Phase 2 N=16 PTG-300 in Subjects With Hereditary Hemochromatosis
Hereditary Hemochromatosis
Primary: Effect of PTG-300 on Transferrin Saturation — -6.07 percent saturation
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=49 Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
Iron Overload · Hereditary Hemochromatosis
Primary: Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) — -389.1; -610.5; -571.6 µg/L
Phase 2 N=70 A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Hereditary Hemochromatosis
Primary: Effect of LJPC-401 Versus Placebo on Blood Iron Levels — -32.8; -2.5 Percent Change — p=< 0.0001
N/A N=142 Massive Iron Deposit Assessment
Iron Overload · Excessive Body Iron Burden
Primary: Hepatic Iron Content in the Liver Using Liver Biopsy — 19.8 mcg
Phase 4 N=243 An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)
Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia
Primary: Percentage of Participants With Cardiac and Liver Iron Overload. — 10; 22; 4; 0 Percentage of participants
N/A N=50 Diet and Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia (HHT)
Primary: Number of Participants That Achieving Personal Recommended Intake of Iron — 43 Participants
Phase 2 N=48 Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Primary: Remission — 6.1; 6.9 months
Phase 3 N=40 Heme Iron Polypeptide for Iron Deficiency Anemia in Chronic Renal Failure
Anemia · Renal Failure
Primary: Hemoglobin Concentration at 6 Months — 117; 113 g/L
Phase 2 N=60 Copper Histidine Therapy for Menkes Diseases
Kinky Hair Syndrome
Primary: Gross Motor Development at 36 Mos of Age or at Death (Mos) — 13.743; 2.455; 15.667 Other - months
N/A N=21 Non-invasive Quantification of Liver Iron With MRI
Iron Overload
Primary: Per Subject Evaluable DICOM Data Sets From Liver MRI — 21 Participants
Phase 2 N=16 Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
Acute Undifferentiated Leukemia · Adult Acute Lymphoblastic Leukemia in Remission · Adult Acute Myeloid Leukemia in Remission
Primary: Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) — 7.8 10^9 Neutrophils per Liter
Phase 1 N=66 Study of SSP-004184 (FBS0701) in Healthy Adults and Elderly Subjects and in Subjects With Impaired Renal Function
Impaired Renal Function
Primary: Area Under the Plasma Concentration-time Curve (AUC) of SSP-004184 — 445892.7; 932688.3; 1531185.8; 1261081.6 ng*hr/ml
Phase 3 N=10 Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Primary: Number of Participants Showing Reduction or Elimination of Skin Blistering — 0; 8 participants
N/A N=156 Hereditary Haemorrhagic Telangiectasia Flight Safety Study
Hereditary Haemorrhagic Telangiectasia
Primary: HHT Patients Who by the Questionnaire Did or Did Not Self Report a Medical Complication During Flight — 77 percentage of patients
N/A N=11 Familial Hyperlipidemia Family Registry
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
Phase 2 N=9 Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
Thalassemia Major With Severe Transfusional Iron Overload
Primary: Number of Participants With Improvement in Liver Iron Concentration — 3 Participants
N/A N=16 Oral Iron for Erythropoietic Protoporphyrias
Erythropoietic Protoporphyria · EPP · X-linked Protoporphyria
Primary: Change in Erythrocyte Protoporphyrin Levels — 2314.6; 2240.4 µg/dL
N/A N=5 Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Primary: Number of LDLR Gene Mutations — 7 gene mutations
N/A N=77 Measurements of Body Iron Excretion of Healthy Adults
Healthy
Primary: Rate of Iron Excretion. — 1.07; 1.69; 1.08 milligrams per day — p=< 0.05
Phase 4 N=17 Feraheme As An MRI Contrast Agent For Pediatric Congenital Heart Disease
Pediatric Congenital Heart Disease
Primary: Composite Image Quality Score Among 7 Anatomical Structures. — 3.9; 2.7 score on a scale
N/A N=22 Does a Daily Iron Tablet Improve Anemia in Cystic Fibrosis
Cystic Fibrosis · Anemia, Iron-Deficiency
Primary: Change From Baseline in Hemoglobin Concentration (gm/dl) — 0.12; 0.09 gm/dl — p=0.81
Phase 3 N=1,683 Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Thalassemia · Sickle Cell Disease · Diamond Blackfan Anemia
Primary: Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events — 0; 0; 1; 4 Participants
Phase 2 N=30 Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Iron Overload Due to Repeated Red Blood Cell Transfusions
Primary: The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern. — 0; 0; 0; 0…
Phase 1 N=8 Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
Sickle Cell Disease
Primary: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide — 17.56; 32.95 μg/mL
Phase 2 N=51 Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
Transfusional Iron Overload · Beta-thalassemia
Primary: Change From Baseline in Liver Iron Concentration (LIC) at 96 Weeks — -1.75 mg/g — p=0.0765
N/A N=38 Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis
Superficial Siderosis
Primary: Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing — 13; 18; 0 Participants
N/A N=2,620 Family Health History in Diverse Care Settings (FHH)
Diabetes · Heart Disease · Cancer
Primary: Number of Participants With Uptake of Genetic Counseling for Those at Risk of Hereditary Conditions at 1 Year — 571; 49; 32; 31 Participants
Phase 2 N=12 Intravenous Iron Metabolism in Restless Legs Syndrome
Restless Legs
Primary: Duration Off Treatment — 6; 3; 2; 0 participants
N/A N=150 Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
Erythropoietic Protoporphyria · EPP · X-Linked Protoporphyria
Primary: The Hospital Anxiety and Depression Scale (HADS) — 4.6; 1.9 score on a scale
Phase 2 N=28 Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
Beta-thalassemia · Iron Overload
Primary: Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2* — 9.92; 11.73; 1.77; 11.93 milliseconds (msec)