Last reviewed · How we verify
NCT04320329
Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis
trial in Morquio B Disease in 30 participants. Status unknown.
31 May 2021
Quick facts
| Lead sponsor | University of British Columbia |
|---|---|
| Status | Status unknown |
| Study type | OBSERVATIONAL |
| Enrollment | 30 |
| Start date | 1 June 2020 |
| Primary completion | 31 May 2021 |
| Estimated completion | 31 May 2022 |
| Sites | 1 location across Canada |
Conditions studied
- Morquio B Disease — all drugs for Morquio B Disease →
- GM1 Gangliosidosis Type III — all drugs for GM1 Gangliosidosis Type III →
Sponsor
University of British Columbia
Who can join
Eligibility, any sex, with Morquio B Disease or GM1 Gangliosidosis Type III. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Mucopolysaccharidosis type IVB (Morquio-B disease, MBD) is an autosomal-recessive lysosomal disease caused by mutations in a gene called GLB1. Clinically, Morquio B presents with progressive skeletal deformities involving mostly long bones and spine. While the information on GLB1 mutations associated with MBD is limited, there is a significant overlap in clinical presentation between Morquio B and late-onset GM1 gangliosidosis with both conditions being caused by mutations in the same GLB1 gene. In this study, the investigators plan to collect retrospective data from patients' medical charts, as well as, information from the prospective follow up clinic visits. There will be two study visits with the interval of one year. The study procedures will include a detailed physical exam, bone scans, heart and lung function, physical endurance tests, hearing test, laboratory tests and quality of life surveys. The purpose of this study is to collect data on the natural history of Morquio B and to create a biobank of laboratory samples (blood, urine and skin cells) for future research. This information will improve the understanding of the natural progression of Morquio B disease.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Morquio B Disease. Disease Characteristics and Treatment Options of a Distinct <i>GLB1</i>-Related Dysostosis Multiplex.
Yuskiv N, Higaki K, Stockler-Ipsiroglu S. · · 2020 · cited 14× · PMID 33266180 · DOI 10.3390/ijms21239121
Verify or expand the search:
- PubMed search for NCT04320329
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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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 NCT04320329 (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 University of British Columbia
- Last refreshed: 30 April 2020
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/NCT04320329.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing