Phase 2 N=48 Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Primary: Remission — 6.1; 6.9 months
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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
Phase 2 N=23 Harvoni Treatment Porphyria Cutanea Tarda
Porphyria Cutanea Tarda · Hepatitis C
Primary: Number of Participants With Resolution of Active PCT by 7 Months After Start of Therapy — 9 Participants
Phase 3 N=4 Sorbent Therapy of the Cutaneous Porphyrias
Erythropoietic Protoporphyria
Primary: Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance — 65.8 minutes
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=16 PTG-300 in Subjects With Hereditary Hemochromatosis
Hereditary Hemochromatosis
Primary: Effect of PTG-300 on Transferrin Saturation — -6.07 percent saturation
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
Phase 3 N=184 Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
EPP · XLP
Primary: Change From Baseline in Average Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight…
Phase 2 N=20 Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria
Acute Porphyrias
Primary: Difference in NRS Pain Score Between Baseline and 12 Hours — -0.2; -0.2 score on a scale
Phase 2 N=20 Pentoxifylline for Primary Biliary Cirrhosis
Primary Biliary Cirrhosis
Primary: Change in Serum Alkaline Phosphatase Levels. — -57.3 U/L — p=0.001
Phase 2 N=102 Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
Erythropoietic Protoporphyria (EPP)
Primary: Change From Baseline in Average Daily Time (Minutes) to First Prodromal Symptom Associated With Sunlight Exposure Between Hour Post Sunrise and 1 Hour Pre-Sunset at Week…
Phase 2 N=35 Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Phase 2 N=6 Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
Variegate Porphyria
Primary: The Change in Disease Severity in Patients With VP as Measured by CGIC. — 2.5 Scores on a scale
Phase 2 N=77 Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Erythropoietic Protoporphyria
Primary: Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days — 8.25; 0.75 Hours
Phase 3 N=93 Phase III Confirmatory Study in Erythropoietic Protoporphyria
Erythropoietic Protoporphyria
Primary: Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). — 69.4; 40.8 hours
Phase 2 N=28 Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients
Erythropoietic Protoporphyria
Clinuvel Europe Limited View details
Phase 2 N=16 Afamelanotide as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer Sodium
Patients Undergoing Photodynamic Therapy Using Porfimer Sodium
Primary: The First Day on Which Patients Are Free From Symptoms of Phototoxicity Following Phototesting
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 1 N=11 Silicon Phthalocyanine 4 and Photodynamic Therapy in Stage IA-IIA Cutaneous T-Cell Non-Hodgkin Lymphoma
Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma · Recurrent Mycosis Fungoides/Sezary Syndrome · Stage I Cutaneous T-cell Non-Hodgkin Lymphoma
Primary: MTD of Photodynamic Therapy — 150 J/cm^2
Phase 3 N=100 Multicentre Phase III Erythropoietic Protoporphyria Study
Erythropoietic Protoporphyria
Primary: Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) — 39; 45; 129; 179 Days
Phase 2 N=50 Pioglitazone to Treat Fatty Liver in People With HIV Infection
HIV Infections · Hepatic Steatosis · Insulin Resistance
Primary: Hepatic Steatosis
Phase 2 N=20 Photoactive Porphyrins (PAP) Levels After Topical Visonac Application in Acne Patients
Acne Vulgaris
Primary: Photoactive Porphyrins Level — 33.0; 24.8; 19.7 Fluorescence intensity unit
Phase 3 N=31 Afamelanotide in Patients Suffering From Polymorphic Light Eruption (PLE)
Polymorphic Light Eruption
Primary: Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale — 0.25; 0.47 score on a scale
Phase 4 N=105 Prevalence of Non-alcoholic Fatty Liver Disease (NAFLD) in Hispanics With Diabetes Mellitus Type 2 (T2DM) and Role of Treatment
Nonalcoholic Steatohepatitis
Primary: Liver Histology (Kleiner's et al Criteria, Hepatology 2005) — 7; 13; 24 Participants
Phase 3 N=168 Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)
High Risk Polycythemia Vera · High Risk Essential Thrombocythemia
Primary: Number of Participants With Complete Remission (CR) — 17; 19; 12; 13 Participants
Phase 3 N=74 Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Erythropoietic Protoporphyria
Primary: The Duration of Direct Sunlight Exposure Between 10:00 and 15:00 Hours on Days When Patients Did Not Report Phototoxicity-related Pain (Likert Pain Scale Score of 0)…
Phase 2 N=144 Pomalidomide for the Treatment of Bleeding in HHT
Telangiectasia, Hereditary Hemorrhagic
Primary: Change From Baseline Epistaxis Severity Score — -1; -1.1; -1.3; -1.2 units on a scale
Phase 2 N=10 Efficacy of Polypodium Leucotomos Extract Decreasing UVA Premutagenic and Photoaging Markers
Aging · Skin Abnormalities
Primary: Change in Common Deletion (CD) Value in DNA of Skin Biopsy Sample — -84; 217 percent change
Phase 3 N=247 Pioglitazone vs Vitamin E vs Placebo for Treatment of Non-Diabetic Patients With Nonalcoholic Steatohepatitis (PIVENS)
Liver Diseases
Primary: Number of Participants With Improvement in Non-alcoholic Fatty Liver Disease (NAFLD) Activity Defined by Change in Standardized Scoring of Liver Biopsies at Baseline and…
Phase 3 N=16 A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Erythropoietic Protoporphyria
Primary: Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). — 27.8; 59.7; 56.9 EPP-QoL score