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Mucopolysaccharidosis IV clinical trials

The registry lists 101 studies for Mucopolysaccharidosis IV across more than 10 countries. 27 are recruiting or about to start. 54 of them are mainly about Mucopolysaccharidosis IV (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.

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Status

Completed
52
Recruiting
20
Terminated
10
Status unknown
6
Enrolling by invitation
4
Not yet recruiting
3

Phase

Phase 2
20
Phase 1
8
Phase 3
8
Phase 4
5

Where

United States
56
United Kingdom
22
Canada
21
France
19
Spain
13
Italy
12

Who runs them

BioMarin Pharmaceutical (11) · QED Therapeutics, a BridgeBio company (8) · University of California, San Francisco (3) · Emory University (2) · State Key Laboratory of Respiratory Disease (2) · Regeneron Pharmaceuticals (2) · CENTOGENE GmbH Rostock (2) · Supplement Formulators, Inc. (2)

What is being tested here

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

DrugOpenPhase 3All
Infigratinib325
Garetosmab111
Risedronic Acid101
Laronidase101
Methylcellulose101
Elosulfase alfa027
Recifercept002
Vosoritide001
Probiotic001
Psyllium001

Coming up

2phase 3 studies under way
9studies finish main data collection within a year1 of them phase 3

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

All 9, phase 3 first

Who these studies take

Of the 20 Mucopolysaccharidosis IV studies recruiting now, 9 (45%) set no upper age limit and 7 (35%) stop at 75 or younger. 15 (75%) take children under 18. 1 (5%) are for women only and 0 (0%) for men only; 7 (35%) take healthy volunteers.

Recruiting studies that take each age, of 20
AgeWomenMen
1014 (70%)14 (70%)
1817 (85%)16 (80%)
3016 (80%)15 (75%)
4016 (80%)15 (75%)
5016 (80%)15 (75%)
6514 (70%)14 (70%)
7513 (65%)13 (65%)
8513 (65%)13 (65%)

Across all 101 studies, recruiting or not: 46% stop at 75 or younger and 36% at 65; 62% take children; 3% are for women only and 1% for men only; 33% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Mucopolysaccharidosis IV studies take

Of 19 interventional studies with no phase under Mucopolysaccharidosis IV, half reached primary completion within 8 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Interventional, no phase8 mo5–13 · 19 studies——

Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out

What changed recently

From the registry's own updates since Oct 14, 2026.

Newly registered1

Studies

Recruiting first, then most recently updated. Showing 50 of 101. Browse all 101 → or search them.