Clinical trials: Genetic Skin Disease
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BMN 110 Phase 3B in Australian Patients
There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment…
- Trial name
- MOR-AUS (NCT01966029)
- Testing
- BMN 110
- Where
- 5 sites in Australia
- Volunteers
- 13 people
- Sponsor
- BioMarin Pharmaceutical
Melanoma, Carcinoma, Non-Small- Cell Lung
A Study of SEA-CD40 Given With Other Drugs in Cancers
This trial is being done to see if an experimental drug (SEA-CD40) works when it's given with other cancer drugs to treat some types of cancer. It will also study side effects from the drug. There are 2 parts in this trial.
- Trial name
- NCT04993677
- Testing
- SEA-CD40, pembrolizumab (KEYTRUDA®), pemetrexed
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 77 people
- Sponsor
- Seagen Inc.
Atopic Dermatitis
Safety and Efficacy of FURESTEM-AD Inj. in Patients With Moderately Subacute and Chronic Atopic Dermatitis (AD)
The purpose of phase I clinical trial is to evaluate safety and efficacy in subjects with over Moderately subacute and chronic Atopic Dermatitis after inject.
- Trial name
- NCT01927705
- Testing
- FURESTEM-AD Inj.
- Where
- South Korea
- Volunteers
- 34 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Dermatitis, Atopic
Long-Term Follow-up Study of FURESTEM-AD in Patients With Atopic Dermatitis(K0106)
Long-Term Follow-up Study of Subjects With Moderate to Severe Chronic Atopic Dermatitis Who Had Administered FURESTEM-AD Inj. in K0106 Study
- Trial name
- NCT07582237
- Where
- South Korea
- Volunteers
- 262 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Dermatitis, Atopic
Distribution of Tacrolimus in Skin, Atopic Dermatitis
This study was designed to further increase the understanding of the pharmacokinetics of tacrolimus in the affected skin of atopic dermatitis patients following repeated topical application of tacrolimus ointment 0.1%.
- Trial name
- NCT00534508
- Testing
- Tacrolimus Ointment 0.1%
- Where
- Ireland
- Volunteers
- 14 people
- Sponsor
- Astellas Pharma Inc
Familial Partial Lipodystrophy
The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in a randomized treatment (RT) period in participants with familial partial lipodystrophy (FPL).
- Trial name
- NCT02527343
- Testing
- volanesorsen
- Where
- 12 sites in United States, Belgium and 5 more
- Volunteers
- 40 people
- Sponsor
- Akcea Therapeutics
Hereditary Angioedema
A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema
The goal of this clinical trial is to test the drug navenibart in participants with hereditary angioedema (HAE). One group of participants will get 1 dose of navenibart, and 2 other groups will get 2 doses of navenibart.
- Trial name
- NCT05695248
- Testing
- Navenibart
- Where
- 20 sites in United States, Bulgaria and 4 more
- Volunteers
- 29 people
- Sponsor
- Astria Therapeutics, Inc.
Atopic Dermatitis
A Study to Evaluate the Efficacy and Safety of LNK01004 Ointment in Adults With Atopic Dermatitis
This study will evaluate the efficacy and safety of two concentrations (0.3 percent [%] and 1%) of twice daily LNK01004 ointment for the topical treatment in adult subjects with atopic dermatitis.
- Trial name
- NCT07071610
- Testing
- LNK01004 ointment 0.3%, LNK01004 ointment 1.0%, Vehicle
- Where
- China
- Volunteers
- 75 people
- Sponsor
- Lynk Pharmaceuticals Co., Ltd
Hidradenitis Suppurativa, Atopic Dermatitis
A Study to Assess Molecular Changes in Adult Participants With Moderate to Severe Hidradenitis Suppurativa or With Moderate to Severe Atopic Dermatitis Receiving Subcutaneous Injections of Lutikizumab
Hidradenitis suppurativa (HS) and atopic dermatitis (AD) are chronic inflammatory skin diseases that lead to the development of skin lesions and symptoms such as pain and discomfort.
