Drs. Ciliberto, Nester, Reinking, and Sharathkumar received clinical trials awards recently:
- Dr. Ciliberto: “A Double-Blind, Randomized, Placebo-Controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients with Tuberous Sclerosis Complex Who Experience Inadequately-Controlled Seizures”, Proteostasis Therapeutics, Inc. for $172,000.
- Dr. Nester: “A Phase 2 Proof-of-Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients with C3 Glomerulopathy (C3G)”, Achillion Pharmaceuticals, Inc. for $171,000.
- Dr. Reinking: “A Prospective, Open-Label, Active-Controlled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Rivaroxaban for Thromboprophylaxis in Pediatric Subjects 2 to 8 Years of Age After the Fontan Procedure”, Janssen Research and Development, Inc. for $105,000.
- Dr. Sharathkumar: “A Phase I/II, Open-Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 [AAV2/6 hFactor VIII Gene Therapy] in Adult Subjects with Severe Hemophilia A”, Sangamo Therapeutics, Inc. for $15,000.
Dr. Tsalikian received some new clinical trials recently as well.
- “fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children with Growth Hormone Deficiency (GHD)”, Ascendis Pharma A/S, for $43,000.
- “A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Alogliptin Compared with Placebo in Pediatric Subjects with Type 2 Diabetes Melliltus”, Takeda Development Center Americas, Inc. for $70,000.
- “Prospective, Multicenter, Single-Arm Study to Evaluate Efficacy, Safety and Pharmacokinetics of Denosumab in Children with Osteogenesis Imperfecta”, Amgen, Inc. for $80,000.
Dr. Ahrens also received several new clinical trial awards recently:
- “A Post Approval Observational Study to Evaluate the Long-term Effectiveness and Safety of Orkambi in U.S. Patients Who Completed Study VX12-809-105 Part A”, Vertex Pharmaceuticals, Inc. for $9,200.
- “A Phase 3 Open-Label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects with Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation”, Vertex Pharmaceuticals, Inc. for $70,000.
- “A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis”, Proteostasis Therapeutics, Inc. for $145,000.
Dr. Tim Starner received a couple new clinical trials award recently:
- “A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of VX-659 in Combination with Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)”, Vertex Pharmaceuticals, Inc. for $68,000.
- “A Phase 3 Randomized, Double-Blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Theraphy in Subjects with Cystic Fibrosis Whoare Homozygous fo rthe F508del Mutation (F/F)”, Vertex Pharmaceuticals, Inc. for $58,000.
Dr. Kathy Mathews, Vice Chair for Clinical Research, received funding for several new clinical trials recently:
- “Expanded Access Protocol of Idebenone in Patients with Duchenne Muscular Dystrophy”, Santhera Pharmaceuticals (Switzerland), Ltd. for $1,500.
- “A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich’s Ataxia”, Reata Pharmaceuticals, Inc. for $224,000.
- “A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy”, Acceleron PHarma, Inc. for $327,000.
- “A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenitcal Myotonic Dystophy”, AMO Pharma, Inc. for $128,000.
- “A Randomized Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cell in Subjects with Duchenne Muscular Dystophy (HOPE-2)”, Capricor Therapeutics for $165,000.