- Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine
- Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
- Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin
- Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
- Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin)
- Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
- Evaluation of TYR Sphere
- Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)
- Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
- Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
- Taste and Palatability of Orfadin Suspension
Clinical Trials
- A Genetic Analysis of Usher Syndrome in Ashkenazi Jews
- Adaptive Optics Imaging of Outer Retinal Diseases
- Auditory Neural Function in Implanted Patients With Usher Syndrome
- Clinical and Genetic Examination of Usher Syndrome Patients’ Cohort in Europe
- Clinical and Genetic Testing of Patients With Usher Syndrome
- Inherited Retinal Degenerative Disease Registry
- Natural History and Genetic Studies of Usher Syndrome
- Natural History Study of Usher Syndrome ( Light4Deaf )
- Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.
- Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa
- Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
- Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome
- 5 Year Registry Study to Track Clinical Application of DecisionDx-UM Assay Results and Associated Patient Outcomes
- A Phase I Study of LXS196 in Patients With Metastatic Uveal Melanoma.
- A Phase Ib/II Study of AEB071 and MEK162 in Adult Patients With Metastatic Uveal Melanoma
- A Phase II Study of BVD-523 in Metastatic Uveal Melanoma
- A Prospective Natural History Study in Uveal Melanoma
- A Safety and Tolerability Study of INCAGN02390 in Select Advanced Malignancies
- A Study of APG-115 in Combination With Pembrolizumab in Patients With Metastatic Melanomas or Advanced Solid Tumors
- A Study of Concurrent Stereotactic Body Radiotherapy With Ipi and Nivo in Metastatic Uveal Melanoma
- A Study of PLX2853 in Advanced Malignancies.
- A Study of RO7293583 in Participants With Unresectable Metastatic Tyrosinase Related Protein 1 (TYRP1)-Positive Melanomas
- A Study of Sorafenib in Patients With Chemonaive Metastatic Uveal Melanoma
- A Study of TBio-4101 (TIL) and Pembrolizumab in Patients With Advanced Solid Tumors
- A Study of the Intra-Patient Escalation Dosing Regimen With IMCgp100 in Patients With Advanced Uveal Melanoma
- A Trial of Niraparib in BAP1 and Other DNA Damage Response (DDR) Deficient Neoplasms (UF-STO-ETI-001)
- Adjuvant Melatonin for Uveal Melanoma
- Assessing the Clinical Effectiveness of Serum Biomarkers in the Diagnosis of Metastatic Uveal Melanoma
- C7R-GD2.CART Cells for Patients With Relapsed or Refractory Neuroblastoma and Other GD2 Positive Cancers (GAIL-N)
- Cabozantinib-S-Malate Compared With Temozolomide or Dacarbazine in Treating Patients With Metastatic Melanoma of the Eye That Cannot Be Removed by Surgery
- CAVATAK® and Ipilimumab in Uveal Melanoma Metastatic to the Liver (VLA-024 CLEVER)
- Combined PET/CT Imaging for the Early Detection of Ocular Melanoma Metastasis Compared to CT Scanning Alone
- Comparison Between Fotemustin to Intensive Surveillance in Patients With High Risk Uveal Melanoma
- Crizotinib in High-Risk Uveal Melanoma Following Definitive Therapy
- Defactinib (VS-6063) Combined With VS-6766 in Patients With Metastatic Uveal Melanoma
- Dendritic Cells Plus Autologous Tumor RNA in Uveal Melanoma
- Dexamethasone Intravitreal Implant for Treatment of Macular Edema After Plaque Radiotherapy of Uveal Melanoma
- Durvalumab (MEDI4736) Plus Cediranib in Patients With Metastatic Uveal Melanoma
- Early Integration of Supportive Care Into Standard Oncology Care for Metastatic Uveal Melanoma Patients
