Wolman Disease clinical trials

The registry lists 33 studies for Wolman Disease across more than 10 countries. 5 are recruiting or about to start. 19 of them are mainly about Wolman Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-07-28.

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Status

Completed: 15
Terminated: 5
Status unknown: 5
Recruiting: 4
Withdrawn: 1
Active, not recruiting: 1

Phase

Phase 2: 8
Phase 1: 3
Phase 3: 3

Where

United States: 23
United Kingdom: 9
Italy: 8
France: 7
Canada: 5
Czechia: 4

Who runs them

Alexion Pharmaceuticals, Inc. (12) · Masonic Cancer Center, University of Minnesota (4) · University of California, San Francisco (2) · CENTOGENE GmbH Rostock (1) · University of Aarhus (1) · Massachusetts General Hospital (1) · Centre Hospitalier Universitaire de Liege (1) · RTI International (1)

What is being tested here

Drugs under test in Wolman Disease 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
Laronidase	1	0	1
Sebelipase alfa	0	2	6
Busulfan	0	1	2
Cyclophosphamide	0	1	2
Alemtuzumab	0	0	2
Antilymphocyte Serum	0	1	1
Clofarabine	0	0	1
Melphalan	0	0	1
Cyclosporine	0	0	1
Mycophenolic Acid	0	0	1

Drug classes tested here Alkylating agents 3

Coming up

0phase 3 studies 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

Nov 2026Early Check: Expanded Screening in Newborns · RTI International

Who these studies take

Of the 4 Wolman Disease studies recruiting now, 1 (25%) set no upper age limit and 3 (75%) stop at 75 or younger. 2 (50%) take children under 18. 2 (50%) are for women only and 0 (0%) for men only; 1 (25%) take healthy volunteers.

Recruiting studies that take each age, of 4
Age	Women	Men
10	2 (50%)	2 (50%)
18	3 (75%)	2 (50%)
30	3 (75%)	2 (50%)
40	3 (75%)	2 (50%)
50	4 (100%)	2 (50%)
65	1 (25%)	1 (25%)
75	1 (25%)	1 (25%)
85	1 (25%)	1 (25%)

Across all 33 studies, recruiting or not: 58% stop at 75 or younger and 55% at 65; 76% take children; 12% are for women only and 3% for men only; 24% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 33 of 33. search them.