Phase 1 N=15 Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Pachyonychia Congenita
Primary: Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels — 15; 15; 15; 14 participants
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=19 A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
Congenital Ichthyosis
Primary: Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) — 7; 7; 5; 5 participants
N/A N=17 Cytokines in Papillon-Lefèvre Syndrome
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml
Phase 3 N=174 Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)
Basal Cell Nevus Syndrome
Primary: Number of New BCCs Per Participant — 2.84; 4.03 New basal cell carcinomas
Phase 3 N=15 A Placebo-Controlled Double-Blind Study on the Safety and Efficacy of Etanercept in Palmoplantar Pustulosis
Palmoplantaris Pustulosis
Primary: Percentage Change in Palmoplantar Pustulosis Severity Index (PPPASI) Before Crossover — 30.1; 7.1 Percentage change
Phase 4 N=22 Use of Platelet-rich Plasma (PRP) Therapy in Patients With Brittle Nail Syndrome
Nail Diseases
Primary: Physician Global Improvement Assessment (PGIA) — 5; 2; 7; 1 Fingernails
Phase 2 N=8 Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis Bullosa Simplex · Epidermolysis Bullosa Simplex Kobner · Weber-Cockayne Syndrome
Primary: Foot Health Status Questionnaire, Foot Function Domain Score — 61.7; 49.3; 60.2; 56.3 score on a scale
Early Phase 1 N=13 An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses
Ichthyosis
Primary: Reduction in Total Ichthyosis Severity Score — -12.2 units on a scale
N/A N=8 308nm Excimer Laser for Treatment of Fingernail Psoriasis
Nail Psoriasis
Primary: Modified NAPSI Score (Nail Psoriasis Severity Index) — 6.3; 6.0 units on a scale
Phase 4 N=12 A Study Evaluating the Efficacy and Tolerability of Oral Apremilast for the Treatment of Nail Psoriasis
Psoriatic Nail · Psoriasis Vulgaris · Psoriasis
Primary: Mean Percent Change of mNAPSI (Modified Nail Area Psoriasis Severity Index) at Week 36 Compared to Baseline for All Nails. — 64.1 percentage of reduction in mNAPSI score…
Phase 2 N=66 N-Acetyl Cysteine in Pathologic Skin Picking
Pathologic Skin Picking · Neurotic Excoriation · Psychogenic Excoriation
Primary: Yale Brown Obsessive Compulsive Scale (YBOCS) Modified for PSP (NE-YBOCS) — 18.8; 17.6; 16.1; 17.3 units on a scale
N/A N=35 Genetic Screening for Filaggrin Mutation in Atopic Dermatitis and Ichthyosis Vulgaris in the African American Population
Atopic Dermatitis · Ichthyosis Vulgaris
Primary: Heterozygous for Filaggrin (FLG) Null Mutations — 1; 4 participants
N/A N=65 CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Pantothenate Kinase-Associated Neurodegeneration
Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50
Phase 2 N=17 Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
Basal Cell Nevus Syndrome
Primary: Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline — 51.29; 26.63; 21.82 percentage…
Phase 2 N=58 A Study to Assess the Efficacy, Safety, and Tolerability of KPL-716 in Reducing Pruritus in Chronic Pruritic Diseases
Chronic Idiopathic Urticaria · Chronic Idiopathic Pruritus · Lichen Planus
Primary: Change From Baseline in Weekly Average WI-NRS at Week 8 — -4.3; -1.1; -4.3; -3.7 score on a scale — p=0.3980
N/A N=10 Dermatosis Papulosa Nigra
Facial Dermatoses · Seborrheic Keratoses
Primary: Percent Clearance of All Lesions — 88; 96; 92.5; 0 percentage of lesion clearance
Phase 2 N=35 A Study to Evaluate RIST4721 in Palmoplantar Pustulosis (PPP)
Palmoplantar Pustulosis
Primary: Log Transformed Ratio of Postbaseline Fresh Pustule Count at Day 28 to Baseline — 0.86; 0.53 Log transformed ratio — p=0.24
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=3 Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome
Netherton Syndrome
Primary: Blood Pimecrolimus Levels — 0; 0; 0.312; 1.260 ng/mL
Phase 3 N=237 Palmoplantar Pustular Psoriasis Efficacy and Safety With Secukinumab
Palmoplantar Pustular Psoriasis
Primary: Percentage of Participants With ppPASI 75 Response at Week 16 (Period 1) — 17.5; 26.6; 14.1 percentage of participants — p=0.5722
Phase 2 N=12 Topical Ruxolitinib Lichen Planus
Lichen Planus
Primary: Change in Modified Clinical Assessment Scale of Severity for Index Lesion Signs and Symptoms (mCAILS) — 11.0 score on a scale
Phase 2 N=4 An Open-Label, Proof-of-Concept Study of Ixekizumab in the Treatment of Pyoderma Gangrenosum
Pyoderma Gangrenosum
Primary: The Proportion of Subjects Achieving 2-point Reduction in the 5-point Investigator Global Assessment (IGA) for the Target Ulcer at Week 12 — 0 Participants — p=1
Phase 4 N=100 Apremilast in Palmo-Plantar Psoriasis
Palmo-plantar Psoriasis
Primary: Palmoplantar Psoriasis Physician Global Assessment (PPPGA) of 0 or 1 — 7; 2 Participants
Phase 2 N=20 The Efficacy and Safety of Secukinumab in Patients With Ichthyoses
Ichthyosis · Autosomal Recessive Congenital Ichthyosis · Lamellar Ichthyosis
Primary: Reduction at Week 16 in the Ichthyosis Area Severity Index (IASI) — 2.4; 4.3 units on a scale
Phase 2 N=21 Pimecrolimus Cream for Oral Lichen Planus
Oral Lichen Planus
Primary: The Primary Efficacy Variable Was the Change in the Investigator's Global Assessment of the Overall Severity of Disease From Baseline to Week 6. — 2.4; 2.45; 1.6; 2.27…
Phase 2 N=71 CP-690-550 Ointment For Chronic Plaque Psoriasis
Psoriasis
Primary: Percent Change From Baseline in Target Plaque Severity Score (TPSS) at Week 4 — -53.97; -41.01; -24.26; -17.24 percent change
Phase 2 N=16 Topical Ruxolitinib in Chronic Hand Dermatitis
Chronic Hand Dermatitis
Primary: Proportion of Patients With Chronic Hand Dermatitis Improvement as Assessed by Investigators Global Assessment — 0.53 proportion of participants
Phase 2 N=7 Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica · Epidermolysis Bullosa
Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds
N/A N=28 Efficacy and Safety of Ustekinumab in Patients With Moderate to Severe Palmar Plantar Psoriasis
Moderate to Severe Palmar Plantar Psoriasis
Primary: Percentage of Patients Achieving a Palmar/Plantar PGA Score of 0 or 1 at Week 16. — 0.375 percentage of participants
Phase 2 N=59 A Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in Adults With Palmoplantar Pustulosis
Palmoplantar Pustulosis
Primary: Change From Baseline in Palmoplantar Pustulosis Psoriasis Area Severity Index (PPPASI) — -6.0; -6.1 score on a scale — p=0.944