Clinical trials: Fetal Developmental Abnormality
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
Erythropoietic Protoporphyria
Sorbent Therapy of the Cutaneous Porphyrias
The investigators demonstrated that cholestyramine is an effective binding agent in vitro for porphyrins. A few isolated case reports of treatment of individuals with a cutaneous porphyria suggest that cholestyramine and…
- Trial name
- EPP (NCT01422915)
- Testing
- Colestipol
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Glycogen Storage Disease Type II, Pompe Disease
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
GSD-II (also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- NCT00051935
- Testing
- Alglucosidase alfa
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Urea Cycle Disorder, Inborn Errors of Metabolism
A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
Original study: Study aims to enroll 3-5 children with confirmed Urinary Cycle Disorder (UCD). Subjects meeting the inclusion and exclusion criteria are included during the baseline visit.
- Trial name
- NCT03911089
- Testing
- UCD Anamix Infant
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Nutricia Research
Friedreich's Ataxia
An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as…
- Trial name
- NCT01921868
- Testing
- Acetyl-L-Carnitine
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of South Florida
Disorder of Consciousness
Metabolic Abnormalities and Intestinal Microecology in Patients With Chronic Disorders of Consciousness
Following severe traumatic brain injury, patients may remain unconscious for many years. It is the intestinal microbiome and metabolomics analysis comparing differentially intestinal microflora and metabolites between patients…
- Trial name
- NCT04530968
- Testing
- No intervention
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Zhejiang University
Genetic Disease, Neurologic Disorder
Use of Long Read Genome Sequencing in Patients Suffering From Neurodevelopmental Troubles
Patients with neurodevelopmental diseases and their families need to identify the genetic cause of the disease to allow for recognition of the disability, genetic counseling, and possible hope for participation in therapeutic…
- Conditions
- Genetic Disease, Neurologic Disorder, Developmental Delay Disorder, Growth Disorders
- Trial name
- HiFi-NDD (NCT05643274)
- Where
- 5 sites in France
- Volunteers
- 10 people
- Sponsor
- Nantes University Hospital
Pediatrics, Neurological Abnormality
Turkish Version of the BRIEF-Hammersmith Infant Neurological Examination (HINE)
The American Academy of Pediatrics defines infants at risk as those with preterm birth, special health care needs, family risk factors, and those at risk of early death.
- Conditions
- Pediatrics, Neurological Abnormality, Neurodevelopment Outcome
- Trial name
- BRIEF-HINE (NCT07182474)
- Where
- Turkey (Türkiye)
- Volunteers
- 120 people
- Sponsor
- Kahramanmaras Sutcu Imam University
Bleeding Disorder, Knee Injuries
Protection of Knee Joints in Bleeding Disorder Patients
INTRODUCTION Knee supports, usually made with sponge cushion, are used to protect knee joint and prevent its injury. Sponge has less elasticity and durability compared with natural rubber.
- Conditions
- Bleeding Disorder, Knee Injuries, Hemophilia
- Trial name
- NCT06039904
- Testing
- Natural rubber knee support, Sponge knee support
- Where
- Thailand
- Volunteers
- 44 people
- Sponsor
- Ramathibodi Hospital
Hypoplastic Left Heart Syndrome
The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe…
- Trial name
- CHILD (NCT03406884)
- Testing
- c-kit+ cells
- Where
- 4 sites in United States
- Volunteers
- 25 people
- Sponsor
- Joshua M Hare
Urothelial Carcinoma, Lynch Syndrome
Urothelial Cancer Screening in Individuals With Lynch Syndrome Using a Urine Tumor DNA Panel (LS-URO Study)
Lynch syndrome (LS) is an inherited cancer predisposition syndrome caused by pathogenic germline variants in DNA mismatch repair (MMR) genes.
- Trial name
- NCT06218433
- Testing
- Urothelial cancer screening using urine tumor DNA test, Urothelial cancer screening using urine cytology (comparator)
- Where
- 2 sites in Canada, Finland
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Tampere University Hospital
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Stroke, Transient Ischemic Attack
International PFO Consortium
The prevalence of patent foramen ovale (PFO) is about 25% in the general population and approximately 40% in patients who have ischemic stroke of unknown cause (cryptogenic stroke).
- Trial name
- NCT00859885
- Testing
- Antithrombotic treatment, percutaneous device closure of PFO
- Where
- 18 sites in United States, Belgium and 4 more
- Volunteers
- 1,500 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Cystic Fibrosis
Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients
Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi
- Trial name
- NCT02170025
- Testing
- Riociguat (Adempas, BAY63-2521)
- Where
- 10 sites in United States, Belgium and 5 more
- Volunteers
- 21 people
- Sponsor
- Bayer
Fractures, Bone, Cardiovascular Abnormalities
Using a Remote Patient Monitoring Alert System to Improve Care
The purpose of this study is to determine the efficacy of a remote patient monitoring platform and alert system in reducing adverse events for hospitalized geriatric orthopedic trauma patients.
- Conditions
- Fractures, Bone, Cardiovascular Abnormalities, Respiratory System Abnormalities, Hemorrhage
- Trial name
- NCT02933840
- Testing
- AlertWatch
- Sponsor
- Massachusetts General Hospital
Haemophilia A, Haemophilia B
A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia
The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents.
- Trial name
- eTHINK (NCT03660774)
- Testing
- No treatment given
- Where
- 22 sites in United States
- Volunteers
- 563 people
- Sponsor
- Novo Nordisk A/S
Cardiac Arrhythmias, Accessory Pathway
Cryotherapy Versus Radiofrequency Ablation for Treatment of Para-hisian Accessory Pathways
A randomized pilot study to evaluate safety and efficacy endpoints for treatment of para-hisian accessory pathways according to two different strategies of lesion formation.
- Conditions
- Cardiac Arrhythmias, Accessory Pathway, Wolf Parkinson White Syndrome
- Trial name
- NCT04361006
- Testing
- Radiofrequency (RF), Cryotherapy (CRYO)
- Where
- Brazil
- Volunteers
- 30 people
- Sponsor
- University of Sao Paulo General Hospital
Hyperinsulinemic Hypoglycemia
Evaluating Exenatide for the Treatment of Postprandial Hyperinsulinemic Hypoglycemia
The purpose of the study is to evaluate the effectiveness of exenatide in adults experiencing episodes of hyperinsulinemic hypoglycemia following Roux-en-Y bariatric surgery.
- Trial name
- NCT02685852
- Testing
- Exenatide, Acarbose
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Minnesota
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Postpartum
Analyse Of Early Skın To Skın Contact's Impact Upon Maternal-Infant Bondıng And Perceptıon Of Traumatıc Bırth
This study will carry out as a randomized controlled experimental study in order to examine the effect of early skin-to-skin contact on mother-infant attachment and traumatic birth perception and to provide new information to the…
- Trial name
- NCT05802147
- Testing
- skin-to-skin contact
- Where
- Turkey (Türkiye)
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Kahramanmaras Sutcu Imam University