The Phase 1b STOP Ototoxicity study (NCT02819856) is led by Dr. Patrick Flume, MD, professor of medicine and pediatrics at the Medical University of South Carolina in Charleston, where the study is taking place. He is an expert in pulmonary disease and cystic fibrosis.
The randomized, placebo-controlled trial is recruiting CF patients by invitation only and plans to include 100 patients with active pulmonary exacerbation receiving intravenous tobramycin. They estimate the study will be completed in December 2018.
Cystic Fibrosis Foundation Therapeutics (CFFT) has awarded a grant of $1.8 million for the trial’s development.
Due to treatment with tobramycin or amikacin (aminoglycoside) antibiotics, CF patients are susceptible to damage to several sensorineural structures within the inner ear, called ototoxicity.
Ototoxicity is a serious and common side effect of aminoglycoside treatment. The inner ear damage causes hearing loss, tinnitus, vertigo and/or dizziness. It is commonly experienced by CF patients, but often goes undiagnosed.
“We recently explored the incidence of tinnitus and vertigo in the CF population, and will present these initial findings at the next North American CF Conference,” Flume said in a press release.
There is currently no approved treatment or clear diagnostic protocols to prevent or treat ototoxicity in CF patients. Sound Parmaceuticals hopes to change this by implementing a new approach to documenting and ultimately treating aminoglycoside-induced ototoxicity.
“We are pleased to have achieved this significant developmental milestone in the CF population,” said Jonathan Kil, MD, Sound’s co-founder, CEO, and chief medical officer.
The clinical trial intends to primarily evaluate the safety and tolerability of Sound Pharmaceuticals’ investigational drug Ebselen (SPI-1005). It also aims to determine the best concentration of oral Ebselen in these patients.
Participants will be randomized to receive either placebo or Ebselen at 200 mg, 400 mg, or 600 mg twice a day for 21 days. Researchers will compare the severity of sensorineural hearing loss, speech discrimination, vertigo severity, tinnitus severity, and lung function between the placebo and Ebselen groups.