Mucopolysaccharidosis I clinical trials

The registry lists 74 studies for Mucopolysaccharidosis I across more than 10 countries. 7 are recruiting or about to start. 54 of them are mainly about Mucopolysaccharidosis I (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-04.

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Status

Completed: 39
Terminated: 14
Active, not recruiting: 6
Recruiting: 6
Withdrawn: 5
Status unknown: 1

Phase

Phase 1: 28
Phase 2: 25
Phase 3: 7
Phase 4: 4

Where

United States: 53
Brazil: 11
Germany: 7
Italy: 7
Canada: 6
United Kingdom: 6

Who runs them

Genzyme, a Sanofi Company (10) · Masonic Cancer Center, University of Minnesota (9) · ArmaGen, Inc (4) · Patricia I. Dickson, M.D. (3) · University of Minnesota (3) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (3) · Orchard Therapeutics (2) · CENTOGENE GmbH Rostock (2)

What is being tested here

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

Drug	Open	Phase 3	All
Alemtuzumab	1	0	3
Hydroxyurea	1	0	1
Fludarabine	1	0	1
Melphalan	1	0	1
Thiotepa	1	0	1
Laronidase	0	3	16
Valanafusp alpha	0	0	4
Cyclophosphamide	0	0	3
Busulfan	0	0	3
Adalimumab	0	0	2

Drug classes tested here Alkylating agents 1 open

Coming up

1phase 3 study under way

5studies finish 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.

All 5

Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
Nov 2026Early Check: Expanded Screening in Newborns · RTI International
Dec 2026MPS (RaDiCo Cohort) (RaDiCo-MPS) · Institut National de la Santé Et de la Recherche Médicale, France
Jan 2027Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Sep 2027Long Term Follow-Up for RGX-111 · REGENXBIO Inc.

Who these studies take

Of the 6 Mucopolysaccharidosis I studies recruiting now, 3 (50%) 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; 0 (0%) take healthy volunteers.

Recruiting studies that take each age, of 6
Age	Women	Men
10	5 (83%)	5 (83%)
18	6 (100%)	6 (100%)
30	6 (100%)	6 (100%)
40	6 (100%)	6 (100%)
50	6 (100%)	6 (100%)
65	3 (50%)	3 (50%)
75	3 (50%)	3 (50%)
85	3 (50%)	3 (50%)

Across all 74 studies, recruiting or not: 47% stop at 75 or younger and 47% at 65; 92% take children; 1% are for women only and 1% for men only; 4% 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 74. Browse all 74 → or search them.