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Sialic Acid Supplementation in N-Acetylneuraminic Acid Synthase (NANS) Deficiency

NCT03545568 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2018-11-14

No results posted yet for this study

Summary

This study is aimed at assessing the impact of short-term (3 days) exogenous sialic acid supplementation on endogenous biomarkers of sialic acid metabolism in NANS deficient patients.

Conditions

  • N-Acetylneuraminic Acid Storage Disease

Interventions

OTHER

Neu5Ac supplementation

Sialic acid as N-Acetyl-neuraminic acid dehydrate (Neu5Ac) 150 mg/kg/d (max 12g/d) in three doses orally in subjects with NANS deficiency compared to controls

Sponsors & Collaborators

  • University of Modena and Reggio Emilia

    collaborator OTHER

  • University of Lausanne

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
60 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2018-05-01
Primary Completion
2018-10-15
Completion
2018-10-15

Countries

  • Italy
  • Switzerland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03545568 on ClinicalTrials.gov