Search clinical trials for: Hereditary study for Primary Hyperoxaluria

Total 386840 results

Mayo Clinic

Completed

Primary Hyperoxaluria Mutation Genotyping/Phenotyping

Primary Hyperoxaluria

United States
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oxalosis...

Completed

Primary Hyperoxaluria Mutation Genotyping

Primary Hyperoxaluria

United States
Octapharma

Not yet recruiting

Phase 3 Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema
Pharvaris Netherlands B.V.

Recruiting

Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema (RAPIDe-3)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States
Foundation For Rare Disease Research
Institute for Asthma & Allergy; Virant Diagnostics, Inc.; MedBio Reference Laboratories...

Not yet recruiting

Angioedema Biomarker Research Study

Angioedema | Urticaria | C1 Inhibitor Deficiency | Angioedemas, Hereditary | Mastocytosis | Indolent Systemic Mastocytosis | Systemic Mastocytoses | ACE Inhibitor-Induced Angioedema
Royal Marsden NHS Foundation Trust

Recruiting

Inherited Cancer Early Diagnosis (ICED) Study (ICED)

Lynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene Mutation

United Kingdom
NYU Langone Health

Not yet recruiting

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
Memorial Sloan Kettering Cancer Center

Recruiting

A Study of Avutometinib for People With Solid Tumor Cancers

Solid Tumor | Neuroblastoma | Solid Tumor, Adult | Primary Brain Tumor | Solid Carcinoma | Low-grade Glioma | Central Nervous System Tumor | Refractory Cancer | CNS Tumor, Adult | CNS Tumors | NF1 | Plexiform Neurofibroma | CNS Tumor, Childhood | Optic Pathway Gliomas | MAP Kinase Family Gene Mutation

United States
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Jazz Pharmaceuticals

Recruiting

A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex (EpiCom)

Tuberous Sclerosis Complex Associated Neuropsychiatric Disease

United States, United Kingdom, Poland
CSL Behring

Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Israel
National Taiwan University Hospital

Recruiting

A Retrospective Study for Multiple Endocrine Neoplasia

Multiple Endocrine Neoplasia

Taiwan
Astria Therapeutics, Inc.

Recruiting

A Study of STAR-0215 in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, United Kingdom, Canada, Germany, Bulgaria
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Takeda

Recruiting

A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

Hereditary Angioedema (HAE)

United Kingdom
Takeda

Recruiting

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United Kingdom
Pharvaris Netherlands B.V.

Recruiting

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-2)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Puerto Rico, Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom
Novartis Pharmaceuticals

Recruiting

A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

Renal Angiomyolipoma

Taiwan
BioMarin Pharmaceutical

Recruiting

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (HAErmony-1)

Hereditary Angioedema | HAE

United States, Spain, Australia
Darcy Krueger

Recruiting

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)

Epilepsy | Tuberous Sclerosis Complex

United States
Alnylam Pharmaceuticals

Recruiting

BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) (BONAPH1DE)

Primary Hyperoxaluria Type 1

France, Spain, United States, Israel, Belgium, Switzerland, Germany, United Kingdom, Italy, Canada
National Institute of Diabetes and Digestive and...

Recruiting

Natural History Study of Parathyroid Disorders

Primary Hyperparathyroidism | Pseudohypoparathyroidism | Parathyroid Cancer | Inheritable Bone Diseases | Multiple Endocrine Neoplasia, Type 1

United States
Paul Szabolcs

Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias

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

Recruiting

A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT

Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Recurrent Kidney Wilms Tumor | Anaplastic Kidney Wilms Tumor

United States, Canada, Puerto Rico, Australia, New Zealand, Saudi Arabia
Taproot Health

Recruiting

The Registry of Oncology Outcomes Associated With Testing and Treatment (ROOT)

Melanoma | Sarcoma | Kidney Cancer | Adenocarcinoma | Renal Cell Carcinoma | Cervical Cancer | Acute Myeloid Leukemia | Stomach Cancer | Breast Cancer | Head and Neck Neoplasms | Nasopharyngeal Carcinoma | Glioblastoma | Astrocytoma | Multiple Myeloma | Colorectal Cancer | Pancreatic Cancer | Esophageal Cancer | Small Bowel Cancer and other conditions

