This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.
Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision. This study is comprised of a Phase 1a, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US. The phase 1a study will be a single dose, double-masked, placebo-controlled ascending dose escalation (SAD +/- food effect) to evaluate the safety, tolerability, and PK of OCT-980 in healthy volunteers. The phase 1b/2 study will be open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
50
OCT-980 capsules administered orally once at dose levels specified in the protocol
Placebo capsules administered orally once at dose levels specified in the protocol
OCT-980 tablets administered orally once at dose levels specified in the protocol
CMAX Clinical Research Pty Ltd
Adelaide, South Australia, Australia
RECRUITINGSafety and tolerability of single (Phase 1a) or multiple doses (Phase 1b/2) of OCT-980 in volunteers or participants with a genetic diagnosis of RHO-adRP, respectively
Time frame: Time Frame: From Baseline to Day 5 (Phase 1a) or Week 60 (Ph 1b/2)
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