Ignite Creation Date:
2025-12-24 @ 1:36 PM
Ignite Modification Date:
2025-12-27 @ 10:32 PM
Study NCT ID:
NCT05882695
Status:
COMPLETED
Last Update Posted:
2025-06-29
First Post:
2023-05-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of SPG302 in Healthy Volunteers and ALS Participants
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Double blinded'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV and a repeat dose expansion in ALS cohort(s)'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 88}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-07-03', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2025-06-27', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-06-27', 'studyFirstSubmitDate': '2023-05-22', 'studyFirstSubmitQcDate': '2023-05-22', 'lastUpdatePostDateStruct': {'date': '2025-06-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-05-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-06-27', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Effect of repeated dosing of SPG302 on electroencephalogram in healthy volunteers (MAD cohort)', 'timeFrame': '12 mon', 'description': 'Change from baseline in EEG parameters'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'Number of respiratory complications'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'Spirometry'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'TMS'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'EEG'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'Change in Nocturnal Pulse Oximetry'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12mon', 'description': 'Edinburgh Cognitive and Behavioural ALS Screen (ECAS)'}, {'measure': 'Effect of SPG302 on proteins and biomarkers in participants with ALS', 'timeFrame': '12mon', 'description': 'Multiple protein and immunological biomarkers'}, {'measure': 'The effect of SPG302 on protein(s) and biomarkers', 'timeFrame': '12mon', 'description': 'Change from baseline in the analysis of Columbia-Suicide Severity Rating Scale (C-SSRS)'}], 'primaryOutcomes': [{'measure': 'Safety and tolerability in healthy volunteers (SAD cohort)', 'timeFrame': '7 days', 'description': '• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)'}, {'measure': 'Safety and tolerability in healthy volunteers (SAD food effect cohort)', 'timeFrame': '15 days', 'description': '• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)'}, {'measure': 'Safety and tolerability in healthy volunteers (MAD cohort)', 'timeFrame': '12 days', 'description': '• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)'}, {'measure': 'Safety and tolerability in participants with ALS', 'timeFrame': '60 days', 'description': '• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)'}], 'secondaryOutcomes': [{'measure': 'Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)', 'timeFrame': '7 days', 'description': 'PK parameters of SPG302 on concentrations in plasma'}, {'measure': 'Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)', 'timeFrame': '15 days', 'description': 'Effects of food on SPG302 PK profile'}, {'measure': 'Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)', 'timeFrame': '12 days', 'description': 'PK parameters of SPG302 on concentrations in plasma'}, {'measure': 'Plasma pharmacokinetics of SPG302 in participants with ALS', 'timeFrame': '12mon', 'description': 'PK parameters of SPG302 on concentrations in plasma'}, {'measure': 'Clinical outcomes of multiple oral doses of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'Spirometry'}, {'measure': 'Clinical efficacy measures of SPG302 in participants with ALS', 'timeFrame': '12 mon', 'description': 'The Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R).'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Amyotrophic Lateral Sclerosis', 'regenerative', 'synapse'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'The first-in-human Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPG302 in healthy volunteers and ALS participants', 'detailedDescription': 'This study is a Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV with food effect cohort, and a repeat dose expansion cohort(s) in participants with ALS.\n\nThe study consists of 3 parts, as follows:\n\n* Part 1: SAD in HV with up to 6 cohorts including a food effect cohort.\n* Part 2: MAD over 5 days in HV with up to 5 cohorts\n* Part 3: ALS cohorts with once daily (QD) dosing over 28 day cycles'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '55 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 18-55\n* Must be in good health with no significant medical history\n* Clinical laboratory values within normal range or \\< 1.2 times ULN\n* BMI 18-32 (inclusive)\n* Contraceptive use by men or women consistent with local regulations\n* Able and willing to provide written informed consent\n\nExclusion Criteria:\n\n* Any physical or psychological condition that prohibits study completion\n* Known cardiac disease\n* Active or history of malignancy in the past 5 years\n* Serious infection within 1 month of screening\n* Acute illness within 30 days of Day 1\n* Surgery, bone fracture, or major musculoskeletal injury in the past 3 months\n* History of suicidal behavior or suicidal ideation\n* Active