Clinical trials: Malformation
Antithrombin III Deficiency, Covid19
Antithrombin III in Infectious Disease Caused by COVID-19
The purpose of this research study is to see if participants who have SARS-CoV-2 and low levels of AT3 in the blood will benefit by being given AT3.
- Trial name
- NCT04899232
- Testing
- Antithrombin III
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Enrique Ginzburg
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or…
- Trial name
- NCT04179409
- Testing
- Amondys 45, Exondys 51, Vyondys 53
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Kevin Flanigan
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
Epidermal Nevus Syndrome
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels.
- Trial name
- ENS (NCT04320316)
- Testing
- Crysvita (burosumab-twza) Treatment
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Coarctation of the Aorta, Pulmonary Artery Stenosis
Growth Trial: Study of the Renata Minima Stent
The objective of the clinical investigation is to access clinical safety and effectiveness of the Minima Stent in neonates, infants, and young children requiring intervention for common congenital vascular stenosis (i.e.…
- Trial name
- NCT05086016
- Testing
- Catheterized Stenting
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Renata Medical
Atopic Dermatitis
A Study to Evaluate Long-Term Maintenance Treatment With Once Daily Crisaborole Ointment 2% in Pediatric and Adult Participants With Mild-to-Moderate Atopic Dermatitis
This study will evaluate crisaborole therapy once daily (QD) as a long-term topical maintenance therapy for the reduction of flare in responders to crisaborole twice daily (BID) treatment.
- Trial name
- NCT04040192
- Testing
- Crisaborole 2%, Vehicle
- Where
- 44 sites in United States, Australia and 4 more
- Volunteers
- 620 people
- Sponsor
- Pfizer
Foot Injury, Foot Deformity
Comparing Clinical Outcomes Using Two Insole Manufacture Techniques
The main purpose of this study is to find out if scanning the foot using a 3D scanner influences the effectiveness of custom made insoles, compared to the more traditional approach of taking a foam-box impression cast of the…
- Conditions
- Foot Injury, Foot Deformity, Foot Sprain, Feet, Flat
- Trial name
- NCT05444192
- Testing
- CAD/CAM insoles
- Where
- United Kingdom
- Volunteers
- 114 people
- Sponsor
- NHS Greater Glasgow and Clyde
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth…
- Trial name
- MOSAIC (NCT03094832)
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 50 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Hemophilia A
BAX 855 Continuation
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
- Trial name
- NCT01945593
- Testing
- BAX855
- Where
- 89 sites in United States, Australia and 21 more
- Volunteers
- 218 people
- Sponsor
- Baxalta now part of Shire
Ichthyosis Vulgaris
A Six Week Topical Cream Study for Subjects With Ichthyosis Vulgaris
The objective of this study is to determine and compare the safety and efficacy of topical 146-9251 cream and vehicle cream applied twice daily for 6 weeks in subjects with moderate to severe ichthyosis vulgaris (IV).
- Trial name
- NCT03173547
- Testing
- 146-9251 cream, Vehicle cream
- Where
- 7 sites in United States
- Volunteers
- 80 people
- Sponsor
- Crown Laboratories, Inc.
Atopic Dermatitis
Study to Evaluate the Efficacy, Safety and Pharmacokinetics of ASB17061 Capsules in Adult Subjects With Atopic Dermatitis
The purpose of this research study is to gather scientific information about the effectiveness of the study drug, ASB17061 capsules, when compared to placebo in adult subjects with atopic dermatitis.
- Trial name
- NCT01756898
- Testing
- 5 mg ASB17061, 10 mg ASB17061, 20 mg ASB17061
- Where
- 40 sites in United States
- Volunteers
- 370 people
- Sponsor
- Daiichi Sankyo
Fabry Disease
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
- Trial name
- MODIFY (NCT03425539)
- Testing
- Lucerastat
- Where
- 49 sites in United States, Australia and 12 more
- Volunteers
- 118 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Healthy
A Study in Healthy People to Test Whether Different Doses of BI 1015550 Have Potential to Induce Heart Rhythm Abnormalities
The main objective of this trial is to evaluate the effects of a single therapeutic and a single supra-therapeutic dose of BI 1015550 following oral administration on cardiac safety parameters in healthy male and female…
- Trial name
- NCT06107036
- Testing
- BI 1015550, Moxifloxacin
- Where
- United Kingdom
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Boehringer Ingelheim
Macular Oedema, Retinitis Pigmentosa
Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) Study
The purpose of this study is to assess the safety and efficacy of intravitreal injections of Aflibercept (Eylea) in treating Cystoid Macula Oedema (CMO) in patients with underlying Retinitis Pigmentosa (RP).
- Trial name
- AMOUR (NCT02661711)
- Testing
- Aflibercept
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Moorfields Eye Hospital NHS Foundation Trust
Osteoarthritis, Avascular Necrosis
Arcos Revision Stem: Evaluation of Clinical Performance
The primary purpose of this study is to evaluate the clinical performance of the Arcos Revision Stem system, determine the stability of the implants, and evaluate any relationship between Paprosky bone defect level and the…
- Conditions
- Osteoarthritis, Avascular Necrosis, Rheumatoid Arthritis, Deformity
- Trial name
- NCT01820611
- Testing
- Arcos Revision Stem System
- Where
- 5 sites in Finland, Spain and 2 more
- Volunteers
- 74 people
- Sponsor
- Zimmer Biomet
Endothelial Dysfunction
A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
This Phase 2, randomized, open-label, 2-treatment, 2-sequence, 2-period crossover, pharmacokinetic (PK) study will compare plasma concentrations of BH4 in subjects with endothelial dysfunction following 14 days of treatment by…
- Trial name
- NCT00532844
- Testing
- Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- BioMarin Pharmaceutical
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Cardiac Disease
Beta Blockers and Angiotensin Receptor Blockers in Bicuspid Aortic Valve Disease Aortopathy (BAV Study)
The purpose of this study is to determine whether long-term treatment with a beta-blocker (BB) such as atenolol and/or an angiotensin receptor blocker (ARB) such as telmisartan, given to adult patients with bicuspid aortic valve…
- Trial name
- BAV (NCT01202721)
- Testing
- Atenolol, Telmisartan
- Where
- 13 sites in Canada
- Volunteers
- 85 people
- Sponsor
- Hamilton Health Sciences Corporation
Patent Ductus Arteriosus
AMPLATZER Duct Occluder II Clinical Study
The objective of this study is to investigate the safety and effectiveness of the ADO II in patients with a PDA.
- Trial name
- ADO II (NCT00713700)
- Testing
- AMPLATZER Duct Occluder II
- Where
- 25 sites in United States
- Volunteers
- 192 people
- Sponsor
- Abbott Medical Devices
Polycystic Ovary Syndrome, c Type Natriuretic Peptide
Comparison of Serum C Type Natriuretic Peptide Levels Between Polycystic Ovary Syndrome Patients and Healthy Women
Recent studies have shown that C natriuretic peptide is produced from granulosa cells, increasing cumulative guanosine monophosphate (cGMP) production by affecting cumulus cells through natriuretic peptide receptors.
- Conditions
- Polycystic Ovary Syndrome, c Type Natriuretic Peptide, Menstrual Irregularity, Hyperandrogenism
- Trial name
- CNP (NCT04006171)
- Testing
- C type natriuretic peptide
- Where
- Cyprus
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Near East University, Turkey
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