Clinical trials: Congenital Diseases
Congenital Heart Disease (CHD), Ventricular Septal Defects (VSD)
Early Warning of Structural Complications in Perimembranous VSD
The goal of this observational study is to develop and test a AI-based prediction model for children with perimembranous ventricular septal defect, also called PMVSD.
- Trial name
- NCT07662343
- Where
- China
- Volunteers
- 11,683 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Solid Tumor Malignancy
Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR…
- Trial name
- NCT03822117
- Testing
- Pemigatinib
- Where
- 90 sites in United States, Denmark and 9 more
- Volunteers
- 111 people
- Sponsor
- Incyte Corporation
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Heart Failure Congenital, Single-ventricle
REHAB Fontan Failure: A Trial of Cardiac Rehabilitation
The goal of this clinical trial is to compare the impact of cardiac rehabilitation on Fontan failure patients' exertional tolerance, frailty, and quality of life. 1.
- Trial name
- NCT06150950
- Testing
- Cardiac rehabilitation, Usual care
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Stanford University
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing…
- Trial name
- NCT06241950
- Testing
- delandistrogene moxeparvovec, imlifidase
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Sarepta Therapeutics, Inc.
Cystic Fibrosis
Use of Capnography for the Follow-up of Cystic Fibrosis Children
The respiratory system involvement in cystic fibrosis(CF) influences the prognosis and course of disease. Respiratory assessment is based on spirometry, but its main parameter, the maximal expiratory volume in the first second…
- Trial name
- MUCOCAP (NCT04814797)
- Testing
- Capnography data included in Lung Clearance Index measurement
- Where
- 2 sites in France
- Volunteers
- 76 people
- Sponsor
- Central Hospital, Nancy, France
Congenital Heart Disease
Quality of Life and Neurodevelopment Assessment of Children With Congenital Heart Disease Aged 2 to 4 Years
Congenital heart diseases (CHD) are the firt cause of congenital malformations (8 for 1000 births). Since the 90's, great advances in prenatal diagnosis, pediatric cardiac surgery, intensive care, and cardiac catheterization have…
- Trial name
- QoLCHD (NCT04812210)
- Where
- France
- Volunteers
- 248 people
- Sponsor
- University Hospital, Montpellier
Mandibular Hypoplasia, Relapse
Evaluation of Early Relapse After Mandibular Lengthening Surgery
The goal of this study is to evaluate whether the use of a bone graft substitute at the osteotomy site will result in better stability and diminish early relapse after mandibular lengthening surgery.
- Trial name
- NCT03710512
- Testing
- Bilateral sagittal split osteotomy
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Universitair Ziekenhuis Brussel
Intraventricular Hemorrhage, Periventricular Leukomalacia
Effects of Delayed Cord Clamp and/or Indomethacin on Preterm Infant Brain Injury
Intraventricular hemorrhage (IVH) and periventricular leukomalacia (PVL) are brain lesions that commonly occur in preterm infants and are well-recognized major contributors to long-term brain injury and related disabilities later…
- Conditions
- Intraventricular Hemorrhage, Periventricular Leukomalacia, Brain Injury, Renal Injury
- Trial name
- NCT02221219
- Testing
- Indomethacin, delay in umbilical cord clamp at birth, immediate cord clamp at birth
- Where
- United States
- Volunteers
- 256 people
- Sponsor
- Hong Huang
Irritable Bowel Syndrome (IBS)
New Microbiota-endocrine Axis in Fructose Malabsorption-caused Visceral Hypersensitivity in Irritable Bowel Syndrome.
Irritable bowel syndrome (IBS) affects around 4% of the general population and remains the most common functional bowel disorder.
- Trial name
- ENDOLORII (NCT07337707)
- Testing
- urine intestinal permeability test or lactulose/mannitol test, Blood test, food diary
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Rouen
Smith-Lemli-Opitz Syndrome
Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
This study will evaluate the safety and effectiveness of simvastatin in treating children with Smith-Lemli-Opitz syndrome (SLOS).
- Trial name
- NCT00064792
- Testing
- Simvastatin Susp., OraPlus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Forbes Porter, M.D.
