Retrotope has secured approval from the US Food and Drug Administration (FDA) to start a Phase ll/lll clinical trial to evaluate efficacy and safety of RT001 in patients with infantile neuroaxonal dystrophy (INAD).
The single-arm open-label trial plans to enrol around 15 INAD patients between the ages of 18 months and ten years who are not dependent on a ventilator for mechanical respiration.
The trial’s primary objective is to define the effectiveness of treatment with RT001 in subjects with INAD as measured via a quantitative scale of both Activities of Daily Living (ADLs) and deficits in vital functions affecting INAD children.
Its secondary objectives comprise how RT001 treatment affects standardised scores in various childhood developmental milestone scales, as well as evaluations of the safety, pharmacokinetics (PK), and tolerability of RT001.
Starting this month, Retrotope plans to enrol patients for the trial, which will be conducted at two medical centres in the US.
Retrotope CEO Robert Molinari said: “Preclinical models suggested that RT001 could reduce high levels of lipid peroxidation induced cell death in PLA2G6 deficient Drosophila and human fibroblast models.
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“These findings have been further supported by the encouraging results we have seen in two children currently receiving Expanded Access treatment.”
RT001 is a new category of drugs called deuterated polyunsaturated fatty acids (D-PUFAs), which are capable of protecting against free radical damage resulting in cell death that is a hallmark of several neurodegenerative diseases, including INAD.
INAD, an ultra-rare, devastating life-shortening neurodegenerative disorder, is estimated to affect only a few hundred patients across the globe.