N/A N=72 Clinical Trial of Factor XIII (FXIII) Concentrate
Hemophilia · Factor XIII Deficiency
Primary: Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions — 30; 1; 0; 3 Number of bleeds
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=61 An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Factor XIII Deficiency
Primary: Adverse Events — 42; 2; 2 participants
Phase 3 N=63 Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Primary: Adverse Events (AEs)(Serious and Non-serious) — 920; 19; 901 Events
Phase 3 N=41 Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Primary: Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period — 0.138 bleeding episodes per subject…
Phase 3 N=6 Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Primary: Area Under the Concentration vs. Time Curve (AUC) — 250.25 IU*h/mL
Phase 2 N=15 A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Factor XIII Deficiency
Primary: Peak FXIII Concentration at Steady State — 0.9 Units/mL
Phase 3 N=41 A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Factor XIII Deficiency
Primary: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) — 0…
Phase 2 N=32 Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency · Carnitine Palmitoyltransferase 2 (CPT2) Deficiency · Mitochondrial Trifunctional Protein (TFP) Deficiency
Primary: Energy Expenditure — -73; 107 kcal/day
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Phase 2 N=25 Defibrotide TMA Prophylaxis Pilot Trial
Thrombotic Microangiopathies
Primary: Percent of Total Doses of Defibrotide That Were Missed [Feasibility] — 0.7 percentage of missed doses
N/A N=51 Thromboelastography-derived Functional Fibrinogen Levels
Focus of Study · Coagulation Status of Cardiac Surgery Patient · Reducing Excess Blood Transfusion
Primary: Change in Thromboelastography-derived Functional Fibrinogen Level (FLEV) — -1.1 mg/dL
Phase 1 N=14 Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
Phase 3 N=6 Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Primary: Number of Treatment Emergent (Serious and Non-serious) Adverse Events — 100; 2; 98 number of events
Phase 3 N=6 A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic Thrombocytopenic Purpura (TTP)
Primary: Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment — 0; 0; 1 Participants
Phase 2 N=14 Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
Homocystinuria
Primary: Difference in Thiobarbituric Acid Reactive Substances (TBARS) in Individuals With Cystathionine Beta Synthase Deficient Homocystinuria (CBSDH) Pre and Post Taurine…
Phase 4 N=251 Survey of Inhibitors in Plasma-Product Exposed Toddlers
Hemophilia A
Primary: To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the…
Phase 2 N=98 Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Non-Neoplastic Hematologic and Lymphocytic Disorder
Primary: Preliminary Efficacy — 83; 10 Participants
Phase 2 N=5 A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Congenital Protein C Deficiency
Primary: PK Part: Protein C Activity Level of TAK-662 — 0.000; 1.746; 1.616; 1.458 international unit per milliliter(IU/ml)
Phase 3 N=18 Recombinant Human Antithrombin (rhAT) in Patients With Hereditary Antithrombin Deficiency Undergoing Surgery or Delivery
Antithrombin III Deficiency
Primary: Incidence of Thromboembolic Events Acute Deep Venous Thrombosis (DVT) and/or Thromboembolic Events Other Than Acute Deep Venous Thrombosis (DVT) — 0 Participants
Phase 3 N=220 Phase 3 Triiodothyronine Supplementation for Infants After Cardiopulmonary Bypass
Congenital Heart Defects
Primary: Time To Extubation — 46.9; 48.0 hours — p=<.05
N/A N=25 Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Premature Infant Disease · Transfusion Related Complication · Fetal Hemoglobin
Primary: Median Percentage of HbF at 32 Weeks of Post Menstrual Age — 93.2; 90.8; 58.0; 69.0 percentage of total Hb — p=<0.0001
Phase 2 N=3 Dose Confirmation Trial of AAV5-hFIXco-Padua
Hemophilia B
Primary: Factor IX Activity Levels — 30.6 Factor IX activity (%)
Phase 3 N=330 American Trial Using Tranexamic Acid in Thrombocytopenia
Thrombocytopenia
Primary: Bleeding Within 30 Days — 73; 78 Participants
Phase 3 N=102 A Trial of Antenatal Treatment of Alloimmune Thrombocytopenia
Alloimmune Thrombocytopenia · Fetal Alloimmune Thrombocytopenia
Primary: Number of Newborns With a Birth Platelet Count > 50,000/uL — 48; 47 number of newborns with >50,000 pets
Phase 1 N=9 Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Alpha 1-Antitrypsin Deficiency
Primary: Adverse Events Possibly, Probably or Definitely Related to Study Drug — 2; 2; 3; 2 participants
Phase 3 N=35 Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
Congenital Afibrinogenemia · Congenital Hypofibrinogenemia
Primary: Single-dose Pharmacokinetics (PK) of BT524: Terminal Elimination Half-life (t1/2) for Fibrinogen Antigen — 67.9 hours
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 1 N=4 Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Primary: Metabolic Stress
Phase 2 N=12 Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
Tetrahydrobiopterin Deficiencies · Hyperphenylalaninemia, Non-Phenylketonuric
Primary: Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints — 132.9; 72.2; 315.0; 104.1 μmol/L
N/A N=49 Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol — 2.61; 1.66; 2.23; 2.69 mM — p=0.001