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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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 2 N=48 Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Primary: Remission — 6.1; 6.9 months
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 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=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=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 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 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=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=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=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=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
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 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=28 Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients
Erythropoietic Protoporphyria
Clinuvel Europe Limited View details
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 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 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=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=52 A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Pyruvate Kinase Deficiency
Primary: Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period — 96.3; 100.0 percentage of participants
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=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 2 N=75 Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP
Erythropoietic Protoporphyria
Primary: Percent Change From Baseline in Whole Blood Metal-free PPIX Levels — 8.07; -21.48; -41.74 Percent Change
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 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
N/A N=187 PCORI Urea Cycle Disorder Study
Urea Cycle Disorders
Primary: Mortality — 18.4; 20.7 participant per 1000 person-years
N/A N=28 Sepsis-Associated Purpura Fulminans International Registry - Europe
Sepsis
Primary: Mortality — 11 Participants
Phase 4 N=30 Study of Daylight Photodynamic Therapy With Aminolevulinic Acid for Actinic Keratoses
Actinic Keratosis
Primary: Percent Change in the Number of Actinic Keratoses — -75 % change in number of actinic keratoses