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Mucopolysaccharidoses clinical trials

The registry lists 59 studies for Mucopolysaccharidoses across more than 10 countries. 11 are recruiting or about to start. 25 of them are mainly about Mucopolysaccharidoses (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-21. Counting its 5 narrower conditions too, such as Mucopolysaccharidosis IV: 308 studies, 53 open.

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Status

Completed
28
Terminated
8
Status unknown
8
Recruiting
6
Not yet recruiting
5
Withdrawn
2

Phase

Phase 2
14
Phase 1
13
Phase 3
5
Phase 4
1

Where

United States
37
United Kingdom
15
Spain
6
Brazil
6
Germany
4
Netherlands
4

Who runs them

Shire (5) · Patricia I. Dickson, M.D. (3) · Assiut University (3) · Ultragenyx Pharmaceutical Inc (3) · Alexion Pharmaceuticals, Inc. (3) · Takeda (2) · University of Minnesota (2) · Children's Hospital of Orange County (2)

What is being tested here

Drugs under test in Mucopolysaccharidoses studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Alemtuzumab102
Hydroxyurea101
Fludarabine101
Melphalan101
Thiotepa101
Laronidase004
Vestronidase alfa023
N-sulfoglucosamine sulfohydrolase002
Cyclophosphamide001
Busulfan001

Coming up

1phase 3 study under way
1study finishes main data collection within a year

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The study

Who these studies take

Of the 6 Mucopolysaccharidoses studies recruiting now, 2 (33%) set no upper age limit and 3 (50%) stop at 75 or younger. 5 (83%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (33%) take healthy volunteers.

Recruiting studies that take each age, of 6
AgeWomenMen
104 (67%)4 (67%)
185 (83%)5 (83%)
305 (83%)5 (83%)
405 (83%)5 (83%)
505 (83%)5 (83%)
653 (50%)3 (50%)
753 (50%)3 (50%)
853 (50%)3 (50%)

Across all 59 studies, recruiting or not: 59% stop at 75 or younger and 59% at 65; 92% take children; 2% are for women only and 10% for men only; 14% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 50 of 59. Browse all 59 → or search them.