NCT00004658

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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 1995

Completed
5 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2001

First QC Date

February 24, 2000

Last Update Submit

June 23, 2005

Conditions

5'-Nucleotidase Syndrome

Keywords

5'-nucleotidase syndromeinborn errors of metabolismrare disease

Interventions

riboseDRUG
thymidineDRUG
uridineDRUG

Eligibility Criteria

Age0 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

5-Nucleotidase syndromeMetabolism, Inborn ErrorsRare Diseases

Interventions

RiboseThymidineUridine

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

PentosesMonosaccharidesSugarsCarbohydratesPyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsDeoxyribonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Alice L. Yu

    University of California, San Diego

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

March 1, 1995

Last Updated

June 24, 2005

Record last verified: 2001-12