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

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	National Center for Research Resources (NCRR) University of California, San Diego
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004658

Purpose

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.

Condition	Intervention	Phase
5'-Nucleotidase Syndrome	Drug: ribose Drug: thymidine Drug: uridine	Phase II

Drug Information available for: Ribose Thymidine Uridine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

Further study details as provided by Office of Rare Diseases (ORD):

Study Start Date:

March 1995

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.

Eligibility

Ages Eligible for Study:	up to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004658

Sponsors and Collaborators

National Center for Research Resources (NCRR)

University of California, San Diego

Investigators

Study Chair:

Alice L. Yu

University of California, San Diego

More Information

No publications provided

Study ID Numbers:	199/11828, UCSD-782
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004658 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

5'-nucleotidase syndrome
inborn errors of metabolism
rare disease

Study placed in the following topic categories:

Metabolism, Inborn Errors
Rare Diseases
5-Nucleotidase Syndrome

Additional relevant MeSH terms:

Pathologic Processes
Disease
Syndrome

ClinicalTrials.gov processed this record on May 07, 2009