Ignite Creation Date:
2025-12-25 @ 2:15 AM
Ignite Modification Date:
2026-03-09 @ 4:28 AM
Study NCT ID:
NCT04069260
Status:
TERMINATED
Last Update Posted:
2020-05-27
First Post:
2019-08-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D003554', 'term': 'Cystinosis'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000709849', 'term': 'ELX-02'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'whyStopped': 'Due to study design limitations, this study has been discontinued and will not proceed with the 2nd cohort as contemplated in the original protocol.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-08-02', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2019-12', 'completionDateStruct': {'date': '2019-12-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-22', 'studyFirstSubmitDate': '2019-08-16', 'studyFirstSubmitQcDate': '2019-08-23', 'lastUpdatePostDateStruct': {'date': '2020-05-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-08-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-12-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'AEs associated with different dose levels of ELX-02', 'timeFrame': 'From the time of first dosing through the follow-up visit, an average of approximately 10 weeks'}, {'measure': 'Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h)', 'timeFrame': 'Day 1 of treatment periods 1, 2, and 3', 'description': 'Full PK profile 12 blood samples over 24 hours'}, {'measure': 'Maximum observed plasma concentration (Cmax)', 'timeFrame': 'Day 1 of treatment periods 1, 2, and 3', 'description': 'Full PK profile 12 blood samples over 24 hours'}, {'measure': 'Observed plasma concentration at 1 hour post dose (C1h)', 'timeFrame': 'Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3', 'description': 'Sparse sampling, blood sampling only, pre-dose and 1 hour post dose'}, {'measure': 'Amount of ELX-02 excreted in urine from 0 to 24 hours (Ae24h)', 'timeFrame': 'Day 1 of treatment periods 1, 2, and 3', 'description': '6 urine collections over 24 hours'}, {'measure': 'Renal clearance on Day 1 (Ae24h/plasma AUC0-24h)', 'timeFrame': 'Day 1 of treatment periods 1, 2, and 3', 'description': '6 urine collections over 24 hours'}], 'secondaryOutcomes': [{'measure': 'Changes from baseline in WBC cystine levels', 'timeFrame': 'Screening; Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3, and 4-week safety follow-up'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['aminoglycoside', 'translational read-through', 'cystinosis', 'nonsense mutation'], 'conditions': ['Genetic Disease', 'Nonsense Mutation', 'Cystinosis']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.eloxxpharma.com', 'label': 'Eloxx Pharmaceuticals Website'}]}, 'descriptionModule': {'briefSummary': 'This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with cystinosis with nonsense mutation in at least one allele.\n\nSix patients will be enrolled in the trial.\n\nThe study will comprise of the following periods for each patient:\n\n* A screening period of up to 6 weeks\n* A total treatment period of 4 weeks\n* A safety follow-up period of 4 weeks after the last treatment\n\nEach patient will receive three escalating doses as follows:\n\n* Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 47.5 µg\\*h/mL)\n* Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 95 µg\\*h/mL)\n* Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14 mg/kg for these two weeks; the daily dose will be individualized to achieve the target weekly exposure of about 190 µg\\*h/mL)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Patients must meet all of the following criteria to participate in this study:\n\n1. Male or female patients who, at the time of screening, are 18 years of age or older (Cohort 1) or ≥12 years of age (Cohort 2)\n2. A diagnosis of nephropathic cystinosis and biallelic CTNS mutations, including at least one nonsense mutation\n3. Patients should have a mild to moderate disease estimated glomerular filtration rate ≥40 mL/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration CKD-EPI formula\n4. Body mass index of 19.0 to 30.0 kg/m2. Patients with a lower BMI may be entered into the study at the discretion of the Investigator following consultation with the Sponsor\n5. Renal transplant permitted with stable graft function (serum creatinine) for 3 months prior to Screening\n\nPatients with any of the following characteristics/conditions will not be included in the study:\n\n1. Participation in clinical study including administration of any investigational drug or device in the last 30 days or 5 half-lives (whichever is longer) prior to investigational product dosing in the current study\n2. Concomitant use of cysteamine bitartrate from 7 days prior to baseline until 7 days following final administration ELX-02\n3. An average systolic blood pressure and/or diastolic blood pressure ≥95th percentile for sex, age, and height on 3 or more occasions during the screening period\n4. Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides\n5. Known relevant allergy or hypersensitivity to aminoglycosides'}, 'identificationModule': {'nctId': 'NCT04069260', 'briefTitle': 'A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Eloxx Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations', 'orgStudyIdInfo': {'id': 'EL-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ELX-02', 'description': 'Eukaryotic ribosomal selective glycoside (ERSG)', 'interventionNames': ['Drug: ELX-02']}], 'interventions': [{'name': 'ELX-02', 'type': 'DRUG', 'description': 'ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG).', 'armGroupLabels': ['ELX-02']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'H3H 2R9', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'McGill University Health Center', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}], 'overallOfficials': [{'name': 'Paul Goodyer, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'McGill University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Eloxx Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}