Ignite Creation Date:
2025-12-24 @ 4:13 PM
Ignite Modification Date:
2025-12-28 @ 1:25 PM
Study NCT ID:
NCT07125066
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-08-15
First Post:
2025-07-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C075773', 'term': '4-phenylbutyric acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 1}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2025-07-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2027-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-07', 'studyFirstSubmitDate': '2025-07-30', 'studyFirstSubmitQcDate': '2025-08-07', 'lastUpdatePostDateStruct': {'date': '2025-08-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-08-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of treatment related adverse events', 'timeFrame': '2 years'}], 'secondaryOutcomes': [{'measure': 'Change in the amount of urine D,L 2-Hydroxyglutaric acid in urine', 'timeFrame': '2 years'}, {'measure': 'Change in phenylacetylglutamine in urine', 'timeFrame': '2 years'}, {'measure': 'Change in the number of seizures reported by parents', 'timeFrame': '2 years'}, {'measure': 'Change in ventilator support', 'timeFrame': '2 years'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Combined D,L-2-hydroxyglutaric aciduria'], 'conditions': ['Combined D,L-2-hydroxyglutaric Aciduria']}, 'descriptionModule': {'briefSummary': 'This is an individual patient research protocol to treat a patient diagnosed with Combined D,L-2-hydroxyglutaric aciduria (C-2HGA) with ACER-001.', 'detailedDescription': 'Patient will continue to be seen during routine outpatient clinic visits every 6 months. At those visits the following will be collected: routine metabolic laboratory tests (including a complete metabolic profile, complete blood count, blood phenylacetylglutamine level, and urine organic acids), physical examinations, and an update of clinical history, especially regarding seizures and respiratory status.\n\nOnce consent is signed, the patient will continue to take ACER-001 as prescribed. The ACER-001 will be provided to the patient at no cost.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Single patient with Combined D,L-2-hydroxyglutaric aciduria\n* Parental permission must be given at the start of the protocol.\n* Parents willing to adhere to the protocol\n\nExclusion Criteria:\n\n* Refusal or withdrawal of consent.\n* Drug Intolerance.'}, 'identificationModule': {'nctId': 'NCT07125066', 'briefTitle': 'An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)', 'organization': {'class': 'OTHER', 'fullName': 'University of Pittsburgh'}, 'officialTitle': 'An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2-hydroxyglutaric Aciduria (C-2HGA)', 'orgStudyIdInfo': {'id': 'STUDY25060137'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'sodium phenylbutyrate', 'description': 'a single patient will take sodium phenylbutyrate (ACER-001) current at a dose of 12 g/day; dose will be modified if the patient gains more than 5 kg of weight', 'interventionNames': ['Drug: Sodium phenylbutyrate']}], 'interventions': [{'name': 'Sodium phenylbutyrate', 'type': 'DRUG', 'otherNames': ['Olpruva'], 'description': 'open-label design with doses of sodium phenylbutyrate', 'armGroupLabels': ['sodium phenylbutyrate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh of UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}], 'overallOfficials': [{'name': 'Gerard Vockley, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "UPMC Children's Hospital of Pittsburgh"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Jerry Vockley, MD, PhD', 'class': 'OTHER'}, 'collaborators': [{'name': 'Zevra Therapeutics', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Chief, Division of Genetic and Genomic Medicine', 'investigatorFullName': 'Jerry Vockley, MD, PhD', 'investigatorAffiliation': 'University of Pittsburgh'}}}}