Aldeyra Therapeutics announced the first patient has enrolled in a pivotal Phase 3 clinical trial of topical dermal reproxalap for the treatment of ichthyosis (scaly, thickened, dry skin) associated with Sjögren-Larsson Syndrome (SLS).
"There is currently no FDA-approved therapy for the patients suffering from SLS, a rare inborn error of metabolism that leads to severe skin and neurological disease," commented Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "Based on the positive results from our Phase 2 clinical trial, which demonstrated consistent improvement in the dermatologic manifestations of SLS, we are pleased to enroll our first subject in the Phase 3 clinical trial as part of our ongoing commitment to develop a targeted therapy for SLS patients and their caregivers."
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In August 2016, Aldeyra announced that the results of a randomized, parallel-group, double-blind, vehicle-controlled clinical trial in SLS demonstrated clinically relevant activity in diminishing the severity of ichthyosis. As assessed by central review, five of six drug-treated subjects (83%) achieved a rating of "almost clear" or "mild" on global assessment. Six of six (100%) drug-treated subjects improved over the course of therapy as assessed by central review (p < 0.05), and the improvement was greater than that observed in vehicle-treated patients (p < 0.05). For drug-treated subjects, mean reductions in ichthyosis severity were greater after eight weeks of therapy than after four weeks of therapy, suggesting disease modifying activity.
The randomized, double-blind, multi-center, parallel-group Phase 3 clinical trial is expected to be performed in two parts: the first part of the trial will assess six months of treatment in select areas of ichthyosis over increasing proportions of body surface area; the second part of the trial is expected to assess six months of treatment in all areas of ichthyosis. Data generated from the first part of the trial will be used to confirm statistical power for the second part of the trial. The primary endpoint for the second part of the trial will be improvement in ichthyosis in drug-treated patients over six months of therapy.