Clinical Trials Resource Center

New Medical Therapies™

Retinitis Pigmentosa

Patient Medical Areas

September 22, 2014

QLT issued results of a phase Ib trial of QLT091001 in subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) due to inherited genetic mutations in retinal pigment epithelium (RPE65) or lecithin: retinol acyltransferase (LRAT). This multicenter, open-label retreatment trial was an extension study in which 27 LCA or RP subjects with RPE65 or LRAT mutations received up to three seven-day courses of QLT091001 to assess visual outcomes and safety following retreatment. Subjects received treatment with QLT091001 at 10mg/m2, 40mg/m2 or 60mg/m2, with the majority of subjects receiving 40mg/m2. Results showed 19 of 27 subjects (70%) had a visual field response (increase in retinal area of at least 20% from the study baseline at two consecutive visits starting within six months of any study treatment), and 19 of 27 subjects (70%) having a visual acuity response (increase of at least five letters at two consecutive visits starting within six months of any study treatment). These responses were durable, with the visual field response lasting an average of 235 days (Min-Max = 7 – 742 days), and the visual acuity response lasting an average of 232 days (Min-Max = 7 – 616 days). Overall, 10 of 13 LCA subjects (77%), and 12 of 14 RP subjects (86%) were classified as responders for either visual field retinal area or visual acuity.

June 8, 2009

Neurotech Pharmaceuticals issued positive results from two phase II trials of NT-501 for the treatment of retinitis pigmentosa (RP). Both trials were a multi-centered, randomized, double-masked, placebo controlled dose ranging design. The first study enrolled 65 subjects with later stage RP (diagnoses of RP and vision between 20/63 and 20/320). The second trial studied 67 subjects with earlier stage RP (diagnoses of RP and vision better than 20/63). The subjects received either high dose or low dose NT-501 in one eye and placebo in the other eye. Best corrected visual acuity (BCVA) and visual field sensitivity (VFS) were evaluated as primary endpoints for the later stage study and the earlier stage RP study, respectively. At 12 months no trend in visual benefit was observed in either study for these functions. However, in both studies, there was a statistically significant, dose-dependent increase in retinal thickness involving photoreceptor layers as measured by optical coherence tomography (p< 0.001 for the high dose group in each study). No treatment-related serious adverse events were reported and both NT-501 and the surgical procedure were well-tolerated.

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