Clinical trials: Congenital Diseases
Enuresis, Nocturnal, Spina Bifida
The Relationship Between Nocturnal Enuresis And Spina Bifida Occulta
It is claimed that SBO may be responsible for bladder dysfunction in patients without known neurological disease. Subsequently, it was reported that the frequency of SBO in NE cases was higher than normal children.
- Trial name
- NCT03543995
- Volunteers
- 330 people
- Sponsor
- Ankara Training and Research Hospital
Sickle Cell Disease (SCD)
Self-administered Acupressure in Patients With Sickle Cell Disease
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
- Trial name
- NCT07875777
- Testing
- Verum Acupressure (using AcuWand)
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- University of Cincinnati
Intraventricular Hemorrhage, Spina Bifida
Micro Ribonucleic Acid (miRNA) Markers of Hydrocephalus in Intraventricular Hemorrhage (IVH)
A collection of biological samples (cerebrospinal fluid [CSF] and blood) from patients under 6 years of age who are diagnosed with intraventricular hemorrhage or spina bifida.
- Trial name
- NCT02386228
- Testing
- collection of CSF and blood
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Joseph's Hospital and Medical Center, Phoenix
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Safety and Tolerability Study of Denufosol in 2-4 Year Olds
The purpose of this study is to evaluate the safety and tolerability of denufosol 60 mg TID in pediatric CF patients 2 to 4 years of age
- Trial name
- REACH-1 (NCT01181622)
- Testing
- denufosol tetrasodium Inhalation Solution, 0.9% w/v sodium chloride solution
- Volunteers
- 25 people
- Sponsor
- Merck Sharp & Dohme LLC
Atopic Dermatitis
A Multi-center, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study of the Safety, Tolerability, and Pharmacokinetics of Subcutaneously Delivered ASLAN004 in Adults With Moderate-Severe Atopic Dermatitis
A Phase 1B, multi-center, double-blind, placebo-controlled, randomized, multiple ascending dose (MAD) clinical study is designed to evaluate ASLAN004 versus placebo in patients who have moderate-severe AD.
- Trial name
- NCT04090229
- Testing
- ASLAN004
- Where
- 11 sites in United States, Australia and 1 more
- Volunteers
- 52 people
- Sponsor
- ASLAN Pharmaceuticals
Genetic Disease, Genomics
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)
Background: People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away.
- Trial name
- NCT02156102
- Where
- 3 sites in United States, Sierra Leone
- Volunteers
- 405 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Amyloidosis Cardiac
Frontline Lenalidomide for AL Amyloidosis Involving Myocardium
This phase II clinical trial aimed at influencing the improvement of major organ functions, especially the objective response rate, in Amyloid light-chain amyloidosis involving myocardium.
- Trial name
- NCT04298372
- Testing
- Lenalidomide 25mg
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Seoul National University Hospital
Ventral Hernia, Ventral Incisional Hernia
Open Preperitoneal Mesh Versus Retromuscular Mesh Versus Suture Repair for Abdominal Wall Hernias
Randomized clinical trial comparing open preperitoneal mesh, retromuscular mesh and suture repair for ventral hernias less than 3 cm diameter
- Conditions
- Ventral Hernia, Ventral Incisional Hernia, Umbilical Hernia
- Trial name
- NCT03255239
- Testing
- Ventral hernia repair
- Where
- Tunisia
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hôpital Universitaire Taher Sfar
Bronchiectasis Adult
Inhaled A1AT in Adult Stable Bronchiectasis
Aim: to investigate the influence of alpha1-antitrypsin (A1-AT) nebulization on levels of A1-AT in BAL and plasma in patients with stable bronchiectasis. Method: single-blind placebo-controlled randomised clinical trial.
- Trial name
- NCT03383939
- Testing
- Group A, Group B
- Volunteers
- 20 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Speckle Tracking, Fetus Fetus
Speckle Tracking Echocardiography in Infants, Prenatally and Postnatally
Comparison of prenatal and postnatal cardiac function assessed by echocardiography using pulsed wave Doppler, Tissue Doppler and speckle tracking (strain and strain rate) between foetuses/neonates with a structural heart disease…
- Conditions
- Speckle Tracking, Fetus Fetus, Neonate, Structural Heart Disease
- Trial name
- STIPP (NCT05601375)
- Testing
- Echocardiography
- Where
- Netherlands
- Volunteers
- 201 people, healthy volunteers welcome
- Sponsor
- Erasmus Medical Center
Cardiomyopathy
Observational Epidemiological Study of Patients Suffering From Systemic Amyloidosis
observational epidemiological study of patients affected by systemic amyloidosis
- Trial name
- AMI-ER (NCT06866912)
- Where
- Italy
- Volunteers
- 750 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Loeys-Dietz Syndrome
Immunopathology of Loeys-Dietz Syndrome
Loeys-Dietz syndrome (LDS) is a rare vascular genetic disorder (estimated prevalence 1/25,000-1/100,000) due primarily to mutations in the Transforming growth factor beta (TGF-β) cytokine receptor 1 and 2 genes.
- Trial name
- I-LoDiS (NCT05472519)
- Testing
- blood samples
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Congenital Heart Disease
Strong Hearts: A Remote, App-Enabled, Exercise Program for Patients With Congenital Heart Disease (Strong Hearts App)
The overall goal of this program is to create a remote, mobile application enabled exercise program for patients with Congenital Heart Disease (CHD).
- Trial name
- NCT05853705
- Testing
- Strong Hearts Exercise Program
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Duke University
Congenital Heart Disease (CHD), Artifical Intelligence
AI-Assisted Versus Independent Heart Sound Screening for Congenital Heart Disease in Children
This is a prospective, school-based, cluster-randomized controlled trial conducted in Qinghai Province, China. The study will evaluate whether artificial intelligence (AI) can help primary care physicians identify congenital…
- Conditions
- Congenital Heart Disease (CHD), Artifical Intelligence, Screening, Cardiac Auscultation
- Trial name
- NCT07830251
- Testing
- AI-Assisted Cardiac Auscultation
- Where
- China
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Kun Sun
Friedreich Ataxia
Measurement of Frataxin mRNA in Biofluids
The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA).
- Trial name
- NCT06496451
- Testing
- Spinal tap for CSF collection, Venipuncture for blood sample
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Florida
Dental Implant Failed, Teeth Absent
Gelatin Sponge VS PTFE Membrane for Socket Sealing After Immediate Implant Placement
Digital panoramas will be made to assess the implant sites. The included patients are assigned to their respective groups randomly.
- Conditions
- Dental Implant Failed, Teeth Absent, Soft Tissue Injuries
- Trial name
- NCT05982353
- Testing
- PTFE application, Gelatin sponge
- Where
- Egypt
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cairo University
Ehlers-Danlos Syndrome, Obstructive Sleep Apnea
Obstructive Sleep Apnoea in Ehlers-Danlos Syndrome
Ehlers-Danlos Syndrome (EDS) is a clinically and genetically heterogeneous group of inherited connective tissue disorders characterized by joint hypermobility, skin hyperextensibility, and tissue fragility.
- Trial name
- OSA in EDS (NCT02435745)
- Where
- Switzerland
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Sleep Disturbance in Infancy (Disorder), Neonatal Disease
Real-time State of Vigilance Monitor for the Neonatal Intensive Care Unit
The goal of this observational study is to collect data to develop a complete package (hardware, user interface software and algorithms) that can monitor sleep-wake stages in neonates.
- Trial name
- NCT04920175
- Testing
- Novel Real-time Neonatal Sleep Stage Detection Algorithm
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- University of Michigan
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