Sunday, October 20, 2019

Sunday, October 20, 2019

FDA awards $15 million grant to 12 new clinical trials for rare-disease treatments


Over the next four years, the U.S. Food and Drug Administration (FDA) will be providing more than $15 million to 12 new clinical trials. The grants will help enhance the development of medical products for patients with rare diseases – mainly those affecting children.

The grants have been awarded to the following recipients with the named principal investigators, and approximate funding amounts:

  • Chemocentryx, Inc. (Mountain View, California), Peter Staehr, phase 2 study of avacopan for the treatment of complement 3 glomerulopathy – $1 million over two years

  • Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio), Maryam Fouladi, phase 1 study of PTC596 for the treatment of diffuse intrinsic pontine glioma & high-grade gliomas – $750,000 over three years

  • Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio), Parinda Mehta, phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia – $1.7 million over four years

  • Columbia University Health Sciences (New York, New York), Gary Brittenham, phase 2 study of daily vitamin D for the treatment of sickle-cell respiratory complications – $2 million over four years

  • Cumberland Pharmaceuticals, Inc. (Nashville, Tennessee), Ines Macias-Perez, phase 2 study for oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy – $1 million over three years

  • Massachusetts General Hospital (Boston, Massachusetts), Sara Pai, phase 2 study of anti-PD1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis – $1 million over three years

  • New York Medical College (Valhalla, New York), Mitchell Cairo, phase 2 study of viral-specific cytotoxic T-lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency – $1.7 million over four years

  • Privo Technologies, LLC (Peabody, Massachusetts), Manijeh Goldberg, phase 1/2 study of cisplatin patch (PRV111) for the treatment of oral cancer – $2 million over four years

  • Targeted Therapy Technologies, LLC (Somerset, New Jersey), Ricardo Carvalho, phase 1 study of episcleral topotecan for the treatment of retinoblastoma – $660,000 over three years

  • University of Alabama at Birmingham (Birmingham, Alabama), Gregory Friedman, phase 1 study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors – $750,000 over three years

  • University of California at San Diego (La Jolla, California), Jason Sicklick, phase 2 study of temozolomide for the treatment of gastrointestinal stromal tumor – $1.5 million over three years

  • University of Texas MD Anderson Cancer Center (Houston, Texas), Michael Andreeff, phase 1/2 study of the imipridone (ONC201) for treatment of acute myeloid leukemia – $1 million over four years
 The grants have been awarded by FDA through the Orphan Products Clinical Trials Grants Program.

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U.S. Food and Drug Administration (FDA)

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