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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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
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=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=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=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=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=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 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=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
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 2 N=14 Phase I/II Thymus Transplantation With Immunosuppression #950
DiGeorge Anomaly · Complete DiGeorge Anomaly · Complete Atypical DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 71 % of participants who survive to 1 year
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=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=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=136 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
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=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=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 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
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
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=77 Phase 2/3 Study of IGSC, 20% in PIDD
Primary Immunodeficiency Diseases (PID)
Primary: Rate of Acute Serious Bacterial Infections Per Year (ASBI) — 0.012 Estimated infections/ year — p=<0.0001
Phase 2 N=2 Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Bone Marrow Failure Syndromes · Immunodeficiencies · Immune Dysregulation Syndromes
Primary: Number of Participants With Engraftment — 2 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
N/A N=1,086 Quality Assurance of Secondary Immunodeficiencies (SID) in CLL/MM Patients
Secondary Immunodeficiencies (SID)
Primary: Guideline Adherence (GLAD) — 889; 75; 122 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
N/A N=314 Primary Immunodeficiency in Kuwait
Primary Immune Deficiency Disorder
Primary: Number of Participants With Different Epidemiological Features — 287; 100; 68; 56 patients
Phase 2 N=30 Sirolimus for Autoimmune Disease of Blood Cells
Autoimmune Pancytopenia · Autoimmune Lymphoproliferative Syndrome (ALPS) · Evans Syndrome
Primary: Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus — 1 participants
Phase 2 N=80 Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy
Chronic Inflammatory Demyelinating Polyneuropathy
Primary: Number of Participants With Survival — 66; 64 Participants