Leukodystrophy, Metachromatic clinical trials

The registry lists 49 studies for Leukodystrophy, Metachromatic across more than 10 countries. 11 are recruiting or about to start. 23 of them are mainly about Leukodystrophy, Metachromatic (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.

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Status

Completed: 21
Recruiting: 8
Terminated: 6
Withdrawn: 3
Active, not recruiting: 3
No longer available: 3

Phase

Phase 2: 19
Phase 1: 11
Phase 3: 3

Where

United States: 28
France: 8
Denmark: 6
Italy: 5
Japan: 4
Germany: 4

Who runs them

Shire (7) · Masonic Cancer Center, University of Minnesota (6) · Orchard Therapeutics (3) · Talaris Therapeutics Inc. (3) · Assistance Publique - Hôpitaux de Paris (3) · Magenta Therapeutics, Inc. (2) · Shenzhen Geno-Immune Medical Institute (2) · Children's Hospital of Philadelphia (2)

What is being tested here

Drugs under test in Leukodystrophy, Metachromatic 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	4
Melphalan	1	0	2
Hydroxyurea	1	0	1
Fludarabine	1	0	1
Thiotepa	1	0	1
Cebsulfase alfa	0	0	4
HGT-1111	0	0	3
Cyclophosphamide	0	1	3
Busulfan	0	1	3
Spanlecortemlocel	0	0	2

Drug classes tested here Alkylating agents 1 open

Coming up

1phase 3 study under way

3studies 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 3

Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
Nov 2026Early Check: Expanded Screening in Newborns · RTI International
Oct 2027UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells · Joanne Kurtzberg, MD

Who these studies take

Of the 8 Leukodystrophy, Metachromatic studies recruiting now, 3 (38%) set no upper age limit and 5 (63%) stop at 75 or younger. 7 (88%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (13%) take healthy volunteers.

Recruiting studies that take each age, of 8
Age	Women	Men
10	7 (88%)	7 (88%)
18	7 (88%)	7 (88%)
30	6 (75%)	6 (75%)
40	6 (75%)	6 (75%)
50	6 (75%)	6 (75%)
65	3 (38%)	3 (38%)
75	3 (38%)	3 (38%)
85	3 (38%)	3 (38%)

Across all 49 studies, recruiting or not: 78% stop at 75 or younger and 76% at 65; 96% take children; 4% are for women only and 2% for men only; 10% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 49 of 49. search them.