- Trial name
- NCT06524635
- Testing
- Lutikizumab
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- AbbVie
Skin Quality, Skin Laxity
Prospective Clinical Investigation to Evaluate the Safety and Effectiveness of Juläine™ in Improving Gluteal Skin Laxity in Adults.
This study aims to evaluate the safety and effectiveness of Juläine™ (poly-L-lactic acid) injections for the treatment of buttock skin laxity.
- Trial name
- NCT07332650
- Testing
- Poly-L-Lactic Acid (Juläine™)
- Volunteers
- 100 people
- Sponsor
- Nordberg Medical AB
Healthy Volunteers
Study to Access the Relative Bioavailability of Subcutaneous Dose of Nemolizumab When Administered Via Auto-Injector Versus Dual-Chamber Syringe
This study was to compare the rate and extent of absorption of a single dose of nemolizumab administered with auto-injectors [AI] (test) versus dual-chamber syringes [DCS] (reference) under controlled conditions in healthy adult…
- Trial name
- NCT05405985
- Testing
- Nemolizumab
- Where
- 2 sites in United States
- Volunteers
- 192 people, healthy volunteers welcome
- Sponsor
- Galderma R&D
Hereditary Angioedema
A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult…
- Trial name
- NCT05259917
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 66 sites in United States, Australia and 19 more
- Volunteers
- 136 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Dermatitis Atopic
Observational Evaluation of Atopic Dermatitis in Pediatric Patients
Primary Objectives: To describe the characteristics of pediatric patients with moderate to severe atopic dermatitis (AD) whose disease is not adequately controlled with topical therapies or when those therapies are not medically…
- Trial name
- PEDISTAD (NCT03687359)
- Testing
- Standard of care
- Where
- 182 sites in United States, Argentina and 18 more
- Volunteers
- 1,860 people
- Sponsor
- Sanofi
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Atopic Dermatitis
Multiple-Dose Pharmacokinetics and Potential for Adrenal Suppression Following Treatment With Halobetasol Spray in Patients With Atopic Dermatitis
This is an open-label, safety study designed to assess the multiple-dose pharmacokinetics and potential for adrenal suppression following topical treatment with halobetasol propionate 0.05% spray applied twice-daily in adult…
- Trial name
- NCT03245385
- Testing
- Treatment with Halobetasol propionate 0.05% topical spray
- Where
- 13 sites in United States
- Volunteers
- 20 people
- Sponsor
- Sun Pharmaceutical Industries Limited
Abdominal Pain, Functional Abdominal Pain
Epidemiological Analysis for Hereditary Angioedema Disease
An international, multicenter, epidemiological, observational study investigating the prevalence of Hereditary Angioedema (HAE) disease among participants with recurrent episodes of abdominal pain of no obvious etiology.
- Trial name
- EHA (NCT03558009)
- Where
- 35 sites in Germany, Italy and 4 more
- Volunteers
- 2,318 people
- Sponsor
- CENTOGENE GmbH Rostock
Atopic Dermatitis
A Trial to Evaluate the Efficacy and Safety of Different Doses of LEO 138559 in Adults With Moderate-to-severe Atopic Dermatitis
The purpose of this trial is to test different doses of the trial medicine (LEO 138559) and see how well they work and how safe they are at treating moderate to severe atopic dermatitis in adults.
- Trial name
- NCT05923099
- Testing
- LEO 138559
- Where
- 73 sites in United States, Canada and 9 more
- Volunteers
- 262 people
- Sponsor
- LEO Pharma
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 18 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Atopic Dermatitis
Phase 1 Study of QX-4533 in Healthy Volunteers and Patients With Moderate-to-Severe Atopic Dermatitis
The goal of this clinical trial is to evaluate the safety and tolerability of QX-453, an investigational treatment, in healthy Chinese volunteers and patients with moderate-to-severe atopic dermatitis.
- Trial name
- NCT07800104
- Testing
- QX4533
- Where
- China
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- QuantX Biosciences, Inc.
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