- Efficacy of Spartalizumab Across Multiple Cancer-types in Patients With PD1-high mRNA Expressing Tumors
- Endoresection of the Tumor Scar or Transpupillary Thermotherapy for the Treatment of Large Uveal Melanomas (Endoresection-Laser)
- Epacadostat and Vaccine Therapy in Treating Patients With Stage III-IV Melanoma
- Evaluation Interest of the Circulating Tumor DNA Dosage in Patient With Hepatic Metastatic Uveal Melanoma Candidate to Complete Resection (ct DNA R0)
- Follow-up of Patients With Uveal Melanoma Adapted to the Risk of Relapse (SALOME)
- Frequency and Clinical Phenotype of BAP1 Hereditary Predisposition Syndrome
- Genetic Predictors of Efficiency and Safety of ICIs in Patients With Different Malignancies (ICIPRESIST-0519)
- Glembatumumab Vedotin in Treating Patients With Metastatic or Locally Recurrent Uveal Melanoma
- Halt Growth of Liver Tumors From Uveal Melanoma With Closure of Liver Artery Following Injection of GM-CSF
- Hypofractionated Stereotactic Linear Accelerator Radiotherapy of Uveal Melanoma
- Influence of Oral Treatment With Citicoline for the Prevention of Radiation Optic Neuropathy in Patients Treated for Uveal Melanomas With Proton Beam Therapy
- Intermittent Selumetinib for Uveal Melanoma
- Intravitreal Ranibizumab for the Prevention of Radiation Maculopathy Following Plaque Radiotherapy
- Isolated Hepatic Perfusion in Combination With Ipilimumab and Nivolumab in Patients With Uveal Melanoma Metastases
- Managed Access Program Supporting Patient Access to Tebentafusp
- Melanoma Vaccine Against Neoantigen and Shared Antigens by CD40 Activation and TLR Agonists In Patients With Melanoma (Including Ocular Melanoma)
- Messenger Ribonucleic Acid (mRNA) Transfected Dendritic Cell Vaccination in High Risk Uveal Melanoma Patients
- New Biopsy Technique for Uveal Melanoma
- Olaparib in Combination With Pembrolizumab for Advanced Uveal Melanoma
- Olaparib in Unresectable/Metastatic Melanoma With BRCA1/2
- Pembrolizumab in Treating Patients With Advanced Uveal Melanoma
- Ph 1 Study in Subjects With Tumors Requiring Arginine to Assess ADI-PEG 20 With Pemetrexed and Cisplatin
- Phase 2 Trial of AU-011 Via Suprachoroidal Administration With a Dose Escalation and Randomized, Masked Dose Expansion Designed to Evaluate Safety and Efficacy of AU-011 in Subjects With Primary Indeterminate Lesions and Small Choroidal Melanoma
- Pilot Trial of Autologous Tumor Infiltrating Lymphocytes (LN-144) for Patients With Metastatic Uveal Melanoma
- Proof of Concept of TBio-4101, Lymphodepleting Chemo, IL-2 for Relapsed/Refractory Melanoma
- Prospective Registration Of Patient Data and Quality of Life in Eye Melanoma Patients
- RAD001 (Everolimus) and Pasireotide (SOM230) LAR in Patients With Advanced Uveal Melanoma
- Radiation Therapy in Preventing Liver Metastases in Patients With Uveal Melanoma Who HaveMonosomy 3 or DecisionDx Class 2 Disease and Are More Likely to Develop Liver Metastases
- Retrospective Register for Uveal Melanoma
- Safety & Activity of Controllable PRAME-TCR Therapy in Previously Treated AML/MDS or Metastatic Uveal Melanoma
- Safety and Efficacy of AEB071 in Metastatic Uveal Melanoma Patients
- Safety and Efficacy of IMCgp100 Versus Investigator Choice in Advanced Uveal Melanoma
- Sargramostim, Vaccine Therapy, or Sargramostim and Vaccine Therapy in Preventing Disease Recurrence in Patients With Melanoma That Has Been Removed By Surgery
- Selumetinib (AZD6244: ARRY-142886) (Hyd-Sulfate) in Metastatic Uveal Melanoma (SUMIT)
- Single Arm Trial of Tumor-Treating Fields in Combination With Nivolumab and Ipilimumab in Metastatic Uveal Melanoma
- SIR-Spheres® 90Y Microspheres Treatment of Uveal Melanoma Metastasized to Liver
- Sitravatinib and Tislelizumab in Patients With Metastatic Uveal Melanoma With Liver Metastases.
