A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
A Phase 1/2, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of OCT-980 in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa
Study Overview
Status
Conditions
Detailed Description
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 in healthy volunteers, which was conducted in Australia and is now complete.
The phase 1b/2 study is an 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).
RHO-adRP participants are currently being recruited for the phase 1b/2 study in the US.
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Octant adRP Clinical Operations
- Phone Number: +1-510-500-5956
- Email: adrpclinicaltrials@octant.bio
Study Locations
-
-
Texas
-
Dallas, Texas, United States, 75231-5080
- Recruiting
- Retina Foundation of the Southwest
-
Principal Investigator:
- Mark Pennesi, MD
-
Contact:
- Charlene Nwajei
- Email: cnwajei@retinafoundation.org
-
Contact:
- Jennifer Herrera
- Email: jherrera@retinafoundation.org
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Phase 1b/2 is active. Inclusion
Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion:
- Participants must have a diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa confirmed by a genetic test that is implemented in accordance with the relevant national requirements
- Male or female greater than or equal to age 18 (inclusive) at the time of signing the informed consent form
- Have a body weight >50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m^2 (inclusive) at screening
- Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) > 55
- Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator
- Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples
Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion:
- Females who are pregnant or breastfeeding
- Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder
- Lifetime history of ocular surgery
- Any prior or current ophthalmologic gene therapy
- Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Phase 1b/2: OCT-980 Cohort 1
OCT-980 tablets administered orally once daily.
|
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
|
|
Experimental: Phase 1b/2: OCT-980 Cohort 2
OCT-980 tablets administered orally once daily.
|
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
|
|
Experimental: Phase 1b/2: OCT-980 Cohort 3
OCT-980 tablets administered orally once daily.
|
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
|
|
Experimental: Phase 1b/2: OCT-980 Cohort 4
OCT-980 tablets administered orally once daily.
|
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
|
|
Experimental: Phase 1b/2: OCT-980 Cohort 5
OCT-980 tablets administered orally once daily.
|
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
Safety and tolerability of multiple doses (Phase 1b/2) of OCT-980 in participants with a genetic diagnosis of RHO-adRP.
Time Frame: Time Frame: From Baseline to Week 60 (Ph 1b/2)
|
Time Frame: From Baseline to Week 60 (Ph 1b/2)
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- OCT-980-101
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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