Clinical trials: Congenital Malformation
Polycystic Kidney Disease
Evaluation of Gut Bacteria in Patients With Polycystic Kidney Disease
Gut microbes can influence numerous aspects of human biology. Alterations in the function and composition of gut microbial flora (gut microbiota ) have been linked to inflammatory bowel disease, chronic inflammation…
- Trial name
- NCT02142101
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Atopic Dermatitis (AD)
A Study for HSK44459 in Participants With Atopic Dermatitis
The purpose of this study is to assess the efficacy and safety of HSK44459 in adults with Atopic Dermatitis
- Trial name
- NCT06996912
- Testing
- HSK44459
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Xizang Haisco Pharmaceutical Co., Ltd
Hyperinsulinemia Hypoglycemia
Evaluation of Single Ascending Doses of Subcutaneous Exendin 9-39 in Patients With Post-Bariatric Hypoglycemia
This study is designed to evaluate the safety, efficacy, and pharmacokinetic profile of single ascending doses of exendin 9-39 administered by subcutaneous route in subjects with post-bariatric hypoglycemia.
- Trial name
- NCT02996812
- Testing
- Exendin (9-39)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Tracey McLaughlin
Congenital Heart Disease
The Relationship Between Brain MRI Phenotypes, Genes and Cognitive Outcome in CHD Adults
The main purpose of this proposal is to perform novel MRI analyses to determine the brain organizational changes associated with altered executive function and the modulating role of variants in neuroresilience and hypoxia…
- Trial name
- NCT05258981
- Testing
- MRI
- Where
- United States
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Cleft Palate, Plasma Rich in Growth Factors
Role of Plasma Rich Growth Factor in Repair Primary Cleft Palate
Does the plasma-rich growth factor (PRGF) with primary cleft palate repair accelerate wound healing and prevent post-operative oronasal fistula occurrence?
- Trial name
- PRGF (NCT06889181)
- Testing
- Plasma Rich in Growth Factors (PRGF)
- Where
- Egypt
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Cairo University
Cystic Fibrosis
Dose Escalation Study of KB001 in Cystic Fibrosis Patients Infected With Pseudomonas Aeruginosa
The primary objective of this study is to evaluate the safety and tolerability of a single dose of KB001 in Cystic Fibrosis patients infected with Pseudomonas aeruginosa (Pa)
- Trial name
- NCT00638365
- Testing
- KB001
- Where
- 11 sites in United States
- Volunteers
- 27 people
- Sponsor
- Humanigen, Inc.
Sickle Cell Disease, Hyperbaric Oxygen Therapy
Hyperbaric-oxygen Therapy (HBOT) Versus Placebo for Treating Vaso-Occlusive Crisis (VOC) in Sickle Cell Disease (SCD)
This is a randomised, controlled, double-blind, placebo trial of HBOT (intervention) superiority in the treatment of VOC in SCD, to demonstrate the effectiveness of HBOT for the decrease in pain level in the treatment of SCD-VOC.
- Conditions
- Sickle Cell Disease, Hyperbaric Oxygen Therapy, Vaso-occlusive Crisis
- Trial name
- HBOT-SCD (NCT05289700)
- Testing
- HBOT in Hyperbaric chamber
- Where
- 3 sites in France, Switzerland
- Volunteers
- 100 people
- Sponsor
- University Hospital, Geneva
Graves' Ophthalmopathy, Hypercholesterolemia
Graves' Orbitopathy and Hypercholesterolemia
The investigators have recently observed retrospectively that the occurrence of Graves' Orbitopathy in patients with Graves' disease is less frequent in patients with normal cholesterol levels, in line with another recent…
- Trial name
- GOCol (NCT02721992)
- Testing
- No intervention
- Where
- Italy
- Volunteers
- 250 people
- Sponsor
- University of Pisa
Beta Thalassaemia
Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)
OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene.
