NCT00004658

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: —
• Sponsor: National Center for Research Resources (NCRR) · NIH
• Sex: All
• Age: 0 Years – 21 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• 5'-Nucleotidase Syndrome

Interventions

Type	Name	Description
DRUG	ribose
DRUG	thymidine
DRUG	uridine

Timeline

• Start date: 1995-03-01
• First posted: 2000-02-25
• Last updated: 2005-06-24