- Stereotactic Body Radiation Therapy and Aflibercept in Treating Patients With Uveal Melanoma
- Study Evaluating Single and Repeated Intravitreal Doses of ICON-1 in Patients With Uveal Melanoma
- Study in Subjects With Small Primary Choroidal Melanoma
- Study Multicentre Evaluating the Effectiveness and Toxicity Sorafenib (Nexavar®) in Adult Patients With Uveal Melanoma and Metastatic Dissemination
- Study of AntiCTLA4 in Patients With Unresectable or Metastatic Uveal Melanoma
- Study of Immunotherapy Plus ADI-PEG 20 for the Treatment of Advanced Uveal Melanoma
- Study of PAC-1 and Entrectinib for Patients With Metastatic Uveal Melanoma
- Study of Safety and Tolerability of BCA101 Alone and in Combination With Pembrolizumab in Patients With EGFR-driven Advanced Solid Tumors
- Study of the Activity of PD-1 Inhibitors in Metastatic Uveal Melanoma
- Study to Evaluate the Safety of IMM-01 in Patients With Advanced Solid Tumours
- Targeted Alpha Particle Radiotherapy for Metastatic Uveal Melanoma
- Temozolomide or Selumetinib in Treating Patients With Metastatic Melanoma of the Eye
- The Role of Genetic Mutations and of Circulating mRNAs in Uveal Melanoma
- Tilting of Radioactive Plaques After Initial Accurate Placement for Treatment of Uveal Melanoma
- Trametinib With or Without GSK2141795 in Treating Patients With Metastatic Uveal Melanoma
- Transarterial Radioembolisation in Comparison to Transarterial Chemoembolisation in Uveal Melanoma Liver Metastasis
- Treatment Of Radiation Retinopathy Trial
- Treatment With Intravitreal Avastin for Large Uveal Melanomas
- Trial of AEB071 in Combination With BYL719 in Patients With Melanoma
- Trial of Nivolumab in Combination With Ipilimumab in Subjects With Previously Untreated Metastatic Uveal Melanoma
- TTT Versus TTT and Triamcinolone to Decrease Exudation in Choroidal Melanoma After Proton Beam Therapy
- Uveal Melanoma and Brachytheraphy: Long-term Outcomes.
- Vascular Response to Brachytherapy Using Functional OCT
- Vorinostat in Patients With Class 2 High Risk Uveal Melanoma
- Vorinostat in Treating Patients With Metastatic Melanoma of the Eye
- Vorinostat in Treating Patients With Metastatic or Unresectable Melanoma
- Yttrium90, Ipilimumab, & Nivolumab for Uveal Melanoma With Liver Metastases
- Examining the Role of Transrectal High Intensity Focused Ultrasound (HIFU) in Rectal Pelvic Cancer
- Gynecologic Cancer Lymphedema Questionnaire as a Clinical Care Tool to Identify Lower Extremity Lymphedema
- Immediate Vaginal Reconstruction After Oncologic Resection: Surgical Outcomes, Patient Satisfaction and Sexual Function
- Immune Persistence After the Whole Vaccination Shcedule With Recombination Quadrivalent HPV Vaccine
- Immunogenicity and Safety of Quadrivalent HPV Vaccine in Healthy Chinese Female Subjects Aged 9 to 19 Years
- Immunotherapy Using Tumor Infiltrating Lymphocytes for Patients With Metastatic Human Papillomavirus-Associated Cancers
- Impact of a Sexological Follow-up on the Sexual Function in Patients With Cervix or Vaginal Cancer Treated by Radiotherapy and Brachytherapy
- Integrated Cancer Repository for Cancer Research
- Internet-based System for Cancer Patients to Self-report Toxicity
- Lymphoscintigraphy in Patients With Vaginal Cancer
- Magnetic Resonance Imaging of Gynecologic Malignancies Involving the Vagina
- Paclitaxel in Treating Patients With Advanced, Refractory, or Recurrent Cervical or Vaginal Cancer
- Pelvic Drains After Radical Hysterectomy in Treating Patients With Uterine, Cervical, or Vaginal Cancer
- Pelvic Fractures and Radiation Therapy for Cervical Cancer
- Peripheral Stem Cell Transplant, White Blood Cell Infusions, Chemotherapy, and Radiation Therapy in Treating Patients With Recurrent Metastatic Cervical or Vaginal Cancer
- Phone or Email Reminder in Increasing Vaginal Dilator Use in Patients With Gynecologic Cancers Undergoing Brachytherapy
- Primary Organoid Models and Combined Nucleic Acids Therapeutics for Anti-HPV Treatments
