Clinical trials: Newborn Diseases
Congenital Cataract, Tooth Abnormalities
Associations Between Dental Anomalies and Ocular, Cutaneous and Skin Appendages Features
Considering recent literature, it is possible to hypothesise a link between dental anomalies and ocular and/or cutaneous findings, given the existence of shared genetic and developmental mechanisms between these two anatomical…
- Conditions
- Congenital Cataract, Tooth Abnormalities, Ocular Pathologies
- Trial name
- NCT06950619
- Testing
- Whole Genome Sequencing, Cephalometric tracing
- Where
- 2 sites in Italy
- Volunteers
- 5 people
- Sponsor
- University of Pavia
Congenital Diaphragmatic Hernia, Persistent Pulmonary Hypertension of the Newborn
Milrinone in Congenital Diaphragmatic Hernia
Infants with congenital diaphragmatic hernia (CDH) usually have pulmonary hypoplasia and persistent pulmonary hypertension of the newborn (PPHN) leading to hypoxemic respiratory failure (HRF).
- Conditions
- Congenital Diaphragmatic Hernia, Persistent Pulmonary Hypertension of the Newborn, Hypoxemic Respiratory Failure, Pulmonary Hypoplasia
- Trial name
- NCT02951130
- Testing
- Milrinone
- Where
- 19 sites in United States
- Volunteers
- 66 people
- Sponsor
- NICHD Neonatal Research Network
Severe Combined Immunodeficiency, Neonatal Screening
Neonatal Screening of Severe Combined Immunodeficiencies
The goal of the proposed research is to observe the prevalence and establish the validity of a newborn screening method for severe combined immunodeficiency (SCID).
- Trial name
- NCT02590328
- Where
- China
- Volunteers
- 180,000 people
- Sponsor
- Children's Hospital of Fudan University
Healthy Subjects, Cystic Fibrosis
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple…
- Trial name
- NCT03647228
- Testing
- IONIS-ENaCRx
- Where
- 8 sites in Germany, United Kingdom
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Atopic Dermatitis
CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry
Prospective observational registry for an adolescent cohort with AD under the care of a dermatology provider. Approximately 1500 subjects and 75 clinical sites in North America and select European countries will be recruited to…
- Trial name
- NCT06238765
- Where
- United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- CorEvitas
Huntington's Disease
Exercise Effects in Huntington's Disease
Huntington's disease (HD) is an incurable and fatal disorder characterised by progressive degeneration of the basal ganglia and the cerebral cortex.
- Trial name
- NCT01879267
- Testing
- Exercise training
- Where
- Switzerland
- Volunteers
- 40 people
- Sponsor
- University of Zurich
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer
Stable Angina Pectoris, Periodontitis
Effect of Periodontal Therapy on Asprosin in Stable Angina
This study aims to evaluate the effect of non-surgical periodontal therapy on serum asprosin levels in patients with stable angina and periodontitis.
- Trial name
- NCT07591844
- Testing
- Non-surgical periodontal therapy (scaling and root planing), Non-surgical periodontal therapy (Professional dental cleaning)
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Cleft Palate, Orofacial Cleft
Neural Network to Calculate Morphology of the Cleft Palate to Reduce Cleft Lip and Palate Treatment Burden.
This study is to develop a neural network to compute palatal three dimensional (3D) geometry by using routinely taken intraoral/palatal photographs and palatal casts of infants with cleft lip and palate deformity for reducing…
- Trial name
- NCT04342234
- Testing
- data collection of palatal casts and palatal images of cleft patients routinely treated at the institution
- Where
- Switzerland
- Volunteers
- 450 people
- Sponsor
- University Hospital, Basel, Switzerland
Cardiomyopathy, Dilated
Registry of Cell Therapy in Non-Ischemic Dilated Cardiomyopathy
Although several studies have demonstrated beneficial effects of stem cell therapy in patients with non-ischemic dilated cardiomyopathy, the long term benefits and predictors of response to therapy remain undefined.
- Trial name
- RECORD (NCT02445534)
- Testing
- Autologous stem cell therapy
- Where
- Slovenia
- Volunteers
- 250 people
- Sponsor
- University Medical Centre Ljubljana
Sickle Cell Disease
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor
Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels.
- Trial name
- NCT06023199
- Testing
- Voxelotor
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Inova Health Care Services
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
Hemodialysis Patient, Hemodialysis Catheter
The Effect of Arteriovenous Fistula Care Training Given to Hemodialysis Patients on Quality of Life, Comfort and Self-Care Behaviours
This study will be conducted to evaluate the effect of Arterio-Venous Fistula (AVF) care training given to hamodialysis patients on quality of life, comfort and self-care behaviours.
- Trial name
- Fistula care (NCT06959914)
- Testing
- Training group
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Kafkas University
Cleft Lip and Palate
Otologic and Rhinologic Outcomes in Children With Clef Palate
OBJECTIVE: To compare otologic and rhinologic outcomes in patients with cleft palate according to surgical protocols and type of cleft. DESIGN: Monocentric retrospective and prospective analysis of medical reports.
- Trial name
- Clef Palate (NCT03708406)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Montpellier
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Nephropathy, Sickle Cell Anemia
Losartan to Reverse Sickle Nephropathy
Sickle cell disease causes kidney damage with increasing age, leading to chronic kidney disease and renal failure in nearly one third of patients with sickle cell disease.
- Trial name
- NCT01479439
- Testing
- Losartan
- Where
- 9 sites in United States
- Volunteers
- 36 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Infant, Premature, Diseases, Mothers
Home Support for Mothers Whose Premature Infants
Premature births are a challenging process for parents in many ways. Especially after the treatment and care in the hospital, they are concerned about whether they will be able to provide adequate care for their baby at home.
- Trial name
- NCT04741672
- Testing
- Providing guidance after discharge routine procedure and calling one week after discharge., Discharge routine procedure.
- Where
- Turkey (Türkiye)
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol
Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction
The study is to assess the long-term safety, tolerability, and efficacy after 48 and 72 weeks with monthly (Q4W [<31 days]) dosing of subcutaneous (SC) LIB003 300 mg administered in patients with CVD or at high risk for CVD…
- Conditions
- Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
- Trial name
- LIBerate-OLE (NCT04798430)
- Testing
- lerodalcibep
- Where
- 12 sites in United States, India and 4 more
- Volunteers
- 2,000 people
- Sponsor
- LIB Therapeutics LLC
Cystic Fibrosis
Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis
OBJECTIVES: I. Evaluate the safety of ascending doses of CPX administered to adult patients with mild cystic fibrosis. II. Evaluate the pharmacokinetics of ascending doses of CPX in this patient population.
- Trial name
- NCT00004428
- Testing
- CPX
- Volunteers
- 35 people
- Sponsor
- FDA Office of Orphan Products Development
Overweight and Obesity, Men
Evaluation of a Supplement for Weight Management in Obese and Overweight Individuals
The purpose of this trial is to examine the effects of a novel, orally consumed dietary supplement (composed of naturally occurring components) on weight management and metabolic rate in individuals who are overweight or obese…
- Conditions
- Overweight and Obesity, Men, Women
- Trial name
- NCT03812211
- Testing
- Active Supplement
- Where
- Canada
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- McMaster University
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