United States
National Cancer Institute (NCI)

Recruiting

A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

Neurofibromatosis Type 1 | Low Grade Glioma | Visual Pathway Glioma

United States, Canada, Puerto Rico
Centre Hospitalier Universitaire Dijon

Recruiting

Study and Follow-up of Multiple Endocrine Neoplasia Type 1 (GENEM)

Multiple Endocrine Neoplasia

France
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruiting

Monogenic Kidney Stone - Genetic Testing

Rare Kidney Stone Diseases

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

Recruiting

Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)

Malignant Solid Neoplasm | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditions

United States, Puerto Rico, Australia
National Cancer Institute (NCI)

Recruiting

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

Malignant Glioma | Rhabdoid Tumor | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditions

United States, Puerto Rico, Australia, Canada
Boston Children's Hospital
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Recruiting

Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations

Autism | ASD | Macrocephaly | PTEN | PTEN Hamartoma Tumor Syndrome

United States
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Gynecologic Oncology Group
National Cancer Institute (NCI)

Not yet recruiting

Cost of Cancer Risk Management in Women at Elevated Genetic Risk for Ovarian Cancer Who Participated on GOG-0199

Fallopian Tube Carcinoma | Ovarian Carcinoma | Hereditary Breast and Ovarian Cancer Syndrome | Primary Peritoneal Carcinoma

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

Recruiting

Study of Kidney Tumors in Younger Patients

Renal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditions

United States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

Rare Kidney Stone Consortium Patient Registry (RKSC)

Cystinuria | Primary Hyperoxaluria | Dent Disease | APRT Deficiency

United States, Iceland
National Human Genome Research Institute (NHGRI)

Recruiting

Study of Chediak-Higashi Syndrome

Chediak-Higashi Syndrome

United States
National Heart, Lung, and Blood Institute (NHLBI)
Uniformed Services University of the Health Sciences

Recruiting

Study of Skin Tumors in Tuberous Sclerosis

Tuberous Sclerosis

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Study of the Disease Process of Lymphangioleiomyomatosis

Pneumothorax | Tuberous Sclerosis | Lung Disease | Lymphangioleiomyomatosis

United States
National Cancer Institute (NCI)

Active, not recruiting

Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)

Recurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Medulloblastoma | Recurrent Neuroblastoma | Recurrent Non-Hodgkin... and other conditions

United States, Puerto Rico
Alnylam Pharmaceuticals

Completed

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

Primary Hyperoxaluria | PH1 | RNAi Therapeutic | siRNA | AGT

France, United Kingdom, Netherlands, Israel, Germany
Alnylam Pharmaceuticals

Active, not recruiting

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (ILLUMINATE-C)

Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria

United States, Italy, France, Lebanon, Belgium, Netherlands, Israel, United Arab Emirates, Australia, Jordan, Turkey
Alnylam Pharmaceuticals

Active, not recruiting

A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)

Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria

United States, France, United Kingdom, Israel, Germany
National Cancer Institute (NCI)

Active, not recruiting

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

Soft Tissue Sarcoma | Renal Cell Carcinoma | Hepatocellular Carcinoma | Osteosarcoma | Ewing Sarcoma | Recurrent Renal Cell Carcinoma | Refractory Malignant Solid Neoplasm | Rhabdomyosarcoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Malignant Solid Neoplasm | Recurrent Osteosarcoma | Recurrent... and other conditions

United States
Amgen

Active, not recruiting

TEPEZZA® (Teprotumumab-trbw) Post-Marketing Requirement Study

Thyroid Eye Disease

United States, Spain, Germany, Italy, France, United Kingdom
National Cancer Institute (NCI)

Active, not recruiting

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma and other conditions

United States, Puerto Rico
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
National Cancer Institute (NCI)

Active, not recruiting

Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)

Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Histiocytic and Dendritic Cell Neoplasm | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditions

United States, Puerto Rico
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

Search clinical trials for: Hereditary study for Primary Hyperoxaluria

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