Ignite Creation Date:
2026-03-26 @ 3:16 PM
Ignite Modification Date:
2026-03-30 @ 12:11 AM
Study NCT ID:
NCT07408232
Status:
RECRUITING
Last Update Posted:
2026-02-13
First Post:
2026-02-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012174', 'term': 'Retinitis Pigmentosa'}, {'id': 'C566706', 'term': 'Retinitis Pigmentosa 4'}, {'id': 'D005128', 'term': 'Eye Diseases'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D058499', 'term': 'Retinal Dystrophies'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Phase 1a: Double (Participant, Investigator); Phase 1b/2: Open Label'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2026-02-09', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-02', 'completionDateStruct': {'date': '2028-11-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-02-06', 'studyFirstSubmitDate': '2026-02-06', 'studyFirstSubmitQcDate': '2026-02-06', 'lastUpdatePostDateStruct': {'date': '2026-02-13', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-11-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety 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', 'timeFrame': 'Time Frame: From Baseline to Day 5 (Phase 1a) or Week 60 (Ph 1b/2)'}]}, 'oversightModule': {'isUsExport': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Rhodopsin-associated autosomal dominant Retinitis Pigmentosa', 'RHO-adRP', 'Retinitis Pigmentosa', 'Inherited Retinal Disease', 'autosomal dominant Retinitis Pigmentosa', 'Retinitis Pigmentosa 4', 'RP4', 'RP', 'Eye Diseases'], 'conditions': ['Retinitis Pigmentosa']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.\n\nThis study is comprised of a Phase 1a, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US.\n\nThe 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.\n\nThe 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).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion\n\nPhase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Inclusion:\n\n* Be in good general health, as determined by the Investigator\n* Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form\n* Have a body weight \\>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\\^2 (inclusive) at screening\n* Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples\n* Participants in Cohort 2b will be asked to participate in an additional food effect cohort and capable of consuming the high-fat meal within the protocol-specified time period and willing to consume 100% of the high-fat meal\n\nPhase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion:\n\n* 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\n* Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form\n* Have a body weight \\>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\\^2 (inclusive) at screening\n* Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) \\> 70\n* Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator\n* Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples Exclusion\n\nPhase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Exclusion:\n\n* Females who are pregnant or breastfeeding\n* Have evidence of any significant ocular or non-ocular disease/ disorder\n* Has lifetime history of ocular surgery\n* 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\n\nPhase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion:\n\n* Females who are pregnant or breastfeeding\n* Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder\n* Lifetime history of ocular surgery\n* Any prior or current ophthalmologic gene therapy\n* 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'}, 'identificationModule': {'nctId': 'NCT07408232', 'briefTitle': 'A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Octant, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'OCT-980-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase 1a: OCT-980', 'description': 'OCT-980 capsules administered orally once daily under fasted or fed conditions per protocol', 'interventionNames': ['Drug: Phase 1a: OCT-980 Capsule']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Phase 1a: Placebo', 'description': 'Matching placebo capsules administered orally once daily under fasted or fed conditions per protocol', 'interventionNames': ['Drug: Phase 1a: Placebo Capsule']}, {'type': 'EXPERIMENTAL', 'label': 'Phase 1b/2: OCT-980', 'description': 'OCT-980 tablets administered orally once daily.', 'interventionNames': ['Drug: Phase 1b/2: OCT-980 Tablet']}], 'interventions': [{'name': 'Phase 1a: OCT-980 Capsule', 'type': 'DRUG', 'description': 'OCT-980 capsules administered orally once at dose levels specified in the protocol', 'armGroupLabels': ['Phase 1a: OCT-980']}, {'name': 'Phase 1a: Placebo Capsule', 'type': 'DRUG', 'description': 'Placebo capsules administered orally once at dose levels specified in the protocol', 'armGroupLabels': ['Phase 1a: Placebo']}, {'name': 'Phase 1b/2: OCT-980 Tablet', 'type': 'DRUG', 'description': 'OCT-980 tablets administered orally once at dose levels specified in the protocol', 'armGroupLabels': ['Phase 1b/2: OCT-980']}]}, 'contactsLocationsModule': {'locations': [{'zip': '5000', 'city': 'Adelaide', 'state': 'South Australia', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Thomas Polasek Polasek, MD', 'role': 'CONTACT', 'email': 'cmax@cmax.com.au', 'phone': '61870887900'}, {'name': 'Jane Kelly', 'role': 'CONTACT', 'email': 'cmax@cmax.com.au', 'phone': '61870887900'}], 'facility': 'CMAX Clinical Research Pty Ltd', 'geoPoint': {'lat': -34.92866, 'lon': 138.59863}}], 'centralContacts': [{'name': 'Octant adRP Clinical Operations', 'role': 'CONTACT', 'email': 'adrpclinicaltrials@octant.bio', 'phone': '+1-510-500-5956'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Octant, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}