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
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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
Phase 2 N=34 A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
Congenital Ichthyosis
Primary: Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement — 4; 7; 4 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 3 N=153 The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis
Ichthyosis
Primary: Change in Investigator Global Assessment (IGA) Score — 41.4; 44.9 percentage of responders — p=0.39
Phase 2 N=80 A Six Week Topical Cream Study for Subjects With Ichthyosis Vulgaris
Ichthyosis Vulgaris
Primary: Treatment Success — 21; 28 Participants
Phase 2 N=12 A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
Sjögren-Larsson Syndrome
Primary: Number of Participants Experiencing a Serious Adverse Event (SAE). — 0; 0 Participants
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
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 2 N=5 Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
Recessive Dystrophic Epidermolysis Bullosa
Primary: Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. — 5; 5; 4; 4 Fluorescence Intensity (MFI) for C7
N/A N=1,260 A Community-based Assessment of Skin Care, Allergies, and Eczema
Atopic Dermatitis · Atopic Disorders · Eczema
Primary: Provider-diagnosed Atopic Dermatitis — 217.7; 268.5 Participants with imputed values — p=0.019
Phase 2 N=6 Gentamicin for RDEB
Recessive Dystrophic Epidermolysis Bullosa
Primary: Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence — 31.8; 28.4; 65.6 C7 Expression (percent of normal skin)
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
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
Phase 3 N=596 Efficacy and Safety of a Retinoid in the Treatment of Severe Chronic Hand Eczema
Eczema
Primary: Number of Participants Who Responded as Per Physician's Global Assessment (PGA) at Week 24 — 58; 14; 60; 30 Participants — p=<0.001
Phase 2 N=4 Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml
Phase 3 N=51 A Test to Determine the Usefulness and Safety of a Cream Used on Babies With Dry Itchy Skin
Eczema
Primary: Eczema Area and Severity Index (EASI) on Day 3 - Change From Baseline — -1.783; -0.767 units on a scale — p=<0.0001
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
N/A N=442 Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
N/A N=9 Reversal of Epidermal Phenotype in Severe Atopic Dermatitis With Cyclosporine Therapy
Atopic Dermatitis · Eczema
Primary: SCORAD Change Score — 33.34 Change Score
N/A N=39 ABCA3 Gene and RDS in Late Preterm and Term Infants
Respiratory Distress Syndrome, Newborn
Primary: Mortality — 5; 3; 5 Participants
Phase 4 N=10 Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs