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

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ClinicalTrials.gov Identifier: NCT00004658
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
Sponsor:
Collaborator:
University of California, San Diego
Information provided by:
Office of Rare Diseases (ORD)

Brief 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.


Condition or disease Intervention/treatment Phase
5'-Nucleotidase Syndrome Drug: ribose Drug: thymidine Drug: uridine Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Study Start Date : March 1995

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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


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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00004658     History of Changes
Other Study ID Numbers: 199/11828
UCSD-782
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
5'-nucleotidase syndrome
inborn errors of metabolism
rare disease

Additional relevant MeSH terms:
Syndrome
Metabolism, Inborn Errors
Disease
Pathologic Processes
Genetic Diseases, Inborn
Metabolic Diseases