Dr. Nester’s research is in the area of complement mediated kidney disease. One such disease, C3 glomerulopathy, can be particularly devastating for affected children. Because no disease-directed treatments exist 50% of affected children progress to end stage renal disease within 10 years of diagnosis and up to 90% recur when a transplant kidney is placed.
Stead Family Children’s Hospital (under the guidance of Dr. Nester and her research team, Meredith Wisniewski and Monica Hall) will be the single site in North America that will offer access to a novel, first in class anti-complement agent (phase 2 trial) – that has the potential to facilitate a complete remission of complement-mediated renal disease both in the native kidney and in the setting of transplant recurrence.
“The major point is that there are A LOT of high powered glomerular disease centers out there (both adult and peds) – yet the pharmaceutical company deliberately sought us out to run this trial. The ability to prove that we can save a renal transplant (if we can) is HUGE!”, says Dr. Nester.
Background and further information can be found in Dr. Nester’s paper in Nature Reviews, “C3 glomerulopathy – understanding a rare complement-driven renal disease“.
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.