Clinical trials: Skin Abnormalities
Pompe Disease, Glycogen Storage Disease Type II
A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease.
- Trial name
- NCT00515398
- Testing
- Observation
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Amicus Therapeutics
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
Rhinitis, Allergic, Asthma, Allergic
Food-specific IgG4-guided Elimination Diets Improve Allergy Symptoms in Children
Allergic diseases in children are major public health concerns due to their widespread and rising prevalence. The most classic pathway to trigger allergy is type I allergy mediated by immunoglobulin E(IgE), but the role of…
- Conditions
- Rhinitis, Allergic, Asthma, Allergic, Conjunctivitis, Allergic, Atopic Dermatitis
- Trial name
- NCT06063044
- Where
- China
- Volunteers
- 407 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
MPS II, Hunter Syndrome (MPS II)
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system.
- Trial name
- NCT07236606
- Testing
- RGX-121-3102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- REGENXBIO Inc.
Ehlers-Danlos Syndrome, Multiple Sclerosis
Vascular Fundus Changes in Patients With High Probability of Chronic Cerebrospinal Venous Insufficiency (CCSVI)
The investigators propose that evidence of chronic cerebrospinal venous insufficiency (CCSVI) may be evident in the vasculature of the fundus.
- Trial name
- NCT01356134
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Genetic Disease Investigators
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Hereditary Angioedema
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This is an open-label, multicenter extension trial to evaluate the long-term safety of KVD900 in patients who are 12 years or older with HAE type I or II.
- Trial name
- NCT05505916
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 71 sites in United States, Australia and 21 more
- Volunteers
- 145 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Atopic Dermatitis, Type 1 Hypersensitivity
Allergic Disease Onset Prevention Study
This is a Phase 1b/2, randomized, double-blind, multi-center study to evaluate the safety, tolerability, and preliminary clinical efficacy of STMC-103H in neonates and infants at risk for developing allergic disease (Type 1…
- Trial name
- adored (NCT05003804)
- Testing
- STMC-103H
- Where
- 31 sites in United States, Australia and 1 more
- Volunteers
- 283 people, healthy volunteers welcome
- Sponsor
- Siolta Therapeutics, Inc.
Fabry's Disease
Analysis of the Nervous System in Patients With Fabry's Disease
Fabry's disease a genetic disorder (X-linked recessive) due to the absence of the enzyme ceramidetrihexosidase. The disease is characterized by abnormal collections of glycolipids in cells (histiocytes) within blood vessel walls…
- Trial name
- NCT00001491
- Where
- United States
- Volunteers
- 325 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Epidermolysis Bullosa
Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
This is a single-center study to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa.
- Trial name
- NCT02793960
- Testing
- topical BPM31510 3.0% Cream
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Shasa Hu
Atopic Dermatitis
Adjunctive Oral Iron Supplementation in Atopic Dermatitis
The present randomized controlled trial aims to evaluate the effect of adjunctive oral iron supplementation on disease severity and quality of life in adolescents with mild to moderate atopic dermatitis.
- Trial name
- NCT07613190
- Testing
- Oral iron
- Volunteers
- 124 people
- Sponsor
- Cairo University
Crohn Disease, Ulcerative Colitis
Expanded Access to Upadacitinib
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to upadacitinib prior to approval by the local regulatory agency. Availability will depend on territory eligibility.
- Conditions
- Crohn Disease, Ulcerative Colitis, Idiopathic Arthritis (Including sJIA, pJIA, or JPsA), Atopic Dermatitis
- Trial name
- NCT04159597
- Testing
- Upadacitinib
- Sponsor
- AbbVie
Atopic Dermatitis
Clobetasol Topical Oil for Children With Moderate to Severe Atopic Dermatitis
Open-Label Study designed to evaluate the HPA axis suppression potential of Clobetasol Topical Oil and pharmacokinetic safety / systemic exposure to clobetasol when Clobetasol Topical Oil is applied to pediatric subjects with…
- Trial name
- NCT03847389
- Testing
- Clobetasol propionate 0.05% Topical Oil
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Hill Dermaceuticals, Inc.
Hereditary Angioedema (HAE)
Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.
- Trial name
- NCT00457015
- Testing
- ecallantide, Phosphate Buffer Saline (PBS), pH 7.0
- Where
- 45 sites in United States, Canada and 1 more
- Volunteers
- 96 people
- Sponsor
- Shire
Jaundice, Neonatal, Hyperbilirubinemia, Neonatal
Identification of Jaundice in Newborns Using Smartphones
Neonatal jaundice is a common and most often harmless condition. However, when unrecognized it can be fatal or cause serious brain injury. Three quarters of these deaths are estimated to occur in the poorest regions of the world.
- Trial name
- NCT04182555
- Testing
- Bilirubin estimates from Smartphone Application, Bilirubin concentration measured in standard blood samples, Bilirubin estimates from standard transcutaneous device
- Where
- 2 sites in Norway
- Volunteers
- 220 people
- Sponsor
- St. Olavs Hospital
Atopic Dermatitis, Food Allergy
Skin Barrier Abnormalities and Oxidative Stress Response
This is a prospective, single-site controlled observational study designed to comprehensively determine whether children and adults with atopic dermatitis (AD) and food allergy (FA) have skin abnormalities which distinguish them…
- Trial name
- NCT04606615
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- National Jewish Health
Epidermolysis Bullosa, Epidermolysis Bullosa Acquisita
Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in…
- Conditions
- Epidermolysis Bullosa, Epidermolysis Bullosa Acquisita, Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- Trial name
- IV IgG (NCT06834035)
- Testing
- Immunoglobulin G
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- M. Peter Marinkovich
Tuberous Sclerosis Complex
Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC
The purpose of this study is to investigate the efficacy and safety of NPC-12Y gel compared with placebo for skin lesions associated with tuberous sclerosis.
- Trial name
- NCT05495425
- Testing
- NPC-12Y gel
- Where
- 5 sites in Japan
- Volunteers
- 43 people
- Sponsor
- Nobelpharma
Hunter Syndrome
Safety and Efficacy of Hunterase
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
- Trial name
- GC1111 (NCT01645189)
- Testing
- Hunterase
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Green Cross Corporation
Mucopolysaccharidosis (MPS)
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01155778
- Testing
- Recombinant human heparan N-sulfatase (rhHNS)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire