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
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=94 ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
Acute Hepatic Porphyria · Acute Intermittent Porphyria · Porphyria, Acute Intermittent
Primary: Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP) — 12.52; 3.22 annualized attack rate — p=<0.0001
Phase 2 N=48 Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Primary: Remission — 6.1; 6.9 months
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 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
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 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
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 2 N=16 A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Acute Intermittent Porphyria
Primary: Percentage of Participants With Adverse Events (AEs) — 100 percentage of participants
Phase 2 N=22 Hydroxyproline Influence on Oxalate Metabolism
Hyperoxaluria
Primary: Mean Percent Conversion of Hydroxyproline (Hyp) to Urinary Oxalate (UOx) — 12.8; 32.9; 14.8 percent converted
Phase 2 N=47 Safety Study of Ornithine Phenylacetate to Treat Patients With Acute Liver Failure/Severe Acute Liver Injury
Acute Liver Failure · Acute Liver Injury
Primary: Number of Participants That do Not Tolerate the Administered Dose and Had Grade 3 or 4 Treatment Emergent Adverse Events as a Measure of Safety and Tolerability — 0; 0…
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=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 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
N/A N=28 Chronic Liver Disease in Urea Cycle Disorders
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
N/A N=455 The Association With Peptic Ulcer Disease and Hepatic Vein Pressure Gradient
Cirrhosis · Chronic Liver Disease
Primary: The Association of HVPG and PUD in Patients With Liver Cirrhosis — 14.5; 15.3 mmHg
Phase 3 N=46 Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
Urea Cycle Disorders
Primary: The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. — 976.6; 865.35 μmol∙h/L
Phase 2 N=32 Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism
Aldehyde Dehydrogenase-2 (ALDH2) Deficiency
Primary: Number of Participants With Adverse Events (AEs), Serious AEs, and AEs Leading to Study Discontinuation — 13; 6; 8; 21 Participants
Phase 3 N=286 Study to Evaluate Adverse Events and Change in Disease Activity in Adult and Adolescent Participants With Acute Hepatitis C Virus (HCV) Infection on Treatment With Oral Tablets of Glecaprevir (GLE)/Pibrentasvir (PIB)
Hepatitis C Virus (HCV)
Primary: Percentage of Participants Achieving Sustained Virologic Response 12 Weeks Post-treatment (SVR12) in the Intention-to-Treat (ITT) Population — 96.2 percentage of…
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 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=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
N/A N=46 Neurologic Injuries in Adults With Urea Cycle Disorders
Brain Diseases, Metabolic, Inborn · Urea Cycle Disorder · Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol by MRS — 4.97; 3.66; 3.78; 4.50 mM — p=0.003
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 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
Phase 3 N=21 A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 · Primary Hyperoxaluria
Primary: Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6 — -33.33 percent change
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