Clinical trials: Newborn Diseases
Micrognathia of Lower Jaw, Retrognathism
Comparison Between Internal and External Distractors in Osteogenesis
Distraction osteogenesis is a powerful technique for creating new bone during significant lengthening of the mandible without the need for bone grafting and associated donor site morbidity.
- Conditions
- Micrognathia of Lower Jaw, Retrognathism, Facial Asymmetry
- Trial name
- NCT03540329
- Testing
- Internal Osteogenesis distractor, External Osteogenesis distractor
- Volunteers
- 30 people
- Sponsor
- Assiut University
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC…
- Trial name
- NCT04214002
- Where
- United States
- Sponsor
- Krystal Biotech, Inc.
Muscular Dystrophy, Muscular Disease
Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy
Background: Duchenne muscular dystrophy (DMD) is a disease in which the muscles are unable to make the protein dystrophin. Without this protein, the muscles become gradually weaker.
- Trial name
- NCT01451281
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Familial Hypercholesterolemia
Familial Hypercholesterolemia Interpretive Comment - Nudging to Detection.
Familial hypercholesterolemia is the most common inherited disease of the lipid metabolism, however it remains underdiagnosed. Only 15 % of 30.000 possible patients have been found in Denmark.
- Trial name
- FIND (NCT05614219)
- Testing
- Biochemistry interpretive comment on elevated LDL-C levels
- Where
- 2 sites in Denmark
- Volunteers
- 2,000 people
- Sponsor
- Odense University Hospital
Cardiomyopathy, Hypertrophic, Familial
Signs and Symptoms of Genetic Abnormalities Linked to Inherited Heart Disease
Genetically inherited heart diseases (familial cardiomyopathies) are conditions affecting the heart passed on to family members through abnormalities in genetic information.
- Trial name
- NCT00001881
- Where
- United States
- Volunteers
- 9,999 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Inflammatory Bowel Diseases, Primary Immune Deficiency Disorder
Immunological Characteristics of Patients With PID and IBD
The investigators wish to characterize alterations in the architecture and function of immune cells in patients with a primary immunodeficiency that also develop inflammatory bowel disease.
- Trial name
- NCT03148028
- Where
- 2 sites in Israel
- Volunteers
- 45 people
- Sponsor
- Sheba Medical Center
Relapsing Remitting Multiple Sclerosis
A Study of Orally Administered BGC20-0134 (Structured Lipid) in Patients With Relapsing Remitting Multiple Sclerosis (RRMS)
To determine the efficacy and safety of an oral drug (BGC20-0134) in patients with relapsing remitting multiple sclerosis. Specifically, the cumulative number of new gadolinium enhancing lesions after 24 weeks of treatment with…
- Trial name
- NCT01037907
- Testing
- Pleneva TM BGC20-0134
- Where
- 35 sites in Belgium, France and 4 more
- Volunteers
- 173 people
- Sponsor
- Boston Scientific Corporation
Gitelman Syndrome
A Translational Approach to Gitelman Syndrome
This study will test the response to a single dose of thiazide diuretic as a diagnostic test for Gitelman syndrome. Individuals with hypokalemic alkalosis will be enrolled.
- Trial name
- NCT00822107
- Testing
- Hydrochlorothiazide
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Oregon Health and Science University
Dermatitis, Atopic
A Study Assessing GW870086's Potential to Cause Skin Thinning
This study is a randomised, double-blind, placebo-controlled study of topical GW870086 to explore the potential for skin thinning in healthy adult volunteers after 42±2 days of treatment.
- Trial name
- NCT01381445
- Testing
- GW870086 0.2%, GW870086 2%, Clobetasol Propionate
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Gout
Safety and Efficacy Study of PEG-uricase in the Treatment of Hyperuricemic Patients With Symptomatic Gout
These are two replicate studies to evaluate the safety and efficacy of PEG (polyethylene glycol)-uricase in controlling the uric acid level in symptomatic gout patients with high uric acid levels who are unable to take standard…
- Trial name
- NCT00325195
- Testing
- pegloticase
- Where
- 56 sites in United States, Canada and 1 more
- Volunteers
- 225 people
- Sponsor
- Savient Pharmaceuticals
Rett Syndrome, MECP2 Duplication
Biobanking of Rett Syndrome and Related Disorders
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, and individuals with MECP2…
- Conditions
- Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
- Trial name
- NCT02705677
- Where
- 12 sites in United States
- Volunteers
- 752 people
- Sponsor
- University of Alabama at Birmingham
Dermatitis, Atopic
WAL 801 CL Dry Syrup in Paediatric Atopic Dermatitis Patients
Study to investigate the safety and efficacy of WAL 801 CL Dry Syrup on pruritus associated with paediatric atopic dermatitis in comparison with that of Ketotifen Fumarate Dry Syrup and to confirm the appropriateness of dosage of…
- Trial name
- NCT02182557
- Testing
- WAL 801 CL Dry Syrup, Ketotifen Fumarate Dry Syrup
- Volunteers
- 162 people
- Sponsor
- Boehringer Ingelheim
Non-Dystrophic Myotonia
Treatment of Myotonia - Lamotrigine Versus Namuscla
In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine.