- Trial name
- NCT03275051
- Testing
- Safety and Efficacy assessments
- Where
- Italy
- Volunteers
- 9 people
- Sponsor
- IRCCS San Raffaele
Velopharyngeal Insufficiency, Real Time MRI
Etiological Study of Persistent Velopharyngeal Insufficiency in Children With Operated Velopalatine Cleft by Analysis of Velopharyngeal Motor Skills in Static and Dynamic MRI
Velopharyngeal insufficiency is defined as the inability of the soft palate to isolate the nasopharynx from the oropharynx. It is a frequent sequela in patients with a velopalatine cleft despite anatomical restoration of the soft…
- Conditions
- Velopharyngeal Insufficiency, Real Time MRI, Cleft Palate
- Trial name
- FENTIRM (NCT06072495)
- Testing
- real time MRI
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Tetralogy of Fallot
Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -
Tetralogy of Fallot (TOF) is the most common cyanotic congenital heart defect with the vast majority of survivors of corrective surgery left with some degree of right ventricular (RV) volume overload due to pulmonary…
- Trial name
- TOFandPVR (NCT03634072)
- Testing
- PVR
- Where
- 6 sites in United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital of Philadelphia
Rheumatoid Arthritis, Osteoarthritis
A Multi-centre Study to Assess the Long-term Performance of the DePuy ASR™ System in Primary Hip Resurfacing Surgery
The purpose of this study is to monitor the performance and determine the metal ion release of the DePuy ASR™ System in the treatment of patients with hip joint disease requiring hip resurfacing surgery.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00208377
- Testing
- DePuy ASR Hip System
- Where
- 3 sites in Australia, Germany and 1 more
- Volunteers
- 228 people
- Sponsor
- DePuy International
Alport Syndrome
Effects of an Intensified Treatment With ACE-I,ATA II and Statins in Alport Syndrome
Alport syndrome (AS) represents a form of progressive hereditary nephritis in which the genetic defect resides in the synthesis of one of several subunits of type IV collagen, the predominant constituent of basement membranes in…
- Trial name
- NCT00309257
- Testing
- ACE I, ATA II and Statins, Benazepril, Valsartan and Fluvastatin
- Where
- Italy
- Volunteers
- 9 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Tetraplegia, Tetraplegia/Tetraparesis
[BrainConnexion] - Neurodevice Phase I Trial
This study aims to evaluate the safety of a wireless implantable neurodevice microsystem in tetraplegic patients, as well as the efficacy of the electrodes for long-term recording of neural activities and the successful control…
- Conditions
- Tetraplegia, Tetraplegia/Tetraparesis, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis
- Trial name
- NCT03811301
- Testing
- BrainConnexion
- Where
- Singapore
- Volunteers
- 5 people
- Sponsor
- National Neuroscience Institute
Atopic Dermatitis
Topical Treatment for Atopic Dermatitis
Medication adherence is a poorly studied phenomenon that challenges both patients and physicians. 50% of individuals with chronic disease are not adherent to their medication regimen.
- Trial name
- NCT06244212
- Testing
- Demonstration of applying triamcinolone cream, Mobile App Use, Standard of Care
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Wake Forest University Health Sciences
Cystic Fibrosis-related Diabetes
EnVision CF Multicenter Study of Glucose Tolerance in Cystic Fibrosis
Cystic Fibrosis Related Diabetes has been identified by the CF community as one of the top ten priorities for CF research. In CF clinical decline due to dysglycemia begins early, prior to diagnosis of diabetes and increases…
- Trial name
- NCT03650712
- Testing
- Oral glucose tolerance test, Continuous glucose monitoring, Dexa scan
- Where
- 4 sites in United States
- Volunteers
- 317 people
- Sponsor
- Katie Larson Ode
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Sickle Cell Disease, Executive Dysfunction
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork…
- Conditions
- Sickle Cell Disease, Executive Dysfunction, Cognitive Impairment, Attention Deficit/Hyperactivity Disorder (ADHD)
- Trial name
- NCT07226219
- Testing
- Extended-Release Methylphenidate
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- St. Jude Children's Research Hospital
Cystic Fibrosis
Inspiratory Muscle Training in Individuals With Cystic Fibrosis
Cystic fibrosis is a genetic disease that affects some organs of the human body. Among them, the lungs tend to be the most affected due to the accumulation of mucus in the airways, which in addition to avoiding the passage of…
- Trial name
- NCT03737630
- Testing
- Inspiratory muscle training
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- Universidade Federal do Rio Grande do Norte
Maxillofacial Abnormalities
Evaluation of the Long-term Results of Extended Supra-basillary Genioplasty of the "Chin Wing" Type Without Bone Graft
The correction of maxillomandibular dysmorphoses, responsible for both functional discomfort (lip incompetence, ventilation disorders, periodontal disease) but also aesthetic, calls in particular for genioplasty, of which there…
- Trial name
- SASCWatch (NCT06183853)
- Where
- France
- Volunteers
- 14 people
- Sponsor
- University Hospital, Strasbourg, France
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