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NCT02845609: SA-thrombo

A Phase 2 Study to Evaluate the Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Related Thrombocytopenia

Completed Phase 2 Last updated 29 January 2018
What this trial tests

Phase 2 trial testing Sialic Acid-Extended Release in Thrombocytopenia in 5 participants. Completed in 1 September 2017.

Timeline
1 September 2016
Primary endpoint
1 September 2017
1 September 2017

Quick facts

Lead sponsorHadassah Medical Organization
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment5
Start date1 September 2016
Primary completion1 September 2017
Estimated completion1 September 2017

Drugs / interventions tested

Conditions studied

Sponsor

Hadassah Medical Organization — full company profile →

Who can join

Adults 18 to 45, any sex, with Thrombocytopenia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Biosynthetic and genetic pathways related to sialic acid metabolism.
    Huang S, van de Ven EGP, Tee T, Lefeber DJ. · · 2026 · PMID 41655697 · DOI 10.1016/j.jbc.2026.111262

Verify or expand the search:

Other recruiting trials for Thrombocytopenia

Currently open trials in the same condition.

Other Hadassah Medical Organization trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02845609.

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