cigarette smokers and users of nicotine-containing products\n* HIV, hepatitis B and hepatitis C positive\n* SBP \\>140 or \\<90\n* DBP \\>90 or \\<40\n* HR \\<40 or \\>100\n* QTcF \\>450ms, cardiac arrhythmia, or clinically significant abnormal ECG\n* Prescriptions, over-the-counter, or herbal medication within 7 days\n* Vaccines within 14 days\n* Other investigational products within 30 days\n* Blood donation within 30 days\n* Plasma donation within 7 days\n* Pregnant or breastfeeding\n* Otherwise unfit, on metabolic-altering lifestyle/diet, positive urine drug screen or intake of alcohol or caffeine-containing products\n\nALS Cohort Inclusion Criteria:\n\n* Age 18-80\n* ALS TRICALS risk score\n* Stable dose of standard of care treatment\n* Contraception use by men or women consistent with local regulations\n* Able and willing to provide written informed consent\n\nALS Cohort Exclusion Criteria:\n\n* Underlying physical or psychological condition prohibiting study completion\n* Known cardiac disease\n* Active or history of malignancy in the past 5 years\n* Serious infection within 1 month of screening\n* Acute illness within 30 days of Day 1\n* History of suicidal behavior or suicidal ideation\n* Active cigarette smokers and users of nicotine-containing products\n* Neurodegenerative disease\n* External respiratory support or supplemental oxygen requirement\n* HIV, hepatitis B and hepatitis C positive\n* SBP \\>140 or \\<90\n* DBP \\>90 or \\<40\n* HR \\<40 or \\>100\n* QTcF \\>450ms, cardiac arrhythmia, or clinically significant abnormal ECG\n* Vaccines within 14 days\n* Other investigational products within 30 days\n* Blood donation within 30 days\n* Plasma donation within 7 days\n* Pregnant or breastfeeding\n* Otherwise unfit'}, 'identificationModule': {'nctId': 'NCT05882695', 'briefTitle': 'Study of SPG302 in Healthy Volunteers and ALS Participants', 'organization': {'class': 'INDUSTRY', 'fullName': 'Spinogenix'}, 'officialTitle': 'A Phase 1/2a, Randomized, Double Blind, Placebo Controlled, Single and Multiple Dose Escalation Study in Healthy Volunteers and an Expansion Cohort in Adult Participants With Amyotrophic Lateral Sclerosis (ALS) to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPG302', 'orgStudyIdInfo': {'id': 'SPG302-ALS-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD)', 'description': '8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule', 'interventionNames': ['Drug: SPG302']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD)', 'description': '8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD)', 'description': '8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).', 'interventionNames': ['Drug: SPG302']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD)', 'description': '8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental Part 3: Active SPG302 to be administered to participants with ALS', 'description': 'Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.', 'interventionNames': ['Drug: SPG302']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS', 'description': 'Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS', 'description': 'Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days for up to 3 cycles in the USA and up to 12 cycles in Australia. A follow-up safety visit will be conducted 30 days after last dose (±7 days).', 'interventionNames': ['Drug: SPG302']}], 'interventions': [{'name': 'SPG302', 'type': 'DRUG', 'description': 'synthetic small molecule', 'armGroupLabels': ['Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD)', 'Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD)', 'Experimental Part 3: Active SPG302 to be administered to participants with ALS', 'Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD)', 'Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD)', 'Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2109', 'city': 'North Ryde', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Macquarie University', 'geoPoint': {'lat': -33.79677, 'lon': 151.12436}}, {'zip': '4029', 'city': 'Herston', 'state': 'Queensland', 'country': 'Australia', 'facility': "Royal Brisbane and Women's Hospital", 'geoPoint': {'lat': -27.44453, 'lon': 153.01852}}, {'zip': '5042', 'city': 'Adelaide', 'state': 'South Australia', 'country': 'Australia', 'facility': 'Flinders Medical center', 'geoPoint': {'lat': -34.92866, 'lon': 138.59863}}, {'zip': '3004', 'city': 'Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Nucleus Melbourne (healthy volunteers)', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}], 'overallOfficials': [{'name': 'Ofer M Gonen, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Nucleus Network (for healthy volunteers)'}, {'name': 'David Schultz (ALS site), MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Finders Medical Center (ALS)'}, {'name': 'Robert Henderson (ALS site), MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Royal Brisbane Hospital (ALS)'}, {'name': 'Dominic Rowe, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Macquarie Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Spinogenix', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Novotech (Australia) Pty Limited', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}