Ignite Creation Date:
2025-12-24 @ 4:31 PM
Ignite Modification Date:
2026-01-03 @ 9:40 PM
Study NCT ID:
NCT06153966
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-12-27
First Post:
2023-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017096', 'term': 'Prion Diseases'}], 'ancestors': [{'id': 'D002494', 'term': 'Central Nervous System Infections'}, {'id': 'D007239', 'term': 'Infections'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 56}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-01-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2027-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-12-24', 'studyFirstSubmitDate': '2023-11-21', 'studyFirstSubmitQcDate': '2023-11-29', 'lastUpdatePostDateStruct': {'date': '2024-12-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-12-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of treatment-emergent adverse events.', 'timeFrame': 'Baseline up to Week 33'}], 'secondaryOutcomes': [{'measure': 'Maximum Observed Plasma Concentration (Cmax) of ION717', 'timeFrame': 'on Day 1 and Week 9'}, {'measure': 'Area Under the Plasma Concentration-time Curve (AUC) of ION717', 'timeFrame': 'on Day 1 and Week 9'}, {'measure': 'Half-life (t1/2λz) of ION717 in Plasma', 'timeFrame': 'on Day 1 and Week 9'}, {'measure': 'Cerebrospinal fluid (CSF) Concentration of ION717', 'timeFrame': 'Pre-dose and at multiple points post-dose up to Week 33'}, {'measure': 'Amount of ION717 Excreted in Urine', 'timeFrame': 'Post-dose on Day 1'}, {'measure': 'Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF', 'timeFrame': 'Pre-dose and at multiple points post-dose up to Week 33'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Prion Disease']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.', 'detailedDescription': 'This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and placebo. The study will consist of a screening period of up to 6 weeks, a 30-week double-blind treatment period, a 70-week open-label extension period and a 32-week post-treatment period. During the double-blind period, the order of ION717 and placebo doses will be randomized and blinded to participants, study sites and the Sponsor. During the open-label extension period, all participants will receive ION717. Multiple dose levels will be tested.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria\n\n* A confirmed diagnosis of probable or definite prion disease.\n* Early-stage prion disease at the time of Screening.\n* Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.\n* Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.\n* Aged ≥ 18 at the time of informed consent.\n\nKey Exclusion Criteria\n\n* Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.\n* Any contraindication or unwillingness to undergo an MRI.\n* Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.\n* Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.\n* Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study."}, 'identificationModule': {'nctId': 'NCT06153966', 'briefTitle': 'PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ionis Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease', 'orgStudyIdInfo': {'id': 'ION717-CS2'}, 'secondaryIdInfos': [{'id': '2023-503355-98', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ION717 + Placebo, Regimen 1', 'description': 'Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.', 'interventionNames': ['Drug: ION717', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'ION717 + Placebo, Regimen 2', 'description': 'Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.', 'interventionNames': ['Drug: ION717', 'Drug: Placebo']}], 'interventions': [{'name': 'ION717', 'type': 'DRUG', 'description': 'ION717 will be administered by IT injection.', 'armGroupLabels': ['ION717 + Placebo, Regimen 1', 'ION717 + Placebo, Regimen 2']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo-matching ION717 will be administered by IT injection.', 'armGroupLabels': ['ION717 + Placebo, Regimen 1', 'ION717 + Placebo, Regimen 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Neurology Clinic', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80204', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado Hospital', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'NYU Langone Health', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'University Hospitals Cleveland Medical Center', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '3050', 'city': 'Parkville', 'country': 'Australia', 'facility': 'Royal Melbourne Hospital', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': 'T6G 2M8', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Alberta', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'H3A 2B4', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'McGill University Health Centre', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Universitaire Pitié Salpêtrière', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '37075', 'city': 'Göttingen', 'country': 'Germany', 'facility': 'University Medical Center Göttingen', 'geoPoint': {'lat': 51.53443, 'lon': 9.93228}}, {'zip': '6423906', 'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Tel Aviv Sourasky Medical Center', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'zip': '20133', 'city': 'Milan', 'country': 'Italy', 'facility': 'Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '372-0006', 'city': 'Gunma', 'state': 'Isesaki-shi', 'country': 'Japan', 'facility': 'Mihara Memorial Hospital'}, {'zip': '187-8551', 'city': 'Tokyo', 'state': 'Kodaira-shi', 'country': 'Japan', 'facility': 'National Center of Neurology and Psychiatry', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '751-0826', 'city': 'Yamaguchi', 'state': 'Shimonoseki-shi', 'country': 'Japan', 'facility': 'Neuromuscular Center Yoshimizu Hospital', 'geoPoint': {'lat': 34.18333, 'lon': 131.46667}}, {'zip': '08036', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Clinic De Barcelona', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/ourmember/ionis/', 'ipdSharing': 'YES', 'description': 'Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ionis Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}