The FDA has cleared commencement for Retrotope’s open-label phase 2/3 clinical trial of its investigational drug RT001 to evaluate its efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD).
The US Food and Drug Administration (FDA) has cleared commencement for Retrotope’s open-label phase 2/3 clinical trial of its investigational drug RT001 to evaluate its efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD).
RT001 is an orally available deuterated polyunsaturated fatty acid (D-PUFA) that incorporates into mitochondrial and cellular membranes and stabilizes them. Lipid peroxidation has been suggested to be the primary source of cell death in degenerative diseases, such as Friedreich’s Ataxia and INAD. D-PUFAs can assist in protecting against such attacks and potentially restore cellular health.
“The phase 2/3 clinical trial is an important milestone in the development pathway of RT001 for the treatment of INAD,” shared Robert Molinari, PhD, CEO of Retrotope, in a recent statement. “We look forward to the start of the trial and expect to begin patient enrollment in the next month.”
Two patients are currently enrolled in 2 separate Expanded Access trials. (The first patient was enrolled in March 2017, and the second patient was enrolled in November 2017.) To date, both of the patients are in ongoing treatment.
“Preclinical models suggested that RT001 could reduce high levels of lipid peroxidation induced cell death in PLA2G6 deficient Drosophila and human fibroblast models,” added Molinari. “These findings have been further supported by the encouraging results we have seen in 2 [patients] currently receiving Expanded Access treatment.”
The RT001 phase 2/3 clinical trial will seek to enroll patients with INAD between 18 months and 10 years of age who are not dependent on a ventilator for mechanical respiration. (Continuous positive airway pressure (CPAP) use is acceptable).
The effectiveness of treatment with RT001 in subjects with INAD, measured via a quantitative scale of both Activities of Daily Living (ADLs), and deficits in vital functions affecting INAD children are the primary endpoints of the trial. Secondary endpoints include the effects of the treatment on standardized scores in a variety of childhood developmental milestone scales, as well as evaluations of the safety, pharmacokinetics, and tolerability of RT001.
The study centers hosting the trial anticipate enrollment in the trial to start as early as July 2018.
“We are grateful for the FDA’s clearance to start our INAD study,” furthered Peter G. Milner, MD, Retrotope’s Chief Medical Officer. “To the best of our knowledge this trial will be the first and only interventional clinical trial available to children with classic INAD. We are excited to be able to offer hope to families for a therapy that could potentially be available in their children’s lifetime.”