FDA awards 11 clinical trial grants for rare diseases, many in children

National and World

(WKBN) – The U.S. Food and Drug Administration awarded grant money Thursday to 11 clinical trials involving rare diseases, many impacting children.

The $25 million funding will be dispersed over the next four years and be used to research medical conditions from skin disease to brain cancer.

Many of the studies involve children as young as newborns, including one evaluating the treatment of a rare inherited skin disease known as recessive dystrophic epidermolysis bullosa, or RDEB, a condition that can lead to painful blisters and wounds that are often disfiguring and fatal.

Another study will evaluate treatment before the onset of seizures in infants with tuberous sclerosis complex, which is an inherited disease that can affect a variety of organs and can result in long-term brain development issues.

A brain cancer study is looking at a vaccine to treat pediatric brain cancer.

Below is a complete list of the grants in alphabetical order:

  • Armgo Pharma, INC. (Ardsley, New York); Eugene Marcantonio; Phase 2 study of S48168 (ARM210) for the treatment of catecholaminergic polymorphic ventricular tachycardia type 1 (CPVT1); $1 million over two years
  • Boston Children’s Hospital (Boston, Massachusetts); Mark Puder; Phase 3 study of RELiZORB for the treatment of short bowel syndrome; $2.7 million over four years
  • Castle Creek Biosciences, LLC (Exton, Pennsylvania); Mary Spellman; Phase 3 study of FCX-007 (genetically modified autologous human dermal fibroblasts) for the treatment of recessive dystrophic epidermolysis bullosa; $1.8 million over four years
  • Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio); Darcy Krueger; Phase 2b study of sirolimus for the prevention of epilepsy in patients with tuberous sclerosis complex; $5 million over four years
  • Cincinnati Children’s Hospital Medical Center; Michael Jordan; Phase 2 study of abatacept for the treatment of common variable immunodeficiency with interstitial lung disease (ABCVILD); $3.1 million over four years
  • Duke University (Durham, North Carolina); Eric Thompson; Phase 2 study of peptide vaccine targeting CMV antigen for the treatment of newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma; $1.8 million over four years
  • Massachusetts General Hospital (Boston, Massachusetts); Amy Dickey; Phase 2 study of oral cimetidine for the treatment of protoporphyrias; $1.6 million over four years
  • Mayo Clinic Rochester (Rochester, Minnesota); Sani Kizilbash; Phase 1 study of WSD0922-FU for the treatment of high-grade astrocytoma; $1 million over three years
  • Mayo Clinic Rochester (Rochester, Minnesota); Wolfgang Singer; Phase 2 study of intrathecally administered autologous mesenchymal stem cells for the treatment of multiple system atrophy; $3.2 million over four years
  • Reveragen Biopharma, Inc.; Eric Hoffman; Phase 2a study of vamorolone for the treatment of becker muscular dystrophy; $1.2 million over two years
  • University of Florida (Gainesville, Florida); Peter Stacpoole; Phase 2A trial of dichloroacetate for the treatment of glioblastoma multiforme; $2.5 million over four years

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