Clinical trials: Congenital Diseases
Cystic Fibrosis
A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)
Inflammation is present in the Cystic fibrosis (CF) airway from the time of infancy, and worsens with the onset of chronic infection. Therapies with proven benefit are associated with decreased airway inflammation.
- Trial name
- NCT01626157
- Where
- United States
- Volunteers
- 122 people
- Sponsor
- Milene Saavedra
Acute Respiratory Distress Syndrome
Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome
A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive…
- Trial name
- SiVent (NCT02582957)
- Testing
- Sigh breaths
- Where
- 15 sites in United States
- Volunteers
- 524 people
- Sponsor
- University of Minnesota
Lung Diseases, Bronchopulmonary Dysplasia
Inhaled Nitric Oxide for Preventing Chronic Lung Disease in Premature Infants
To determine whether or not inhaled nitric oxide (iNO) safely decreases the incidence of chronic lung disease (CLD) in premature infants.
- Trial name
- NCT00006401
- Testing
- iNO
- Where
- 14 sites in United States
- Volunteers
- 793 people
- Sponsor
- University of Colorado, Denver
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Cardiac Disease
Myocardial Perfusion and Scarring in Adults With Congenital Heart Disease
This is a study of the perfusion of the myocardium in adults with specific forms of repaired congenital heart disease using established cardiac MRI techniques and correlating perfusion with clinical outcomes.
- Trial name
- NCT02861365
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Children's National Research Institute
Retinitis Pigmentosa
Stress and Vision Fluctuations in Retinitis Pigmentosa
Retinitis pigmentosa (RP) patients experience a slowly progressive, unpredictable loss of vision which eventually leads to bare or no light perception, posing a continuous threat to patients' independence.
- Trial name
- NCT00475254
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Johns Hopkins University
Dentofacial Deformities
Full-3D Computer-Assisted Workflow for the Diagnosis and Correction of Deformities? Dentofacial
"Dentofacial deformity refers to deviations from the norm of facial proportions and tooth ratios of such magnitude as to represent a disability." [1] These conditions afflicted two aspects of the patient's health: the first…
- Trial name
- NCT06806605
- Testing
- 3d analisys
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Stuttering, Down Syndrome
How Stuttering and Gestures Influence the Intelligibility of Individuals With Down Syndrome
This study wants to determine the relationship between spontaneous hand gestures, stuttering and intelligibility in individuals with Down syndrome. One third of these individuals has fluency problems, such as stuttering.
- Conditions
- Stuttering, Down Syndrome, Intelligibility, Speech, Gestures
- Trial name
- NCT03698539
- Testing
- hand gestures
- Where
- Belgium
- Volunteers
- 60 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Upper Limb Deformities, Congenital
Customized Self-Controlled Power-Driven or Mechanical Orthoses for the Upper Limb - A Pilot Study
Patients with congenital or acquired upper limb structural abnormalities and peripheral nerve or brachial plexus palsies have significant loss of function.
- Trial name
- NCT04199975
- Testing
- Orthoses
- Where
- Hong Kong
- Volunteers
- 10 people
- Sponsor
- Chinese University of Hong Kong
End Stage Renal Failure on Dialysis
Anesthetic Technique for AV Fistulae Creation
This study is being done to compare the two anesthesia techniques which are commonly used in the formation of arteriovenous fistulas: local anesthesia and regional anesthesia.
- Trial name
- NCT01727557
- Testing
- regional anesthesia, local anesthesia
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Montefiore Medical Center
Schwannomatosis, Pain
Phase 2 Study of Tanezumab in Subjects With Moderate to Severe Pain Due to Schwannomatosis
The primary objective of this study is to determine whether the administration of tanezumab, an anti-nerve growth factor (NGF) antibody, improves pain relief in schwannomatosis patients receiving background non-NSAID therapy.
- Trial name
- NCT04163419
- Testing
- Tanezumab
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Massachusetts General Hospital
Thyroid Eye Disease, Graves Orbitopathy
A Study of the Efficacy and Safety of Secukinumab 300 mg in Patients With Thyroid Eye Disease (TED)
Thyroid eye disease (TED) is a rare autoimmune, inflammatory disorder of the orbit and represents the most common extra-thyroidal manifestation of Graves' disease (GD).
- Trial name
- ORBIT (NCT04737330)
- Testing
- Secukinumab
- Where
- 5 sites in Germany
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Granulomatous Disease
Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation in patients with chronic granulomatous disease
- Trial name
- NCT03547830
- Testing
- Plerixafor, Gcsf
- Where
- Russia
- Volunteers
- 17 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Newborn Respiratory Distress, Preterm Infant
Short Term Effects of Synchronized vs. Non-synchronized NIPPV in Preterm Infants.
Comparing the cardio-respiratory adaptation and differences to non invasive ventilation techniques, nasal intermittent positive pressure ventilation (NIPPV) non synchronized vs synchronized (SNIPPV) in preterm newborns…
- Trial name
- SyncNIV (NCT03289936)
- Testing
- NIPPV, SNIPPV
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- University of Turin, Italy
Sickle Cell Disease
Neurovascular Determinants of Cognitive Function in Adults With Sickle Cell Disease
Cognitive impairment is a poorly understood, serious, and emerging complication for adult patients with sickle cell disease. Because there is extensive microvascular damage from oxidative damage in sickle cell disease, the…
- Trial name
- NCT02946905
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Heart Defects, Congenital
Rejuvenated, Washed Packed Red Blood Cells in Pediatric Cardiac Surgery
Blood transfusion is nearly always needed during open heart surgery in children less than 15 kg (35 pounds). The purpose of the red blood cells in the blood is to deliver oxygen to the organs and tissues of the body.
- Trial name
- NCT02485366
- Testing
- Rejuvesol, Cardiac surgery
- Where
- United States
- Sponsor
- Duke University
Sickle Cell Disease, Sickle Cell Trait
Bone Loss, Physical Function and Frailty in Older Women With Sickle Cell Trait Sickle Cell Trait
This is a cross-sectional, clinical research study comparing postmenopausal women of African Descent (AD) with different hemoglobin genotypes: normal and sickle cell trait (SCT). This research study has two purposes.
- Trial name
- NCT07083531
- Testing
- Healthy volunteers without SCT, Healthy volunteers with SCT
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- UConn Health
Hemophilia A or B
Safety of KN057 Prophylaxis in Patients With Haemophilia A or B
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the…
- Trial name
- NCT07545395
- Testing
- KN057
- Where
- China
- Volunteers
- 70 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Crystalline Cataract, Lens; Anomaly
Age-related Changes of Crystalline Lens Tilt and Decentration: A Swept-source Optical Coherence Tomography Study
230 participants at Eye Hospital of Wenzhou Medical University during June, 2019 and August, 2019 were enrolled in this study. 230 right eyes and 88 left eyes were included for the measurement.
- Trial name
- NCT04487795
- Where
- China
- Volunteers
- 230 people, healthy volunteers welcome
- Sponsor
- Yune Zhao
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.