Ignite Creation Date:
2025-12-26 @ 10:17 PM
Ignite Modification Date:
2025-12-26 @ 10:17 PM
Study NCT ID:
NCT06411912
Status:
COMPLETED
Last Update Posted:
2025-10-27
First Post:
2024-05-08
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of NIDO-361 in Patients With SBMA
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D055534', 'term': 'Bulbo-Spinal Atrophy, X-Linked'}], 'ancestors': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Patients will be randomly assigned to receive NIDO-361 or placebo at the recommended Phase 2 dose (RP2D) of 100 mg once daily. Patients will receive daily oral doses of NIDO-361 or placebo for 12 months.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 54}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2024-03-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2025-10-22', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-23', 'studyFirstSubmitDate': '2024-05-08', 'studyFirstSubmitQcDate': '2024-05-10', 'lastUpdatePostDateStruct': {'date': '2025-10-27', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-05-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-10-22', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change from baseline in thigh and total lean muscle volume as assessed by whole-body MRI', 'timeFrame': 'Through study completion, an average of 1 year'}, {'measure': 'Number of patients with adverse events or serious adverse events. Number of patients discontinuing study and number of deaths. Number of mild, moderate, and severe adverse events.', 'timeFrame': 'Through study completion, an average of 1 year'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Spinal and Bulbar Muscular Atrophy', "Kennedy's Disease"]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Ambulatory male\n* Documented SBMA diagnosis confirmed by DNA genetic testing\n* Able to complete six-minute walk test (6MWT)\n\nExclusion Criteria:\n\n* Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, immunologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the participant's safe participation in the study or would interfere with the study assessments\n* Inability to undergo MRI (mild sedation may be allowed)"}, 'identificationModule': {'nctId': 'NCT06411912', 'acronym': 'PIONEER KD', 'briefTitle': 'A Study of NIDO-361 in Patients With SBMA', 'organization': {'class': 'INDUSTRY', 'fullName': 'Nido Biosciences, Inc.'}, 'officialTitle': 'A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)', 'orgStudyIdInfo': {'id': 'NIDO-361-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NIDO-361', 'description': 'Participants receive 100 mg of NIDO-361 given orally once daily.', 'interventionNames': ['Drug: NIDO-361']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Participants receive matched dose placebo given orally once daily.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'NIDO-361', 'type': 'DRUG', 'description': 'Tablets containing 100mg of NIDO-361 for oral administration.', 'armGroupLabels': ['NIDO-361']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo oral tablets', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'city': 'Milan', 'country': 'Italy', 'facility': 'IRCCS Istituto Neurologico Carlo Besta', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'Padua', 'country': 'Italy', 'facility': 'Azienda Ospedale Università di Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Daegu', 'country': 'South Korea', 'facility': 'Kyungpook National University Chilgok Hospital', 'geoPoint': {'lat': 35.87028, 'lon': 128.59111}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'University of College London Hospital (UCLH)', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Nido Biosciences, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}