Phase 2 N=8 Gene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Primary: Safety of Reduced Conditioning Regimen — 8 Participants
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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=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 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
Phase 3 N=19 Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
Myelokathexis · Infections · Neutropenia
Primary: Severity of Infection — 12.5; 9 score on a scale (with no maximum)
Phase 2 N=8 A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
WHIM Syndrome
Primary: Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) — -12574.63; -6206.75; -6949.58; 24175.00 cells*hour/μL
Phase 3 N=12 Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
Von Willebrand Disease
Primary: Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. — 4.6; 3.7 Annualized number of bleeding episodes
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=10 Wilm's Tumor 1 Protein Vaccine to Treat Cancers of the Blood
Leukemia, Acute Myelogenous (AML) · Leukemia, Acute Lymphocytic (ALL) · Leukemia, Chronic Myelogenous (CML)
Primary: Toxicity — 5 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=47 Tofacitinib for Immune Skin Conditions in Down Syndrome
Down Syndrome · Alopecia Areata · Atopic Dermatitis / Eczema
Primary: Number of Serious Adverse Events (SAE) Definitely Related to Tofacitinib Treatment. — 0 Serious Adverse Events
Phase 1 N=6 AMD 3100 for Treatment of Myelokathexis
Neutropenia
Primary: Blood Neutrophil Counts. — 4.48 10^9 per Liter — p=<0.05
Phase 2 N=11 Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 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
Phase 3 N=15 Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
NLRC4-MAS · XIAP Deficiency
Primary: Prevention of Flares — 2.71; NA Weeks
N/A N=265 Type 3 Von Willebrand International Registries Inhibitor Prospective Study
Type 3 Von Willebrand's Disease
Primary: Centralized Factor VIII (FVIII) Procoagulant Activity (FVIII:C) Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis — 2.42 IU/dL
Phase 3 N=47 Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
Familial Cold Autoinflammatory Syndrome (FCAS) · Familial Cold Urticaria · Muckle-Wells Syndrome (MWS)
Primary: Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) — 2.4; 3.1; 2.1; 0.5 Units of a Scale — p=<.0001
Phase 2 N=21 Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
Williams Beuren Syndrome
Primary: Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography — 0.028; 0.012 mm
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
Phase 2 N=44 Study of BTK Inhibitor BGB-3111 in Chinese Subjects With Relapsed/Refractory Waldenström's Macroglobulinemia (WM)
Waldenström's Macroglobulinemia (WM)
Primary: Major Response Rate (MRR) as Assessed by the Independent Review Committee — 69.8 Percentage of participants — p=<0.0001
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 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=6 Immune Tolerance Study With Aldurazyme® (Laronidase)
Mucopolysaccharidosis I
Primary: Number of Participants Who Achieved Immune Tolerance Induction — 0; 1 participants
Phase 2 N=63 Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)
Acute Lymphoblastic Leukemia
Primary: Number of Participants With Transplant-related Mortality — 8 participants
Phase 1 N=21 Anti-CD19 White Blood Cells for Children and Young Adults With B Cell Leukemia or Lymphoma
ALL · B Cell Lymphoma · Leukemia
Primary: Number of Participants Who Received Preparative Chemotherapy Followed by Cluster of Differentiation (CD)19-Chimeric Antigen Receptor (CAR): CD19 CAR T-cells With < Grade…
Phase 2 N=33 Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia
Primary: Overall Response Rate of RAD001 in Patients With Previously Untreated WM — 22 participants
Phase 2 N=37 Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia
Primary: Overall Response (OR) Rate — 38 percentage of patients
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=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