Clinical Trials on Congenital Diaphragmatic Hernia in United States

Total 4288 results

Imperial College London

Recruiting

Multiple Breath Washout, a Clinimetric Dataset

Bronchiectasis | Sleep Disorder | Asthma | Cystic Fibrosis | Bronchitis | Primary Ciliary Dyskinesia

United Kingdom
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Study of Blood Samples From Newborns With Down Syndrome

Myeloid Proliferations Associated With Down Syndrome

United States, Canada, Australia, Puerto Rico
Reata, a wholly owned subsidiary of Biogen

Completed

A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome - CARDINAL (CARDINAL)

Alport Syndrome

United States, United Kingdom, Japan, Australia, France, Germany, Puerto Rico, Spain
Sol-Gel Technologies, Ltd.

Terminated

Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

Basal Cell Nevus Syndrome

United States, France, Belgium, Denmark, Germany, Italy, Netherlands, Spain, United Kingdom
Mayo Clinic

Recruiting

Prospective Research Rare Kidney Stones (ProRKS) (ProRKS)

Cystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase Deficiency

United States, Canada, Iceland, Israel
Silence Therapeutics plc

Completed

A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

Non-transfusion-dependent Thalassemia | Low Risk Myelodysplastic Syndrome | Very-Low Risk Myelodysplastic Syndrome

Thailand, Israel, Jordan, United Kingdom, Germany, Italy, Malaysia
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors (adept™2)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Taiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey
Imperial College London

Recruiting

Advanced Ultrasound Applications for Predicting AVF Outcomes (AUAPAVFO)

Chronic Kidney Diseases | Renal Failure | End Stage Renal Diseases

United Kingdom
Peloton Therapeutics, Inc., a subsidiary of Merck...

Active, not recruiting

A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)

VHL - Von Hippel-Lindau Syndrome | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL-Associated Renal Cell Carcinoma | VHL-Associated Clear Cell Renal Cell Carcinoma

United States, Denmark, United Kingdom, France
Pharnext S.C.A.
Worldwide Clinical Trials

Active, not recruiting

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PREMIER)

Charcot-Marie-Tooth Disease

United States, Spain, Belgium, Italy, Israel, Canada, Denmark, France, Germany
Cardiff University
Wyeth is now a wholly owned subsidiary of Pfizer; University of Nottingham; Royal... and other collaborators

Unknown

Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM (TESSTAL)

Tuberous Sclerosis | Lymphangioleiomyomatosis

United Kingdom
Manchester University NHS Foundation Trust
University College, London; University Hospital Southampton NHS Foundation... and other collaborators

Recruiting

Comparing the Side-lying Sleep Positioning to Back-lying in Infants With Cleft Palate (SLUMBRS2)

Obstructive Sleep Apnea | Cleft Palate

United Kingdom
Columbia University
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

A Randomized Trial of Unruptured Brain AVMs (ARUBA)

Arteriovenous Malformations, Cerebral

United States, Brazil, Canada, Australia, Austria, Finland, France, Germany, Italy, Korea, Republic of, Netherlands, Spain, Switzerland, United Kingdom
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Novartis Pharmaceuticals

Completed

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) (EXIST-1)

Tuberous Sclerosis | Subependymal Giant Cell Astrocytoma

United States, Belgium, Canada, Germany, Italy, Russian Federation, Netherlands, Poland, Australia, United Kingdom
Novo Nordisk A/S

Completed

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, Japan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Puerto Rico, Norway, Korea, Republic of, Bulgaria, Russian...
Otsuka Pharmaceutical Development & Commercialization...

Completed

Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant

Belgium, Germany, Italy, Spain, Sweden, United States, Canada, Australia, Netherlands, Czechia, Poland, Denmark, Israel, Hungary, Russian Federation, South Africa, United Kingdom, Romania, Argentina, Norway
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

Stroke With Transfusions Changing to Hydroxyurea

Hematologic Diseases | Anemia, Sickle Cell | Cerebrovascular Accident | Hemochromatosis

United States
Acceleron Pharma Inc. (a wholly owned subsidiary...

Terminated

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Charcot-Marie-Tooth Disease | Facioscapulohumeral Muscular Dystrophy

United States, Spain, Canada
Novo Nordisk A/S

Completed

High Dose of Activated Recombinant Human Factor VII for Treatment of Mild/Moderate Joint Bleeds in Haemophilia Patients With Inhibitors

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France, United Kingdom, Spain, Israel, Poland, Turkey, Hungary
King's College Hospital NHS Trust
King's College London

Completed

SELF-BREATHE RCT for Chronic Breathlessness

Liver Failure | Cancer | Asthma | Renal Failure | Cystic Fibrosis | Chronic Heart Failure | Sickle Cell Disease | Interstitial Lung Disease | COPD | Dyspnea | Post COVID-19 | Bronchiectasis Adult

United Kingdom
Sanifit Therapeutics S. A.
Clinipace Worldwide

Completed

Effect of SNF472 on Progression of Cardiovascular Calcification in End-Stage-Renal-Disease (ESRD) Patients on Hemodialysis (HD)

Cardiovascular Diseases | Cardiovascular Abnormalities | ESRD | Coronary Artery Calcification | Endstage Renal Disease | Calcifications, Vascular

Spain, United States, United Kingdom
Cardiff University
University of Canterbury

Recruiting

Mindful Parenting - A Single Case Experimental Design Study

Cleft Lip and Palate | Parenting | Neurofibromatoses | Skin Condition | Alopecia | Craniofacial | Limb Difference

United Kingdom
Novo Nordisk A/S

Completed

Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Belgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
Novartis Pharmaceuticals

Active, not recruiting

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Tuberous Sclerosis Complex

United States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States
Otsuka Pharmaceutical Development & Commercialization...

