5'-Nucleotidase Syndrome Clinical Trial
|Source||Office of Rare Diseases (ORD)|
|Start date||March 1995|
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.
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.
Primary Purpose: Treatment