Clinical trials: Defect, Congenital
Sickle Cell Disease
Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring
Brief Summary: * A short description of the clinical study, including a brief statement of the clinical study's hypothesis, written in language intended for the lay public. Limit: 5000 characters.
- Trial name
- OPTIMDREP (NCT06464458)
- Testing
- management on hydroxyurea by therapeutic pharmacological monitoring
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Strasbourg, France
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
Virtual Reality Devices as an Adjunct to Usual Care for Patients With Sickle Cell Disease Experiencing Vaso-Occlusive Crises
This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Acute Pain
- Trial name
- NCT06184126
- Testing
- Virtual Reality Headset and Hand-Held Controllers, Blindfolding and Noise Cancelling
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Maryland, Baltimore
Tourette Syndrome
A Study to Examine the Efficacy of a Therapeutic THX-110 for Tourette Syndrome
This study evaluates the efficacy and safety of THX-110 in the management of tics and other symptoms (e.g. rage attacks, anxiety, depression, sleep difficulties) in patients with Tourette syndrome.
- Trial name
- NCT03651726
- Testing
- THX-110 (dronabinol plus PEA)
- Where
- 2 sites in Germany
- Volunteers
- 60 people
- Sponsor
- Neurothera Labs Inc.
Intracranial Arteriovenous Malformations
Nationwide Treatment Survey of Intracranial Arteriovenous Malformation in China
This register study will collect the treatment information of the intracranial arteriovenous malformation patients in China. We aim to understand the current treatment situation of the disease in China.
- Trial name
- NTSIAVMC (NCT01803685)
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Dr. Yong Cao
Prader-Willi Syndrome
PWS European Blood Bank for Infants and Controls From 0 to 48 Months
The present project aims to determine the underlying mechanisms for the switch from failure to thrive to excessive weight gain and hyperphagia with impaired satiety in PWS.
- Trial name
- NCT02529085
- Testing
- blood samples
- Where
- 6 sites in Belgium, France and 4 more
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Infant, Extremely Premature, Acute Lung Injury
Intra-tracheal Instillation of Budesonide to Prevent Chronic Lung Disease
Preterm infants are randomized to received either Intra-tracheal instillation of budesonide using surfactant as vehicle or a placebo.
- Conditions
- Infant, Extremely Premature, Acute Lung Injury, Chronic Lung Disease of Prematurity, Budesonide
- Trial name
- STOPCLD (NCT05364385)
- Testing
- Infants in the study group (S/B group) will receive surfactant 2.5 mls/kg and Budesonide 0.5 mg (1mL)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Arizona
Fontan Procedure, Hypoplastic Left Heart Syndrome
Impact of Vitamin C on Endothelial Function and Exercise Capacity in Fontan-Palliated Patients
In this study, investigators will evaluate the effect of vitamin C on endothelial function, exercise tolerance and quality of life in patients with a single ventricle who have been palliated to Fontan physiology.
- Conditions
- Fontan Procedure, Hypoplastic Left Heart Syndrome, Tricuspid Atresia
- Trial name
- NCT00974025
- Testing
- Vitamin C
- Where
- United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Papillomavirus Infections
Diversity Analysis of Vaginal Microbiota on Women With High-risk Human Papillomavirus Infection
Since other genital infections enhance HIV susceptibility by inducing inflammation, the investigators study the relationship between the vaginal microbiota composition and the risk of HPV infection, cervical cytological…
- Trial name
- NCT03548740
- Testing
- Human Papillomavirus
- Where
- China
- Volunteers
- 151 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Genetics Disease, Type2 Diabetes
Effects of Sitagliptin in Individuals With Genetically Decreased DPP4
This is a pilot clinical trial to test the hypothesis that during sitagliptin (DPP4 inhibitor), individuals heterozygous for DPP4 loss of function variants will have a reduction in DPP4 activity and antigen, lower glucose after a…
- Conditions
- Genetics Disease, Type2 Diabetes, Heart Failure
- Trial name
- NCT04323189
- Testing
- Sitagliptin 100mg
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pennsylvania
Preg Complications: Hemorrhagic, Infant, Premature, Diseases
Maternal & Infant Morbidity and Mortality
The purpose of this project is to evaluate maternal and infant outcomes based on race at UVA hospital. Health inequities are influenced by a combination of Social, Political, and Clinical determinants of health.
- Conditions
- Preg Complications: Hemorrhagic, Infant, Premature, Diseases, Preg Complications: Hemorrhagic Intervillous of Placenta, Preg Complications: Bleeding From Female Genital Tract
- Trial name
- NCT06145568
- Testing
- Pregnant Women
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Temozolomide and O6-benzylguanine in Treating Children With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00020150
- Testing
- O6-benzylguanine, temozolomide
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Pompe Disease
Pregnancy and Birth Outcome in Women With Pompe Disease
This study explores the outcome and effect of pregnancy on Pompe Disease. The results are expected to guide clinicians in counseling and care of women with Pompe disease, who are planning to become pregnant, and during the…
- Trial name
- NCT01556516
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- O & O Alpan LLC
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).
- Trial name
- NCT06460506
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 18 sites in Australia, Canada and 5 more
- Volunteers
- 50 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A, Hemophilia B
Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability
Using anonymized patient data collected as part of the WAPPS-Hemo project to explore the sources of variability in individual pharmacokinetics (PK); use the sources of variability to improve the performance of the WAPPS-Hemo…
- Trial name
- WAPPS-Hemo (NCT03533504)
- Testing
- Variability in individual PK
- Where
- Canada
- Volunteers
- 1,000 people
- Sponsor
- McMaster University
Pompe Disease
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and profound muscle weakness.
- Trial name
- NCT00976352
- Testing
- rAAV1-CMV-GAA (study agent) Administration, RMST
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Florida
22q.11.2 Deletion/Duplication
Non-Invasive Chromosomal Evaluation of 22q11.2
This study is being conducted to develop and evaluate a cell-free fetal DNA test (Harmony) for non-invasive prenatal detection of 22q11.2 chromosomal deletion or duplication.
- Trial name
- 22Q (NCT02541058)
- Where
- Belgium
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Chest Wall Disorder
Comparison of Analgesic Requirements in Patients Receiving Nuss Operation Using 2 Different Anesthetic Adjuvants
Nowadays, general anaesthesia is carried under "balanced anesthesia technique" in which many anesthetic adjuvants are used simultaneously, including opioid analgesics in order to reduce the amount of inhalation agents.
- Trial name
- ARNRD (NCT04073758)
- Testing
- Dexmedetomidine
- Where
- South Korea
- Volunteers
- 62 people
- Sponsor
- Jung Min Koo
Sleep Apnea, Obstructive, Respiration Disorders
Treatment of Children With Obstructive Sleep Apnea and Laryngomalacia: the Role of Laser Supraglottoplasty
This is a research study of the effect of treating laryngomalacia (floppiness of tissue on top of the voice box that can possibly block breathing) found in association with obstructive sleep apnea (blockage of breathing while…
- Trial name
- NCT00394550
- Testing
- supraglottoplasty with laser
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Indiana University
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Background: CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age.
- Trial name
- NCT03307304
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Missing Teeth, Edentulous Alveolar Ridge
Hybrid Funnel Technique a Innovative Technique for Implant Site Preparation
Hybrid Funnel Technique arises from the union of subtractive and non-subtractive techniques for implant site preparation. Hybrid funnel technique is performed with specially designed drills and osteotome.
- Conditions
- Missing Teeth, Edentulous Alveolar Ridge, Tooth Loss
- Trial name
- NCT05499533
- Testing
- implant site preparation
- Where
- Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Luigi Canullo