treatment Failures
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
Phase 2 N=15 Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease
Chronic Granulomatous Disease
Primary: Percentage of Participants With Engraftment — 100 percentage of participants
Phase 2 N=31 Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA) · Severe Aplastic Anemia (SAA)
Primary: Number of Participants Who Engrafted by Day 42 — 30 Participants
Phase 2 N=20 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months — 9 participants
Phase 2 N=46 Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome
Primary: Number of Patients With Donor Derived Engraftment — 42 Participants
Phase 2 N=7 Thymus Transplantation Dose in DiGeorge #932
DiGeorge Anomaly · DiGeorge Syndrome · Complete DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 83 % of participants who survive to 1 year
Phase 3 N=37 Study of Efficacy of CDZ173 in Patients With APDS/PASLI
Common Variable Immunodeficiency (CVID), APDS / PASLI
Primary: Part I: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 2; 0; 2; 0 Participants