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NCT01509768
Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
trial in Sanfilippo Syndrome Type B in 19 participants. Completed in 10 October 2013.
10 October 2013
Quick facts
| Lead sponsor | Shire |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 19 |
| Start date | 9 April 2012 |
| Primary completion | 10 October 2013 |
| Estimated completion | 10 October 2013 |
| Sites | 4 locations across United Kingdom, United States, Germany |
Conditions studied
- Sanfilippo Syndrome Type B — all drugs for Sanfilippo Syndrome Type B →
Sponsor
Shire — full company profile →
Who can join
Adults 1 to 10, any sex, with Sanfilippo Syndrome Type B. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including standardized clinical, biochemical, neurocognitive, developmental, and imaging measures.
Publications & conference data
4 peer-reviewed publications reference this trial (live from Europe PMC):
-
The Neurobehavioral Phenotype in Mucopolysaccharidosis Type IIIB: an Exploratory Study.
Shapiro E, King K, Ahmed A, Rudser K, et al · · 2016 · cited 35× · PMID 26918231 · DOI 10.1016/j.ymgmr.2016.01.003 -
Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B.
Whitley CB, Cleary M, Eugen Mengel K, Harmatz P, et al · · 2018 · cited 25× · PMID 29661560 · DOI 10.1016/j.jpeds.2018.01.044 -
The significance of triple-capsid-mutant AAV8 for treatment of Sanfilippo Syndrome Type B.
Ashby F, Heldermon C. · · 2022 · cited 2× · PMID 38596745 · DOI 10.46439/stemcell.3.013 -
Neuroinflammation as a Novel Therapeutic Frontier for Sanfilippo Syndrome.
Rigante D, Veredice C. · · 2025 · PMID 41300647 · DOI 10.3390/children12111530
Verify or expand the search:
- PubMed search for NCT01509768
- Europe PMC full search
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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 NCT01509768 (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 Shire
- Last refreshed: 17 March 2021
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/NCT01509768.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing