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.
The Department received a generous gift of $4.5 million from the Jacobson Foundation based out of Des Moines. This gift from the Jacobson Foundation will create an endowed chair for the Division of Medical Genetics and Genomics, as well as an additional Chair to be named, for $2 million apiece. The gift will also provide $500,000 to support training and recruitment of needed pediatric specialists for the state of Iowa.
Drs. Norris and Uc published a new article in EBioMedicine titled “A Novel Stomach-Pancreas Connection: More Than Physical”.EBioMedicine. 2018 Oct 9. pii: S2352-3964(18)30422-5. doi: 10.1016/j.ebiom.2018.10.011. [Epub ahead of print]
Dr. Tom Scholz, Director of the Division of Child and Community Health and Director of Community Relations for the Department of Pediatrrics, received the “Friend of Pediatrics” Award from Medical Associates Pediatrics in Dubuque, IA. This award is presented to someone external to the group in Dubuqe that helps makes a difference to the group and to the patients they serve in the Dubuque area.