Clinical trials: Newborn Diseases
Cholestasis
Ursodiol for Treating Parenteral Nutrition Associated Cholestasis in Neonates
The purpose of this study is to determine whether ursodiol is effective in the treatment of parenteral nutrition associated cholestasis in neonates.
- Trial name
- URSONEONAT (NCT00846963)
- Testing
- Ursodiol
- Where
- Canada
- Volunteers
- 26 people
- Sponsor
- Ibrahim Mohamed
Phenylketonuria (PKU)
A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability
A home-use self-test blood phenylalanine monitoring system under the brand name Egoo Phe System from manufacturer Egoo Health Aps has been developed for measurement of phenylalanine (Phe) in individuals diagnosed with…
- Trial name
- NCT06940193
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Egoo Health Aps
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
Huntington's Disease
Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD)
The purpose of this study is to evaluate the safety, tolerability and clinical impact of 15-grams daily of sodium phenylbutyrate (phenylbutyrate) in Huntington's disease and to lay the groundwork for possible subsequent trials…
- Trial name
- NCT00212316
- Testing
- sodium phenylbutyrate
- Where
- 8 sites in United States
- Volunteers
- 60 people
- Sponsor
- University of Rochester
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human…
- Trial name
- NCT04049084
- Testing
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 70 people
- Sponsor
- University of California, Los Angeles
Adenomatous Polyposis Coli, Familial Adenomatous Polyposis
Cold Atmospheric Plasma for the Endoscopic Treatment of Duodenal Polyps in Patients With Familial Adenomatous Polyposis
The objective of this study is to investigate the feasibility for the treatment of precancerous peri-ampullary FAP polyps in the duodenum using low-thermal argonplasma.
- Conditions
- Adenomatous Polyposis Coli, Familial Adenomatous Polyposis, Duodenal Adenoma
- Trial name
- coldAPC (NCT06435533)
- Testing
- low energy argonplasma coagulation
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf
Down Syndrome
Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome
This trial protocol is designed to evaluate primarily whether the use of sargramostim (recombinant human GM-CSF), administered five days per week for four consecutive weeks (20 treatment days), will be well tolerated by and safe…
- Trial name
- NCT05482334
- Testing
- Sargramostim for Injection
- Where
- United States
- Sponsor
- University of Colorado, Denver
Coronary Disease, Hypolipoproteinemia
Exercise Versus Niacin in Patients With Coronary Artery Disease (CAD) and Low High-Density Lipoproteins (HDL)
The investigators want to study the relative effects of physical exercise vs. extended-release niacin (lipid-lowering drug) in patients with coronary heart disease and low HDL cholesterol ("good cholesterol") on lipid profile…
- Trial name
- NCT00298909
- Testing
- physical exercise, niaspan (extended-release niacin), niacin
- Where
- Germany
- Volunteers
- 48 people
- Sponsor
- University of Leipzig
Prader-Willi Syndrome
GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity.
- Trial name
- NCT03616509
- Testing
- Growth hormone
- Where
- Spain
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Corporacion Parc Tauli
MPS III B
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144…
- Trial name
- NCT05492799
- Testing
- AX 250
- Where
- 6 sites in United States, Colombia and 3 more
- Volunteers
- 15 people
- Sponsor
- Allievex Corporation
Cystic Fibrosis
Muscle Accrual and Function in Cystic Fibrosis-Impact of Glucose Intolerance
This study will investigate the link between glucose abnormalities and elements critical to muscle function including mass, composition and energy metabolism.
- Trial name
- NCT02776098
- Where
- 2 sites in United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Familial Mediterranean Fever
Pharmacokinetics Study of Colchicine in Familial Mediterranean Fever (FMF) Patients
Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or…
- Trial name
- NCT01075906
- Testing
- colchicine sprinkle capsules
- Where
- 10 sites in United States, Armenia and 2 more
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Mutual Pharmaceutical Company, Inc.
Innate Immune Response
A Trial Investigating the Influence of BCG and Hepatitis B Immunisation at Birth on Neonatal Immune Responses: The Early Life Vaccines and Immunity Study
Neonatal morbidity and mortality from infectious diseases is of global concern. Childhood disease-specific immunisation is irrefutably linked to the decline in deaths from these targeted infections over the last century.
- Trial name
- ELVIS (NCT02444611)
- Testing
- BCG Vaccine, Hepatitis B Vaccine
- Where
- Australia
- Volunteers
- 185 people
- Sponsor
- Murdoch Childrens Research Institute
Gaucher's Disease
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages.
- Trial name
- NCT00001234
- Testing
- human glucocerebrosidase cDNA
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Fetal Growth Restriction, Antiphospholipid Syndrome
Antiphospholipid Antibodies and Fetal Growth Restriction
The Antiphospholipid Syndrome is an immune disease where the presence of antibodies directed against cell membrane phospholipids (antiphospholipid antibodies) can cause an hypercoagulable state that causes thrombosis and…
- Trial name
- NCT01538134
- Where
- Panama
- Volunteers
- 90 people
- Sponsor
- Saint Thomas Hospital, Panama
Duchenne Muscular Dystrophy
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT01037309
- Testing
- PRO044 SC, PRO044 IV
- Where
- 4 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Cystic Fibrosis, Prediabetes
Prevention of Cystic Fibrosis Diabetes
Acute systemic hyperglycemia causes oxidative stress and a pro-inflammatory response. The pro-inflammatory cytokines induced by hyperglycemia are toxic to islet insulin producing cells, and thus worsen glucose intolerance.
- Trial name
- Prevent (NCT00967798)
- Testing
- Sitagliptin
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- Emory University
Gaucher Disease
Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
The main aim of this study is to measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect…
- Trial name
- NCT04429984
- Where
- 2 sites in India
- Volunteers
- 21 people
- Sponsor
- Shire
Nevus, Dysplasia; Epithelial
Skin Fluorescent Imaging (SFI) System in Patients With Nevi
The Sponsor has developed the Skin Fluorescent Imaging (SFI) system, an in vivo imaging modality, for the purpose of providing physicians with insight into the biological changes occurring during tissue remodeling in a nevus.
- Trial name
- NCT04368247
- Testing
- Skin Fluorescent Imaging
- Where
- 4 sites in United States
- Volunteers
- 300 people
- Sponsor
- Orlucent, Inc
Phenylketonuria
Sapropterin Expanded Access Program
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded…
- Trial name
- NCT00484991
- Testing
- Sapropterin dihydrochloride
- Where
- 36 sites in United States
- Sponsor
- BioMarin Pharmaceutical
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