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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=2 Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Hereditary Pulmonary Alveolar Proteinosis
Primary: Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test — 17; 33 Percent Change
N/A N=50 Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
Inflammatory Bowel Disease · Glycogen Storage Disease Type Ia
Primary: Serologic, Genetic and Inflammatory Markers Consistent With Inflammatory Bowel Disease — 11 participants testing positive
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=50 Ixazomib Citrate in Treating Patients With Chronic Graft-versus-Host Disease
Chronic Graft Versus Host Disease
Primary: Incidence of Adverse Events — 19; 18 participants
Phase 2 N=42 Study of the Bruton's Tyrosine Kinase Inhibitor in Subjects With Chronic Graft Versus Host Disease
Graft Versus Host Disease
Primary: Phase 1b: To Evaluate the Safety and Tolerability of Ibrutinib in Steroid Dependent/Refractory cGVHD. — 0 Participants
Phase 2 N=3 Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency
Gastrointestinal Inflammation Associated With CVID · CVID Enteropathy
Primary: Number of Participants With Increase in Infection or Serious Adverse Events (SAEs) Related to Single Dose Administration of Ustekinumab — 0 participants
Phase 3 N=114 Safety and Effectiveness of Granulocyte Transfusions in Resolving Infection in People With Neutropenia (The RING Study)
Neutropenia · Infection
Primary: Percentage of Participants Who Are Alive at 42 Days After Treatment and Have Had Microbial Response — 42.9; 41.7 percentage of participants — p=0.73
Phase 2 N=20 Carfilzomib in Treating Patients With Chronic Graft-Versus-Host Disease
Chronic Graft Versus Host Disease
Primary: Incidence of Adverse Events — 8; 7 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 3 N=32 A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher Disease
Primary: Change From Baseline in Spleen Volume Measured by MRI. — -26.91; -38.01 percentage of change — p=<0.0001
Phase 2 N=32 Pilot Trial of Inhaled Molgramostim in Non-tuberculous Mycobacterial (NTM) Infection
Mycobacterium Infections, Nontuberculous
Primary: Number of Participants With Sputum Culture Conversion to Negative — 2; 3 Participants
Phase 3 N=49 Study of Immune Globulin Intravenous (Human) GC5107 in Subjects With Primary Humoral Immunodeficiency
Immunologic Deficiency Syndromes
Primary: The Incidence of Acute Serious Bacterial Infections (SBI) — 0.04; 0; 0.02 Number of events per patient per year
Phase 2 N=7 Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa
Primary: Percent Change of Active Blisters and in Total Blister/Erosion Counts — -29.6 percent change — p=0.82
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=18 Inhaled GM-CSF Therapy of Autoimmune PAP
Autoimmune Pulmonary Alveolar Proteinosis
Primary: Time to Rescue WLL — 18; 30 Months — p=0.0078
Phase 3 N=151 Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801)
Chronic GVHD
Primary: Proportion of Participants With Treatment Success — 33; 35; 33; 37 Participants — p=0.63
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=80 Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy
Chronic Inflammatory Demyelinating Polyneuropathy
Primary: Number of Participants With Survival — 66; 64 Participants
Phase 2 N=20 Imatinib Mesylate to Treat Skin Changes in Patients With Chronic Graft-Versus-Host Disease
Sclerotic Graft Versus Host Disease · Imatinib Mesylate
Primary: Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months — 94; 35; 16; 21 Percent change from baseline
Phase 2 N=20 Abatacept in Treating Adults With Mild Relapsing Wegener's Granulomatosis
Wegener's Granulomatosis
Primary: Safety of Abatacept - Number of Participants With Adverse Events — 7; 16; 14; 1 participants
Phase 2 N=36 Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease
Graft vs Host Disease
Primary: Clinical Activity — 4; 4 participants
Phase 2 N=17 Discontinuation or Continuation of Immunosuppressive Therapy in Participants With Chronic Graft Versus Host Disease
Chronic Graft Versus Host Disease
Primary: Feasibility of Enrolling Patients — 21 Participants
N/A N=60 Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
Gaucher Disease
Primary: Number of Patients With GD Diagnosis Confirmed by : Enzyme Testing of acidβ-glucosidase Activity Activity <15% in Blood Leucocytes Completed When Necsssary by GB1…
Phase 3 N=34 Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Gaucher Disease, Type 1
Primary: Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. — 1.624; 1.488 gram per deciliter (g/dl)
Phase 3 N=48 Voriconazole For Chronic Bronchopulmonary Aspergillosis
Aspergillosis
Primary: Number of Subjects With Successful Global Outcome at 6 Months: Chronic Bronchopulmonary Aspergillosis — 13; 28 participants
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
Phase 2 N=44 Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment
Graft Versus Host Disease
Primary: Number of Participants Experiencing Treatment Success — 8 Participants
Phase 2 N=38 Pentostatin in Treating Patients With Refractory Chronic Graft-Versus-Host Disease
Graft Versus Host Disease
Primary: Response Rate — 20 percentage of participants
N/A N=17 Cytokines in Papillon-Lefèvre Syndrome
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml