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

Sponsor
National Center for Research Resources (NCRR) (NIH)
Overall Status
Completed
CT.gov ID
NCT00004658
Collaborator
University of California, San Diego (Other)

Study Details

Study Description

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).

  1. 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
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 Design

Study Type:
Interventional
Primary Purpose:
Treatment
Study Start Date :
Mar 1, 1995

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Years to 21 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    No locations specified.

    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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004658
    Other Study ID Numbers:
    • 199/11828
    • UCSD-782
    First Posted:
    Feb 25, 2000
    Last Update Posted:
    Jun 24, 2005
    Last Verified:
    Dec 1, 2001
    Keywords provided by , ,
    5'-nucleotidase syndrome
    inborn errors of metabolism
    rare disease
    Additional relevant MeSH terms:
    Syndrome

    Study Results

    No Results Posted as of Jun 24, 2005