Ignite Creation Date:
2025-12-25 @ 12:04 AM
Ignite Modification Date:
2025-12-28 @ 10:26 PM
Study NCT ID:
NCT00004658
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase II Study of Ribose, Uridine, and Thymidine for a Complex Syndrome Involving Excessive 5'-Nucleotidase Activity
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535321', 'term': '5-Nucleotidase syndrome'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'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'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D012266', 'term': 'Ribose'}, {'id': 'D013936', 'term': 'Thymidine'}, {'id': 'D014529', 'term': 'Uridine'}], 'ancestors': [{'id': 'D010429', 'term': 'Pentoses'}, {'id': 'D009005', 'term': 'Monosaccharides'}, {'id': 'D000073893', 'term': 'Sugars'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D011741', 'term': 'Pyrimidine Nucleosides'}, {'id': 'D011743', 'term': 'Pyrimidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D003853', 'term': 'Deoxyribonucleosides'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D012263', 'term': 'Ribonucleosides'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1995-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2001-12', 'lastUpdateSubmitDate': '2005-06-23', 'studyFirstSubmitDate': '2000-02-24', 'studyFirstSubmitQcDate': '2000-02-24', 'lastUpdatePostDateStruct': {'date': '2005-06-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2000-02-25', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ["5'-nucleotidase syndrome", 'inborn errors of metabolism', 'rare disease'], 'conditions': ["5'-Nucleotidase Syndrome"]}, 'descriptionModule': {'briefSummary': "OBJECTIVES: I. Evaluate the efficacy of oral ribose in patients with a complex 5'-nucleotidase syndrome who have not received uridine (UR) and thymidine (TDR).\n\nII. Evaluate the efficacy of UR/oral ribose and UR/TDR. III. Evaluate the efficacy of oral ribose given in combination with UR/TDR.", 'detailedDescription': 'PROTOCOL OUTLINE: Newly identified patients receive ribose daily for 8 weeks. Patients who improve continue therapy. If improvement then reaches a plateau, uridine (UR) and thymidine (TDR) are added to the regimen.\n\nPatients who receive no benefit after 8 weeks of ribose are treated with UR/TDR. Patients who improve after 8 weeks of this combination continue therapy.\n\nRibose is added to the treatment regimen for patients on UR/TDR at entry. At maximal improvement, TDR and UR are sequentially tapered.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '0 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics-- Complex syndrome with excessive skin fibroblast 5'-nucleotidase activity characterized by: Recurrent infection Seizure disorder Speech impairment Attention deficit and behavior problems --Prior/Concurrent Therapy-- Concurrent uridine and thymidine therapy allowed --Patient Characteristics-- Other: No pregnant women"}, 'identificationModule': {'nctId': 'NCT00004658', 'briefTitle': "Phase II Study of Ribose, Uridine, and Thymidine for a Complex Syndrome Involving Excessive 5'-Nucleotidase Activity", 'organization': {'class': 'NIH', 'fullName': 'Office of Rare Diseases (ORD)'}, 'orgStudyIdInfo': {'id': '199/11828'}, 'secondaryIdInfos': [{'id': 'UCSD-782'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'ribose', 'type': 'DRUG'}, {'name': 'thymidine', 'type': 'DRUG'}, {'name': 'uridine', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Alice L. Yu', 'role': 'STUDY_CHAIR', 'affiliation': 'University of California, San Diego'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Center for Research Resources (NCRR)', 'class': 'NIH'}, 'collaborators': [{'name': 'University of California, San Diego', 'class': 'OTHER'}]}}}