Clinical trials: Neonatal
Epidermolysis Bullosa Dystrophica
Growth Hormone in EB
Growth is extremely affected in epidermolysis bullosa patients
- Trial name
- NCT05390073
- Testing
- growth hormone level and insulin like growth factor 1
- Volunteers
- 50 people
- Sponsor
- Cairo University
Cystic Fibrosis, Glucose Metabolism Disorders
One Year Glargine Treatment in CFRD Children and Adolescents
Glargine treatment can improve the clinical features in Cystic Fibrosis patients affected by glucose derangements
- Trial name
- NCT00483769
- Testing
- Glargine
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Federico II University
Congenital Heart Disease, Atherosclerosis
Cardiovascular Disease Screening
This study will evaluate volunteers 18 years of age and older to see if they qualify for one of NHLBI s research studies. Tests include the following: General medical evaluation, which may include blood tests, chest x-ray…
- Conditions
- Congenital Heart Disease, Atherosclerosis, Myocardial Ischemia, Myocardial Infarction
- Trial name
- NCT00483951
- Testing
- Toshiba Aquilion ONE CT, SWiemens MRI scanner
- Where
- 2 sites in United States
- Volunteers
- 1,325 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Genetic Disorder, Achondroplasia
Special Survey for Long Term Application
This study is conducted in Japan. The aim of this study is to assess the incidence rate of adverse drug reactions (ADRs) when using somatropin (Norditropin®) for treatment of for achondroplasia without epiphyseal line closure…
- Trial name
- NCT01516229
- Testing
- somatropin
- Where
- 2 sites in Japan
- Volunteers
- 395 people
- Sponsor
- Novo Nordisk A/S
Bloom Syndrome
Biological Significance of the Bloom's Syndrome Protein
Since 1960, persons with the very rare disorder Bloom's syndrome (BS) have been followed clinically, documenting clinical matters as obtained from their doctors.
- Trial name
- NCT00021437
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Familial Hypercholesterolemia
Soy Food Intervention Trial
Familial hypercholesterolemia (FH MIM#143890), an inherited disorder of lipoprotein metabolism, is a risk for early cardiovascular disease (CVD).
- Trial name
- SOYFIT (NCT00924339)
- Testing
- soy protein diet, rapeseed oil
- Where
- Austria
- Volunteers
- 30 people
- Sponsor
- Medical University of Vienna
Eczema Atopic Dermatitis, Eczema
A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)
This is a Phase 2, two-stage, adaptive, 1:1 randomized, double-blind, vehicle-controlled clinical study evaluating the efficacy, safety, and tolerability of GX-03 topical ointment in adult subjects with atopic dermatitis…
- Conditions
- Eczema Atopic Dermatitis, Eczema, Atopic Dermatitis
- Trial name
- GX-03 in AD (NCT07355075)
- Testing
- GX-03, Vehicle
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Turn Therapeutics
Cystic Fibrosis
Capsule Endoscopy in Cystic Fibrosis
Cystic Fibrosis patients continue to have bowel problems even after adequate pancreatic enzyme supplementation. There may be pathology of the lining of the bowel. Capsule endoscopy will be used to photograph the entire bowel.
- Trial name
- NCT00333957
- Where
- Israel
- Volunteers
- 200 people
- Sponsor
- Hadassah Medical Organization
Pulmonary Disease
Inhaled Nitric Oxide to Prevent and Treat Bronchopulmonary Dysplasia
Inhaled nitric oxide in preterm babies with respiratory failure or ventilator dependence will: 1. decrease the incidence of Bronchopulmonary Dysplasia (BPD) or death 2.