Completed

Tolvaptan Extension Study in Participants With ADPKD (TEMPO 4/4)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
Marinus Pharmaceuticals

Recruiting

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (TrustTSC)

Tuberous Sclerosis Complex

United States, Spain, Israel, United Kingdom, Germany, Canada, France, Italy
Regulus Therapeutics Inc.

Recruiting

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant | Autosomal Dominant Polycystic Kidney Disease | ADPKD

United States
University of California, San Francisco
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Epilepsy Phenome/Genome Project (EPGP)

Epilepsy | Infantile Spasms | Lennox-Gastaut Syndrome | Polymicrogyria | Localization-related Epilepsy | Periventricular Heterotopias

United States, Argentina, Australia
Otsuka Pharmaceutical Development & Commercialization...

Completed

Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Germany, Italy, Belgium, United Kingdom
Heart of England NHS Trust
University of Warwick; University of Birmingham

Completed

Chest Wall Motion Analysis in Disease (CWM)

Lung Neoplasms | Pulmonary Disease, Chronic Obstructive | Surgery | Cystic Fibrosis | Mesothelioma | Empyema, Pleural | Pectus Excavatum | Pectus Carinatum

United Kingdom
Hereditary Neuropathy Foundation

Recruiting

Natural History Study for Charcot Marie Tooth Disease

Charcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... and other conditions

United States
National Tuberous Sclerosis Association

Recruiting

TSC Biosample Repository and Natural History Database

Tuberous Sclerosis | Lymphangioleiomyomatosis

United States, Canada
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, Israel, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon, Netherlands
Novo Nordisk A/S

Completed

Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Saudi Arabia, Algeria, Turkey, Germany, Poland, Netherlands, Italy, Belgium, South Africa, United Kingdom, Sweden, Austria, Portugal, France, Czech Republic, Venezuela
Vascular Therapies, Inc.

Recruiting

A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery (ACCESS2)

End Stage Renal Disease | ESRD | End Stage Kidney Disease | Arteriovenous Fistula | Vascular Access Complication | Chronic Kidney Failure | Complication of Hemodialysis | Complication of Renal Dialysis

United States, United Kingdom
Novo Nordisk A/S

Completed

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France, United States, Spain, Brazil, Philippines, South Africa, Poland, Turkey, Romania, Bulgaria, Russian Federation, Argentina
Simons Searchlight
Boston Children's Hospital; Geisinger Clinic; Simons Foundation

Recruiting

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

SMARCA4 Gene Mutation | DDX3X | 16P11.2 Deletion Syndrome | 16p11.2 Duplications | 1Q21.1 Deletion | 1Q21.1 Microduplication Syndrome (Disorder) | ACTL6B | ADNP | AHDC1 | ANK2 | ANKRD11 | ARID1B | ASH1L | BCL11A | CHAMP1 | CHD2 | CHD8 | CSNK2A1 | CTBP1 | CTNNB1 Gene Mutation | CUL3 | DNMT3A | DSCAM | DYRK1A | FOXP1 | GRIN2A | GRIN2B | HIVEP2-Related Intellectual... and other conditions

United States
Sanguine Biosciences

Recruiting

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Heart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditions

United States
Marinus Pharmaceuticals

Enrolling by invitation

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, Australia, China, Italy, Israel, Germany, France, Canada, Belgium
Hoffmann-La Roche

Completed

A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas

Basal Cell Carcinoma

United States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada
PellePharm, Inc.

Completed

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

Basal Cell Nevus Syndrome

United States, Spain, Germany, United Kingdom, Italy, France, Belgium, Canada, Denmark, Netherlands
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (TEMPO3:4)

Polycystic Kidney Disease, Autosomal Dominant

United States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
TScan Therapeutics, Inc.

Recruiting

A Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors

Melanoma | Cervical Cancer | Head and Neck Cancer | HPV-Related Malignancy | Ovarian Cancer | HPV-Related Carcinoma | HPV-Related Cervical Carcinoma | HPV Positive Oropharyngeal Squamous Cell Carcinoma | Non-small Cell Carcinoma | HPV - Anogenital Human Papilloma Virus Infection | HPV-Related Adenocarcinoma | HPV-Related Adenosquamous Carcinoma and other conditions

United States
Roswell Park Cancer Institute
National Cancer Institute (NCI)

Completed

Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders

Primary Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditions

United States
Novartis Pharmaceuticals

Completed

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures (EXIST-3)

Tuberous Sclerosis Complex-associated Refractory Seizures

United States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
Reata, a wholly owned subsidiary of Biogen

Terminated

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON (FALCON)

Autosomal Dominant Polycystic Kidney | ADPKD

United States, Spain, United Kingdom, Australia, France, Japan, Germany, Belgium, Czechia, Italy

Clinical Trials on Congenital Diaphragmatic Hernia in United States

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