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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=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 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 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 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=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=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
N/A N=11 Blue Light Phototherapy
Grover's Disease · Psoriasis Vulgaris
Primary: Efficacy of Blue Light Therapy for Patients With Grover's Disease — 27; 6 lesion count
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=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=90 Trial of Oral Valproic Acid for Retinitis Pigmentosa
Retinitis Pigmentosa
Primary: Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter — -122.9; -112.0; -293.7; -237.1 Visual field area (degrees squared) — p=0.035
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=16 Evaluation of the Protection Activity of Microfine Titanium Dioxide (Ti02), Pigmentary Ti02 and Bisoctrizole and Their Combinations in Voluntary Patients With Idiopathic Solar Urticaria (SU)
Idiopathic Solar Urticaria
Primary: Assessment of the Minimal Urticaria Dose (MUD) on Each Test Site, After Application of the Test Products — 4.33; 3.93; 3.80; 3.67 Photodermatosis Protection Factor
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=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=10 Early Molecular Changes in Vitiligo After Narrowband Ultraviolet Therapy
Vitiligo · Dermatology/Skin - Other
Primary: Molecular Changes From NB-UVB Therapy
N/A N=40 Potential Impact of Patient Vitamin D Status in AK Response to MAL-PDT
Actinic Keratosis
Primary: Changes From Baseline in Actinic Keratosis - Lesion Base Count. — 1.15; 2.65; 1.15; 2.60 lesions — p=<0.001
N/A N=340 Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau Disease
Primary: Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant…
N/A N=17 Combined Photodynamic and Pulsed Dye Laser Treatment of Port Wine Stains
Port Wine Stains
Primary: Blanching of Port Wine Stain Birthmark — 16 participants
Phase 2 N=20 Photodynamic Therapy (PDT) With Methyl Aminolevulinate (MAL) Cream in Patients With Skin Type V or IV With Acne Vulgaris
Acne Vulgaris
Primary: Proportion of Patients With Moderate to Severe Hypopigmentation and Hyperpigmentation Assessed After Treatment — 0; 0 participants
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 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=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=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
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 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