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Efficacy of Sialic Acid GNE Related Thrombocytopenia

NCT02845609 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2018-01-30

No results posted yet for this study

Summary

Sialic Acid-Extended Release (SA-ER, aceneuramic acid, UX001) is an extended release formulation of sialic acid (SA, also known as N-acetylneuraminic acid or NANA). The SA-ER is currently studied as a substrate replacement therapy for patients with GNE myopathy. The investigators plan to study the SA-ER compound in a cohort of five patients with GNE-related thrombocytopenia.

Conditions

Interventions

DRUG

Sialic Acid-Extended Release

Oral drug of Sialic acid

Sponsors & Collaborators

  • Hadassah Medical Organization

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-09-30
Primary Completion
2017-09-30
Completion
2017-09-30

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Entities

Diseases

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 NCT02845609 on ClinicalTrials.gov