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Mucopolysaccharidosis II clinical trials

The registry lists 77 studies for Mucopolysaccharidosis II across more than 10 countries. 8 are recruiting or about to start. 51 of them are mainly about Mucopolysaccharidosis II (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.

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Status

Completed
40
Active, not recruiting
15
Terminated
9
Recruiting
4
Enrolling by invitation
3
Withdrawn
3

Phase

Phase 2
31
Phase 1
20
Phase 3
19
Phase 4
4

Where

United States
51
United Kingdom
21
Canada
13
Brazil
13
Spain
11
France
10

Who runs them

Shire (15) · JCR Pharmaceuticals Co., Ltd. (7) · REGENXBIO Inc. (6) · Takeda (5) · Green Cross Corporation (5) · Denali Therapeutics Inc. (4) · AO GENERIUM (3) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (3)

What is being tested here

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

DrugOpenPhase 3All
Pabinafusp alfa147
Tividenofusp alfa123
Alemtuzumab102
Hydroxyurea101
Fludarabine101
Melphalan101
Thiotepa101
Idursulfase0712
Idursulfase beta023
Adalimumab002

Coming up

9phase 3 studies under way
9studies finish main data collection within a year2 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 4 Mucopolysaccharidosis II studies recruiting now, 1 (25%) set no upper age limit and 3 (75%) stop at 75 or younger. 4 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.

Recruiting studies that take each age, of 4
AgeWomenMen
104 (100%)4 (100%)
184 (100%)4 (100%)
303 (75%)3 (75%)
403 (75%)3 (75%)
503 (75%)3 (75%)
651 (25%)1 (25%)
751 (25%)1 (25%)
851 (25%)1 (25%)

Across all 77 studies, recruiting or not: 61% stop at 75 or younger and 60% at 65; 92% take children; 0% are for women only and 52% for men only; 6% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Mucopolysaccharidosis II studies take

Of 16 observational studies under Mucopolysaccharidosis II, half reached primary completion within 51 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Observational51 mo24–119 · 16 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

Studies

Recruiting first, then most recently updated. Showing 50 of 77. Browse all 77 → or search them.