- Sexual Rehabilitation Programme After Radiotherapy for Gynaecological Cancer
- Study of Psychosexual Impact of Cancer-Related Infertility in Women: Third Party Reproductive Assistance
- T Cell Receptor Immunotherapy Targeting HPV-16 E6 for HPV-Associated Cancers
- Triapine, Cisplatin, and Radiation Therapy in Treating Patients With Cervical Cancer or Vaginal Cancer
- Vinorelbine and Paclitaxel Plus Radiation Therapy in Treating Patients With Advanced Cancer Arising in the Pelvis
- Reactivation of Varicella Zoster Virus and Risk of Vascular Disease
- Safety Study of NBP608 in Healthy Adult Volunteers
- Safety, Tolerability, and Immunogenicity of CRV-101 in Healthy Adult Subjects
- Treatment of Chronic Postherpetic Pain With Autologous Fat Grafting – A RCT
- Tzanck Smear With Methylene Blue Stain for Herpes
- Zostavax in Rheumatoid Arthritis
- A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
- Acute Porphyria Biomarkers for Disease Activity
- Clinical Diagnosis of Acute Porphyria
- ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
- ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
- Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
- Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
- EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
- INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
- Panhematin for Prevention of Acute Attacks of Porphyria
- Prevalence of Acute Hepatic Porphyria
- A Phase I Study of Dexmedetomidine Bolus and Infusion in Corrective Infant Cardiac Surgery: Safety and Pharmacokinetics
- A Study Evaluating the Efficacy and Safety of IV L-Citrulline for the Prevention of Clinical Sequelae of Acute Lung Injury Induced by Cardiopulmonary Bypass in Pediatric Patients Undergoing Surgery for Congenital Heart Defects
- A Trial to Evaluate the Safety and Efficacy of a Fully Degradable Ventricular Septal Defect (VSD) Closure
- Aberrations in Carnitine Homeostasis in Congenital Heart Disease With Increased Pulmonary Blood Flow
- Cardiac Function After Transcatheter VSD Closure
- Cardiopulmonary Function in Adults Born With a Ventricular Septal Defect
- Closure of Muscular Ventricular Septal Defects (VSDs) With the AMPLATZER Muscular VSD (MuVSD) Occluder – Post Approval Study
- Closure of Perimembranous Ventricular Septal Defects With The AMPLATZER® Membranous VSD OCCLUDER
- Evaluation of Latent Pulmonary Arterial Hypertension in Congenital Shunt Lesions
- Follow-up Study to Monitor the Efficacy and Safety of the Occlutech® mVSD Cases
- Hybrid Closure of Congenital Heart Disease
- Improving Care for Children With Congenital Heart Disease.
- Intravenous L-Citrulline to Treat Children Undergoing Heart Bypass Surgery : Revised Protocol
- Lifetech MFO Post-Market Clinical Follow-Up Study
- Longterm Outcome After Ventricular Septal Defect Closure
- Measures to Lower the Stress Response in Pediatric Cardiac Surgery
- Optical Tissue Identification for Myocardial Architecture
- Optimal Timing for Repair of Left to Right Shunt Lesions
- Regional Anesthesia Following Pediatric Cardiac Surgery
- The Cardiopulmonary Effect of Inhaled Beta-2-agonists on Adult Patients Born With Ventricular Septum Defects.
- The Effect of Remote Ischemic Preconditioning on the Ischemic Reperfusion Injury in Infants With Ventricular Septal Defect and Pulmonary Hypertension
- The Effect of Transcatheter Ventricular Septal Defect Closure on Heart Rate Variability Parameters
- The Effect of Transcatheter VSD Closure on Children’s Appetite, Hormones and Growth
- Transcatheter Closure Versus Surgery of Perimembranous Ventricular Septal Defects
- Use of Ketamine Prior to Cardiopulmonary Bypass in Children
- 177Lu-DOTATOC for the Treatment of Patients With Somatostatin Receptor Positive NETs
- DOTATOC PET/CT for Imaging NET Patients
- Everolimus and Octreotide Acetate With or Without Bevacizumab in Treating Patients With Locally Advanced or Metastatic Pancreatic Neuroendocrine Tumors That Cannot Be Removed by Surgery
- Laser Tissue Welding – Distal Pancreatectomy Sealing Study