Clinical TrialsInformation and insight on the latest clinical trials
- Phase 3 Trials of Rare Genetic Obesity Disorders Completes Enrollment
Rhythm Pharmaceuticals announced that it has completed enrollment of the pivotal cohorts of 10 patients in two separate, ongoing, registration-enabling Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity.
- Patient-Centric Perspective Offers Key Insights into Upcoming Mitochondrial Treatment Studies
As clinical trials gear up with the aim of attaining the first FDA-approved treatments for mitochondrial disease, a new study reports for the first time what patients and families say would motivate them for or against participating in such research trials. Mitochondrial genetic disorders refer to a group of conditions that affect the mitochondria (the structures in each cell of the body that are responsible for making energy).
- FDA Approves Combination of Venclexta and Rituxan for Lymphocytic Leukemia
The Food and Drug Administration (FDA) granted approval to venetoclax for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.
- FDA Grants Expedited Status for bluebird bio’s Gene Therapy for CALD
The FDA has granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder.
- Positive Results from Phase 3 OLYMPUS Trial for UTUC
UroGen Pharma presented positive data from an interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel), an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).
- Phase 3 Trial Initiated for Progressive Familial Intrahepatic Cholestasis
Albireo Pharma announced that the first patient has been enrolled, a Phase 3 clinical trial (called PEDFIC-1) for patients with progressive familial intrahepatic cholestasis (PFIC)
- Trial Shows Adding Chemotherapy Helps Kids Live With Pulmonary Vein Stenosis
Pulmonary vein stenosis (PVS) is a rare disease in which abnormal cells build up inside the veins responsible for carrying oxygen-rich blood from the lungs to the heart.
- FDA Votes in Favor of Familial Chylomicronemia Syndrome Treatment
An FDA advisory committee voted to recommend the approval of volanesorsen (Waylivra), an RNAi drug for rare lipid disorder familial chylomicronemia syndrome (FCS).
- Overview of Savara Pharmaceutical’s Clinical Trial Programs
Rob Neville of Savara Pharmaceuticals provides an overview of his company’s Molgradex (for PAP and NTM lung infection) and AeroVanc (an inhaled formulation of vancomycin, for the treatment of persistent methicillin-resistant Staphylococcus aureus, or MRSA, lung infection in cystic fibrosis)
- FDA Approves Combination Therapy for Melanoma Mutation
The U.S. Food and Drug Administration (FDA) approved the combination of dabrafenib (Tafinlar) and trametinib (Mekinist) for the adjuvant treatment of patients with resected, stage III melanoma with BRAFV600 mutations. The combination is already approve …