Clinical trials: Congenital Anomaly
Heart Defects, Congenital
Safety and Performance of a Vascular Patch in Pediatric Patients Undergoing Bidirectional Cava-pulmonary Anastomosis
Xeltis developed biodegradable patch prosthesis, the Vascular Patch Model COR-VP-001, to be used as a vascular patch to augment the pulmonary trunk and pulmonary arteries.
- Trial name
- XEL-CR-06 (NCT02377700)
- Testing
- Xeltis Vascular Patch, Model COR-VP-001
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Xeltis
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase.
- Trial name
- NCT02279498
- Testing
- Liprotamase, porcine (pig) PERT
- Where
- 54 sites in United States, Canada and 5 more
- Volunteers
- 128 people
- Sponsor
- Anthera Pharmaceuticals
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Atopic Dermatitis
Study of Dupilumab in Adult Patients With Extrinsic Moderate-to-Severe Atopic Dermatitis
The primary objective was to assess the clinical efficacy of repeated subcutaneous (SC) doses of Dupilumab in adult participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01548404
- Testing
- Dupilumab
- Where
- 21 sites in Czechia, France and 3 more
- Volunteers
- 109 people
- Sponsor
- Regeneron Pharmaceuticals
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to…
- Trial name
- NCT05659901
- Where
- 10 sites in United States, France and 5 more
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn More About How Much of the Study Treatment Elinzanetant (or BAY3427080) Gets Absorbed, How Safe it is and How it Affects the Body in Healthy Female and Male Participants
Researchers are looking for a better way to treat men and women with vasomotor symptoms, a condition of having hot flashes caused by hormonal changes.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05351892
- Testing
- Elinzanetant (BAY3427080)
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Bayer
Hypercholesterolemia, Familial Hypercholesterolemia
A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00704444
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 11,332 people
- Sponsor
- Organon and Co
Autism Spectrum Disorder, Fra(X) Syndrome
Establishing a Diagnostic and Therapeutic Index in Autism Spectrum Disorder (ASD) and Fragile X Syndrome
The purpose of this study is to accumulate and quantitatively analyze data on the microbiome, serotonin signaling and genetics, and inflammatory cytokines from patients with Autism Spectrum Disorder and Fragile X Syndrome.
- Trial name
- NCT04869930
- Testing
- specimen collection
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Nova Mentis Life Science Corp
Healthy
A Study to Assess the Effects of 2 Different Prothrombin Complex Concentrates on the Pharmacodynamics of Rivaroxaban in Healthy Adult Volunteers
The purpose of this study is to assess the use of 2 different Prothrombin Complex Concentrates (PCCs) on their ability to reverse (normalize) the pharmacodynamic effects of rivaroxaban in healthy adult volunteers.
- Trial name
- NCT01656330
- Testing
- Rivaroxaban 20 mg twice daily, Rivaroxaban 20 mg once daily, Profilnine SD
- Where
- Belgium
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Janssen Research & Development, LLC
Duchenne Muscular Dystrophy
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531…
- Trial name
- NCT07209332
- Testing
- WVE-N531
- Where
- 3 sites in Jordan, United Kingdom
- Volunteers
- 175 people
- Sponsor
- Wave Life Sciences USA, Inc.
Hyperlipoproteinemia Type I
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood.
- Trial name
- NCT01447901
- Where
- Canada
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Erythropoietic Protoporphyria (EPP)
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
- Trial name
- NCT03520036
- Testing
- MT-7117 low dose, MT-7117 high dose
- Where
- 9 sites in United States
- Volunteers
- 102 people
- Sponsor
- Tanabe Pharma America, Inc.
Fabry Disease
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease
- Trial name
- NCT00526071
- Testing
- migalastat HCl
- Where
- 8 sites in United States, Australia and 3 more
- Volunteers
- 23 people
- Sponsor
- Amicus Therapeutics
Acute Bacterial Infections, Acute Viral Infections
300 Antibody Diagnostic Test Kit
For the development of a Point of Care IVD test kit for acute phase disease detection against a variety of bacterial and viral infections.
- Trial name
- Antibody 300 (NCT01646411)
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- The Plasma Source
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074971
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 20 sites in United States, France and 3 more
- Volunteers
- 58 people
- Sponsor
- Genzyme, a Sanofi Company
Duchenne Muscular Dystrophy
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies.
- Trial name
- NCT05689164
- Testing
- fordadistrogene movaparvovec
- Where
- 12 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pfizer
Type II Hyperlipidemia
A Safety and Efficacy Study of DRL-17822, a Cholesteryl Ester Transfer Protein (CETP) Inhibitor, in Patients With Abnormal Cholesterol Levels
The purpose of this study is to determine if a new drug, DRL-17822, is safe and effective in elevating high density lipoprotein cholesterol (HDL-C) and reducing low density lipoprotein cholesterol (LDL-C) in people with abnormal…
- Trial name
- NCT01388816
- Where
- 12 sites in Italy, Poland and 1 more
- Volunteers
- 176 people
- Sponsor
- Dr. Reddy's Laboratories Limited
Breast Implant Revision, Congenital Breast Defect Correction
Scaffold-guided Breast Surgery
The clinical trial is testing the use of a novel method to grow new tissue within the breast injecting fat tissue harvested from patient's own fat deposits.
- Trial name
- NCT05437757
- Testing
- Surgical implantation of the PCL Breast scaffold with autologous fat grafting
- Where
- Australia
- Volunteers
- 19 people
- Sponsor
- BellaSeno Pty Ltd
Atopic Dermatitis
A Study of Upadacitinib in Adult Participants With Moderate-to-Severe Atopic Dermatitis and Inadequate Response to Dupilumab
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic…
- Trial name
- Switch-Up (NCT06389136)
- Testing
- Upadacitinib 15mg Dose, Dupilumab 300mg Dose, Upadacitinib 30mg Dose
- Where
- 129 sites in United States, Canada and 7 more
- Volunteers
- 184 people
- Sponsor
- AbbVie
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