- Trial name
- NCT05639257
- Testing
- Lamotrigine, Namuscla
- Where
- Denmark
- Volunteers
- 32 people
- Sponsor
- Grete Andersen, MD
Infant, Premature, Diseases
Efficacy of an Oral Stimulation Protocol in Preterm Infants to Shorten the Period Between Enteral and Oral Nutrition
Oral feeding in the neonatal period requires an accurate coordination between suction, swallowing and breathing. In premature new-borns and, depending on their gestational age, the coordinated work of those processes becomes…
- Trial name
- NCT03016767
- Testing
- Manual oral stimulation protocol
- Where
- Spain
- Volunteers
- 44 people
- Sponsor
- Fundacion Para La Investigacion Hospital La Fe
Atopic Dermatitis, Atopic Hand Eczema
A 32-week Trial to Evaluate the Efficacy and Safety of Tralokinumab in Subjects With Moderate-to-severe Atopic Hand Eczema Who Are Candidates for Systemic Therapy
The purpose of this study is to test if treatment with tralokinumab is safe and efficacious in treating moderate-to-severe atopic hand eczema.
- Trial name
- ADHAND (NCT05958407)
- Testing
- Tralokinumab
- Where
- 69 sites in United States, Belgium and 8 more
- Volunteers
- 235 people
- Sponsor
- LEO Pharma
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENERGYC Study
This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of…
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, MELAS, Subacute Necrotizing Encephalomyelopathy
- Trial name
- NCT04846036
- Testing
- Sonlicromanol
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Khondrion BV
PCOS, Offspring, Adult
Offspring Born to Mothers With Polycystic Ovary Syndrome in Guangzhou Cohort Study
The Offspring Born to Mothers with Polycystic Ovary Syndrome in Guangzhou Cohort study (PCOS-BIG) was established to investigate the short- and long-term effects of intrauterine exposure to maternal PCOS on the health of…
- Conditions
- PCOS, Offspring, Adult, Hyperandrogenism, Epigenetics
- Trial name
- PCOS-BIG (NCT03742011)
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Guangzhou Women and Children's Medical Center
Anemia
Impact of Maternal Iron Status on Neonatal Iron Status and Auditory Brainstem Response in the Newborn
This study aims to determine how maternal Fe status influences placental and neonatal Fe status in pregnant adolescents and to assess the impact of the Fe endowment of birth on functional outcomes as assessed by auditory…
- Trial name
- NCT01019902
- Where
- United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Cornell University
Corneal Edema, Fuchs
Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction
This study will evaluate the safety and effectiveness of a single injection of EO2002 at one of two dose levels (150,000 or 500,000 magnetic human corneal endothelial cells [mHCECs]), compared with a placebo injection, in…
- Conditions
- Corneal Edema, Fuchs, Fuchs Dystrophy, Fuchs' Endothelial Dystrophy
- Trial name
- EMERALD (NCT07729137)
- Testing
- 150K cells, 500K cells
- Where
- 10 sites in United States
- Volunteers
- 121 people
- Sponsor
- Emmecell
Respiratory Distress Syndrome, Hyaline Membrane Disease
Weaning Preterm Neonates From Nasal Continuous Positive Airway Pressure
There is little data published concerning the best approach to nasal continuous positive airway pressure (nCPAP) weaning. Potential complications associated with prolonged nCPAP therapy include gastric distension, nasal…
- Conditions
- Respiratory Distress Syndrome, Hyaline Membrane Disease, Preterm Infants, Premature Infants
- Trial name
- NCT01093495
- Testing
- CPAP, Nasal Cannula
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Mansoura University
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