Behçet Disease
Dusquetide for the Treatment of Behcet's Disease
This is a clinical study to see if dusquetide can treat flares of oral and genital ulcers caused by Behcet's Disease. Study participants will receive an infusion of dusquetide twice a week for 4 weeks (8 treatments total), with…
- Trial name
- NCT06386744
- Testing
- Dusquetide
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Soligenix
Thyroid Associated Ophthalmopathies, Graves´ Ophthalmopathy
Tocilizumab Treatment in Graves´ Ophthalmopathy (Graves´ Orbitopathy or Thyroid Eye Disease)
The purpose is to investigate tocilizumab administration in patients with moderately to severely or sight-threatening GO (Graves' ophthalmopathy) without response to treatment with corticoid intravenous pulses.
- Conditions
- Thyroid Associated Ophthalmopathies, Graves´ Ophthalmopathy, Thyroid Eye Disease
- Trial name
- GRC (NCT01297699)
- Testing
- Tocilizumab (RoActemra®), Sterile 0.9% Sodium Chloride
- Where
- 9 sites in Spain
- Volunteers
- 32 people
- Sponsor
- Hospital Clinico Universitario de Santiago
Leukemia
Chemotherapy With or Without Gemtuzumab Ozogamicin in Treating Older Patients With Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies can locate cancer cells and either kill them or deliver cancer-killing substances to…
- Trial name
- AML-17 (NCT00052299)
- Testing
- cytarabine, etoposide, gemtuzumab ozogamicin
- Where
- 55 sites in Austria, Belgium and 5 more
- Volunteers
- 472 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Nanophthalmos
Extensive Circumferential Partial Thickness Sclerectomy in Nanophthalmic Eyes
Purpose: To describe an extensive scleral excision technique to treat or prevent uveal effusion in nanophthalmic eyes. Design: Prospective interventional case series. Methods: Setting: Institutional.
- Trial name
- NCT03748732
- Testing
- sclerectomy
- Where
- Lebanon
- Volunteers
- 30 people
- Sponsor
- Rafic Hariri University Hospital
Hereditary Angioedema
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This is an open-label, multicenter extension trial to evaluate the long-term safety of KVD900 in patients who are 12 years or older with HAE type I or II.
- Trial name
- NCT05505916
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 71 sites in United States, Australia and 21 more
- Volunteers
- 145 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Prostate Cancer
Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer
Background: Enzalutamide is given to treat metastatic prostate cancer. But it takes 4 pills to deliver the effective dose. This can be difficult for people already taking multiple drugs.
- Trial name
- NCT03478904
- Testing
- Enzalutamide Liquid, Enzalutamide Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Pain, Marfan Syndrome
Single-Session "Empowered Relief" Class for Marfan Syndrome and Related Conditions
Empowered Relief (ER) is a 1-session pain relief skills intervention that is delivered in-person or online by certified clinicians to groups of patients with acute or chronic pain.
- Conditions
- Chronic Pain, Marfan Syndrome, Vascular Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome
- Trial name
- NCT05980104
- Testing
- Empowered Relief
- Where
- United States
- Volunteers
- 92 people
- Sponsor
- Stanford University
Atopic Dermatitis, Type 1 Hypersensitivity
Allergic Disease Onset Prevention Study
This is a Phase 1b/2, randomized, double-blind, multi-center study to evaluate the safety, tolerability, and preliminary clinical efficacy of STMC-103H in neonates and infants at risk for developing allergic disease (Type 1…
- Trial name
- adored (NCT05003804)
- Testing
- STMC-103H
- Where
- 31 sites in United States, Australia and 1 more
- Volunteers
- 283 people, healthy volunteers welcome
- Sponsor
- Siolta Therapeutics, Inc.
Neonatal SEPSIS
Neonatal Sepsis at Neonatal Intensive Care Units in Ghana
Background: Neonatal sepsis is a major contributor to global under five mortality. In developing countries a major proportion of neonatal sepsis is thought to emanate from the healthcare setting, due to challenges in infection…
- Trial name
- NCT03755635
- Testing
- The WHO multimodal hand hygiene strategy
- Where
- Ghana
- Volunteers
- 5,433 people, healthy volunteers welcome
- Sponsor
- Stephanie Bjerrum
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