Phase II Study of Ribose, Uridine, and Thymidine for a Complex Syndrome Involving Excessive 5'-Nucleotidase Activity

5'-Nucleotidase Syndrome Clinical Trial

Status Completed

Clinical Trial Summary

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).

II. Evaluate the efficacy of UR/oral ribose and UR/TDR. III. Evaluate the efficacy of oral ribose given in combination with UR/TDR.

Clinical Trial Description

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.

Patients 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.

Ribose is added to the treatment regimen for patients on UR/TDR at entry. At maximal improvement, TDR and UR are sequentially tapered. ;

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• 5'-Nucleotidase Syndrome
• Metabolism, Inborn Errors

• NCT number: NCT00004658
• Study type: Interventional
• Source: Office of Rare Diseases (ORD)
• Status: Completed
• Phase: Phase 2
• Start date: March 1995