- Trial name
- NO-BPD (NCT01503801)
- Testing
- Nitric Oxide, respiratory support
- Where
- China
- Volunteers
- 400 people
- Sponsor
- Fudan University
Wilson Disease, D-Penicillamine
Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity
Wilson's disease is a genetic disorder, resulting from an anomaly present on the ATP7B gene located on chromosome 13, causing a progressive accumulation of copper in various organs such as the liver, nervous system and cornea…
- Conditions
- Wilson Disease, D-Penicillamine, Effect of D-penicilline on Cutaneous Elastity of Wilson's Patient
- Trial name
- WILDERME (NCT06945081)
- Testing
- Preparation of forearm molds with SILFLO® silicone
- Where
- 2 sites in France
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Cardiac Amyloidosis, Cardiac Disease
High Resolution Three-dimensional Maps of the Right Chambers in Patient Diagnosed With Cardiac Amyloidosis
Amyloid heart disease is an accumulation of fibrillar proteins in the extracellular sector of the heart. Identified on echocardiography as Ventricular hypertrophy.
- Trial name
- CARTO-AMYLO (NCT04956965)
- Testing
- Cardiac electro-mapping
- Where
- Martinique
- Volunteers
- 24 people
- Sponsor
- University Hospital Center of Martinique
Cystic Fibrosis, Digestive System Disease
A Comprehensive Approach To Relief Of Digestive Symptoms In Cystic Fibrosis: CARDS-CF
Development of a new patient reported outcome measure (PROM) that will measure the daily burden of gastrointestinal symptoms over the previous 24 hour period for people with cystic fibrosis.
- Conditions
- Cystic Fibrosis, Digestive System Disease, Genetic Disease, Respiratory Tract Diseases
- Trial name
- CARDS-CF (NCT05251467)
- Testing
- Focus group, Patient interview (n = approximately 10), Questionnaire (n = 180)
- Where
- United Kingdom
- Volunteers
- 350 people
- Sponsor
- Nottingham University Hospitals NHS Trust
Cystic Fibrosis (CF)
Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics.
- Trial name
- NCT07809867
- Testing
- Elexacaftor, Tezacaftor, Ivacaftor, Ivacaftor (VX-770)
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Istituto Giannina Gaslini
Stargardt, Stargardt's Disease
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75…
- Conditions
- Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
- Trial name
- POLARIS (NCT06435000)
- Where
- 20 sites in United States, Germany and 1 more
- Volunteers
- 75 people
- Sponsor
- Splice Bio
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged.
- Trial name
- NCT05981092
- Where
- 15 sites in United States, Netherlands and 3 more
- Volunteers
- 35 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma
Testing Obeticholic Acid for Familial Adenomatous Polyposis
This phase IIa trial investigates if giving obeticholic acid (OCA) is safe and has a beneficial effect on the number of polyps in the small bowel and colon in patients with familial adenomatous polyposis (FAP).
- Conditions
- Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma, Familial Adenomatous Polyposis
- Trial name
- NCT05223036
- Testing
- Biopsy Procedure, Biospecimen Collection, Gastrointestinal Endoscopy
- Where
- 7 sites in United States, Puerto Rico
- Volunteers
- 15 people
- Sponsor
- National Cancer Institute (NCI)
Stroke, Patent Foramen Ovale
International Multicenter Patent foRamen OVale & strokE (IMPROVE) Registry
Patent foramen ovale (PFO) is a prevalent cause of ischemic stroke and transient ischemic attack (TIA) in young patients. Its role as a cause of cerebrovascular events in the older population is more controversial because of…
- Conditions
- Stroke, Patent Foramen Ovale, Atrial Fibrillation, Atrial Flutter
- Trial name
- IMPROVE (NCT04738071)
- Testing
- PFO closure
- Where
- Canada
- Volunteers
- 3,730 people
- Sponsor
- Luciano A. Sposato
Pregnancy Complications, Maternal Death
Nutrition Optimalization Among Pregnant Women to Improve Maternal and Neonatal Outcome in DKI Jakarta
The goal of this MONAS Study is to learn about comprehensive monitoring and nutritional intervention among pregnant women in order to improve maternal and neonatal outcomes. The main questions it aims to answer are: 1.
- Conditions
- Pregnancy Complications, Maternal Death, Preeclampsia, Intrauterine Infection
- Trial name
- MONAS (NCT06268509)
- Testing
- Nutritional panel laboratory examination, Pregnancy supplements (other than provided by Ministry of Health), Fetomaternal ultrasound
- Where
- Indonesia
- Volunteers
- 245 people
- Sponsor
- Dr Cipto Mangunkusumo General Hospital
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics