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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
Phase 2 trial testing Sialic Acid-Extended Release in Thrombocytopenia in 5 participants. Completed in 1 September 2017.
1 September 2017
Quick facts
| Lead sponsor | Hadassah Medical Organization |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 1 September 2016 |
| Primary completion | 1 September 2017 |
| Estimated completion | 1 September 2017 |
Drugs / interventions tested
- Sialic Acid-Extended Release — full drug profile →
Conditions studied
- Thrombocytopenia — all drugs for Thrombocytopenia →
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.
-
Increase in platelet count from baseline
Time frame: 3 month
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):
-
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:
- PubMed search for NCT02845609
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Thrombocytopenia
Currently open trials in the same condition.
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- NCT07257809 — Herombopag Treated T-DM1 Induced Platelet Reduction · Phase 2 · recruiting
- NCT06676904 — Neonatal Platelet Transfusion Threshold Trial · NA · recruiting
- NCT06886516 — Apixaban in Thrombocytopenia · EARLY_PHASE1 · recruiting
Other Hadassah Medical Organization trials
Trials by the same sponsor.
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- NCT07333430 — Study of Naive HBI0101 CAR-T Therapy in Relapsed/Refractory Multiple Myeloma · Phase 1 · recruiting
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT02845609 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Hadassah Medical Organization
- Last refreshed: 29 January 2018
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.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing