Ignite Creation Date:
2025-12-24 @ 10:21 PM
Ignite Modification Date:
2026-01-02 @ 5:31 PM
Study NCT ID:
NCT06839235
Status:
RECRUITING
Last Update Posted:
2025-12-24
First Post:
2025-02-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C536414', 'term': 'Primary hyperoxaluria type 1'}, {'id': 'D006960', 'term': 'Hyperoxaluria, Primary'}], 'ancestors': [{'id': 'D006959', 'term': 'Hyperoxaluria'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Single ascending dose escalation/adaptive design, followed by single dose expansion'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 23}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-06-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2043-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-23', 'studyFirstSubmitDate': '2025-02-17', 'studyFirstSubmitQcDate': '2025-02-17', 'lastUpdatePostDateStruct': {'date': '2025-12-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-02-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)', 'timeFrame': 'Up to 6 months'}], 'secondaryOutcomes': [{'measure': 'Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6', 'timeFrame': 'Up to 6 months'}, {'measure': 'Absolute change in UOx corrected for body surface area', 'timeFrame': 'Up to 6 months'}, {'measure': 'Percent change in plasma glycolate from Baseline to Month 6', 'timeFrame': 'Up to 6 months'}, {'measure': 'Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24', 'timeFrame': 'Up to 24 months'}, {'measure': 'Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)', 'timeFrame': 'Up to 6 months'}, {'measure': 'Urine concentrations for LNP lipids', 'timeFrame': 'Up to 6 months'}, {'measure': 'Antidrug antibodies to ABO-101 and anti-Cas protein antibodies', 'timeFrame': 'Up to 6 months'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PH1', 'Primary hyperoxaluria', 'AGXT', 'CRISPR', 'Gene Editing', 'Pharmacokinetics', 'Pharmacodynamics', 'ABO-101', 'redePHine'], 'conditions': ['Primary Hyperoxaluria Type 1 (PH1)']}, 'descriptionModule': {'briefSummary': 'The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, pediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring program to comply with local and national requirements.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '64 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria for Parts A and B\n\n* Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase (AGXT) gene (valid historical laboratory data will be reviewed and approved by the Sponsor)\n* Age at time of signing the informed consent/assent form:\n\n * Part A: ≥18 years to ≤64 years\n * Part B: ≥6 years to \\<18 years\n* 24-hour UOx ≥0.7 mmol/24 hours/1.73 m²\n* eGFR ≥30 mL/min/1.73m²\n* Weight ≤90 kg\n\nKey Exclusion Criteria for Parts A and B\n\n* Confirmed diagnosis of primary hyperoxaluria type 2 or type 3\n* History of a liver, kidney or combined liver/kidney transplant\n* Currently on dialysis\n* Participant has previously used (within past 24 months) or is currently receiving an approved or investigational urinary oxalate lowering RNA interference (RNAi) or siRNA therapy\n* Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)'}, 'identificationModule': {'nctId': 'NCT06839235', 'briefTitle': 'Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Arbor Biotechnologies'}, 'officialTitle': 'A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants With Primary Hyperoxaluria Type 1 (PH1)', 'orgStudyIdInfo': {'id': 'ABO-101-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design', 'interventionNames': ['Drug: ABO-101']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental: Part B: Single Dose Expansion', 'interventionNames': ['Drug: ABO-101']}], 'interventions': [{'name': 'ABO-101', 'type': 'DRUG', 'description': 'Intravenous (IV) infusion', 'armGroupLabels': ['Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design', 'Experimental: Part B: Single Dose Expansion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Clinical Trial Site', 'role': 'CONTACT', 'phone': '800-664-4542'}], 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '55114', 'city': 'Saint Paul', 'state': 'Minnesota', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Nucleus Network', 'geoPoint': {'lat': 44.94441, 'lon': -93.09327}}, {'city': 'Lyon', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'role': 'CONTACT', 'phone': '04 27 85 61 28'}], 'facility': 'Hospices Civils de Lyon- Hôpital Femmes Mères Enfants', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Bonn', 'status': 'NOT_YET_RECRUITING', 'country': 'Germany', 'contacts': [{'role': 'CONTACT', 'email': 'info@knz-bonn.de', 'phone': '0228 6883860'}], 'facility': 'Kindernierenzentrum Bonn', 'geoPoint': {'lat': 50.73438, 'lon': 7.09549}}, {'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'role': 'CONTACT', 'email': 'ClinicalResearchFacilityEnquiries@uhb.nhs.uk', 'phone': '0121 371 3170'}], 'facility': 'Queen Elizabeth Hospital Birmingham', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Royal Free Clinical Research', 'role': 'CONTACT', 'email': 'rf.crf@nhs.net', 'phone': '020 7794 0500', 'phoneExt': '32038'}], 'facility': 'Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Daniel Ory, MD', 'role': 'CONTACT', 'email': 'arbortrials@arbor.bio', 'phone': '617-500-8941'}], 'overallOfficials': [{'name': 'Winston Yan, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Arbor Biotechnologies'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Arbor Biotechnologies', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}