Ignite Creation Date:
2025-12-24 @ 5:45 PM
Ignite Modification Date:
2025-12-27 @ 12:35 PM
Study NCT ID:
NCT04993768
Status:
UNKNOWN
Last Update Posted:
2023-07-13
First Post:
2021-07-19
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013494', 'term': 'Supranuclear Palsy, Progressive'}], 'ancestors': [{'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D009886', 'term': 'Ophthalmoplegia'}, {'id': 'D015835', 'term': 'Ocular Motility Disorders'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D024801', 'term': 'Tauopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D010243', 'term': 'Paralysis'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-10-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-07', 'completionDateStruct': {'date': '2023-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-07-12', 'studyFirstSubmitDate': '2021-07-19', 'studyFirstSubmitQcDate': '2021-07-29', 'lastUpdatePostDateStruct': {'date': '2023-07-13', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-08-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)', 'timeFrame': '48 weeks', 'description': 'Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) associated with TPN-101 v. placebo administered for up to 48 weeks in patients with PSP'}], 'secondaryOutcomes': [{'measure': 'Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)', 'timeFrame': '48 weeks'}, {'measure': 'Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL)', 'timeFrame': '48 weeks'}, {'measure': 'Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)', 'timeFrame': '48 weeks', 'description': 'The PSPRS is comprised of 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline. Scores range from 0 to 100, each item is graded 0-2 (six items) or 0-4 (22 items), with lower scores indicating better clinical and functional status.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Progressive Supranuclear Palsy']}, 'descriptionModule': {'briefSummary': 'This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.', 'detailedDescription': 'This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 4-arm study with an open-label treatment phase in patients with PSP. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open label Treatment Period, and a Follow-up Visit 4 weeks post treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '86 Years', 'minimumAge': '41 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Clinical diagnosis of probable progressive supranuclear palsy (PSP)\n2. Presence of PSP symptoms for less than 5 years\n3. Has a reliable caregiver/informant to accompany the patient to all study visits.\n4. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening\n5. Patient must reside outside a skilled nursing facility or dementia care facility at the time of Screening, and admission to such a facility must not be planned. Residence in an assisted living facility is allowed\n\nExclusion Criteria:\n\nPatients must not meet any of the following criteria:\n\n1. Presence of other significant neurological or psychiatric disorders\n2. History of clinically significant brain abnormality\n3. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa\n4. Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean\n5. Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or any other frontotemporal lobar degeneration causative genes not associated with underlying tau pathology\n6. History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease'}, 'identificationModule': {'nctId': 'NCT04993768', 'briefTitle': 'A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Transposon Therapeutics, Inc.'}, 'officialTitle': 'A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)', 'orgStudyIdInfo': {'id': 'TPN-101-PSP-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'TPN-101, Dose A', 'interventionNames': ['Drug: TPN-101, 100 mg/day']}, {'type': 'EXPERIMENTAL', 'label': 'TPN-101, Dose B', 'interventionNames': ['Drug: TPN-101, 200 mg/day']}, {'type': 'EXPERIMENTAL', 'label': 'TPN-101, Dose C', 'interventionNames': ['Drug: TPN-101, 400 mg/day']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'TPN-101, 100 mg/day', 'type': 'DRUG', 'description': '100 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).', 'armGroupLabels': ['TPN-101, Dose A']}, {'name': 'TPN-101, 200 mg/day', 'type': 'DRUG', 'description': '200 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).', 'armGroupLabels': ['TPN-101, Dose B']}, {'name': 'TPN-101, 400 mg/day', 'type': 'DRUG', 'description': '400 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).', 'armGroupLabels': ['TPN-101, Dose C']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85013', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': "St. Joseph's Hospital and Medical Center, Barrow Neurological Institute", 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '92037', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'UC San Diego Altman Clinical And Translational Research Institute', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'UCSF Neurosciences Clinical Research Unit (NCRU)', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80113', 'city': 'Englewood', 'state': 'Colorado', 'country': 'United States', 'facility': 'Rocky Mountain Movement Disorders Center', 'geoPoint': {'lat': 39.64777, 'lon': -104.98776}}, {'zip': '33486', 'city': 'Boca Raton', 'state': 'Florida', 'country': 'United States', 'facility': "Parkinson's Disease and Movement Disorders Center of Boca Raton", 'geoPoint': {'lat': 26.35869, 'lon': -80.0831}}, {'zip': '32608', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'UFHealth Fixel Institute for Neurological Diseases', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University School of Medicine', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48334', 'city': 'Farmington Hills', 'state': 'Michigan', 'country': 'United States', 'facility': 'Quest Research Institute', 'geoPoint': {'lat': 42.48531, 'lon': -83.37716}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '89106', 'city': 'Las Vegas', 'state': 'Nevada', 'country': 'United States', 'facility': 'Cleveland Clinic Lou Ruvo Center for Brain Health', 'geoPoint': {'lat': 36.17497, 'lon': -115.13722}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Irving Center for Clinical and Translational Research', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Hospital of the University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Transposon Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}