Clinical trials: Congenital Diseases
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
Dermatitis, Atopic
Study Of Atopic Dermatitis In Pediatrics
To verify of cetirizine dry syrup to ketotifen dry syrup in the change in the severity of pruritus of the treatment period.
- Trial name
- NCT00257569
- Testing
- Cetirizine Dry Syrup
- Volunteers
- 278 people
- Sponsor
- GlaxoSmithKline
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07159841
- Testing
- Atumelnant
- Where
- 52 sites in United States, Argentina and 11 more
- Volunteers
- 153 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Hepatitis B
To Evaluate Immunogenicity & Safety of GSK Bio's DTPa-HBV-IPV/Hib (Mixed Vaccine) and DTPa-IPV/Hib + HBV Vaccines
This study assessed the immunogenicity and safety of two vaccination regimens that employed either GSK Biologicals' combined DTPa-HBV-IPV/Hib vaccine or DTPa-IPV/Hib vaccine.
- Trial name
- NCT00366366
- Testing
- Infanrix-Hexa
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Atopic Dermatitis
Dose-Finding Study of M516102 in the Treatment of Pruritus Associated With Atopic Dermatitis
This study is to investigate the efficacy and safety of M516102 and to evaluate the dose response relationship of M516102 and placebo, in the treatment of pruritus associated with atopic dermatitis.
- Trial name
- NCT00980629
- Testing
- M516102
- Where
- 4 sites in France, Germany and 2 more
- Sponsor
- Maruho Co., Ltd.
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Primary Immune Deficiency
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
- Trial name
- NCT04640142
- Testing
- Newnorm
- Where
- 23 sites in United States, Germany and 5 more
- Volunteers
- 50 people
- Sponsor
- Octapharma
Dermatitis, Atopic
Intervention Study to Evaluate a Probiotic in Mild Atopic Dermatitis Young Patients
The aim of the study is to evaluate the beneficial effect of a probiotic preparation with an antiinflammatory and modulating activity on immunological processes, with positive results on different inflammatory and atopic…
- Trial name
- NCT02585986
- Testing
- Probiotic
- Where
- Spain
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Biopolis S.L.
Epilepsy, Dravet Syndrome
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- GWPCARE5 (NCT02224573)
- Testing
- GWP42003-P
- Volunteers
- 681 people
- Sponsor
- GW Research Ltd
Atopic Dermatitis
A Study to Determine the Efficacy of ZPL-3893787 in Subjects With Atopic Dermatitis
This was a randomized, double blind, placebo controlled, parallel group study in approximately 90 subjects with moderate-severe AD Eczema Area and Severity Index (EASI) ≥12 and ≤ 48 (0-72 scale).
- Trial name
- NCT02424253
- Testing
- ZPL-3893787
- Where
- 17 sites in Belgium, Germany and 2 more
- Volunteers
- 98 people
- Sponsor
- Ziarco Pharma Ltd
Cystic Fibrosis
Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P.
- Trial name
- NCT03905642
- Testing
- Arikayce™
- Where
- 11 sites in Belgium, Hungary and 5 more
- Volunteers
- 49 people
- Sponsor
- Insmed Incorporated
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia…
- Trial name
- NCT05447494
- Testing
- Low-dose CAN103, High-dose CAN103
- Where
- China
- Volunteers
- 40 people
- Sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
Hemophilia A
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation…
- Trial name
- NCT00157053
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 24 sites in United States, Austria and 7 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Inherited Epidermolysis Bullosa
Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
The purpose of this study was to compare intra-individually the reepithelialization of skin lesion(s) in inherited Epidermolysis bullosa (either 1 wound ≥10 cm2 and ≤200 cm2 in size divided in 2 equal halves or 2 comparable…
- Trial name
- NCT01294241
- Testing
- Oleogel-S10, Mepilex® soft silicone faced polyurethane foam dressing
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Birken AG
Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema
Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
A Phase 2b study investigating the efficacy of zabalafin in people with mild to moderate atopic dermatitis (eczema).
- Conditions
- Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema, Atopic Dermatitis, Atopic Dermatitis (AD)
- Trial name
- CLEAR-AD1 (NCT06855745)
- Testing
- zabalafin
- Where
- 12 sites in Australia
- Volunteers
- 72 people
- Sponsor
- Alphyn Biologics
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.