Niemann-Pick Disease, Type A clinical trials

The registry lists 10 studies for Niemann-Pick Disease, Type A across more than 10 countries. 1 are recruiting or about to start. 6 of them are mainly about Niemann-Pick Disease, Type A (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.

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Status

Completed: 6
Active, not recruiting: 2
Recruiting: 1
Approved for marketing: 1

Phase

Phase 2: 4
Phase 1: 3
Phase 3: 1

Where

United States: 8
Italy: 4
Brazil: 4
Germany: 4
France: 4
Belgium: 3

Who runs them

Genzyme, a Sanofi Company (3) · Sanofi (2) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (2) · RTI International (1) · Hubei Cancer Hospital (1) · Joanne Kurtzberg, MD (1)

Coming up

0phase 3 studies under way

2studies 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 2

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 1 Niemann-Pick Disease, Type A studies recruiting now, 0 (0%) set no upper age limit and 1 (100%) stop at 75 or younger. 1 (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 1
Age	Women	Men
10	1 (100%)	1 (100%)
18	1 (100%)	1 (100%)
30	0 (0%)	0 (0%)
40	0 (0%)	0 (0%)
50	0 (0%)	0 (0%)
65	0 (0%)	0 (0%)
75	0 (0%)	0 (0%)
85	0 (0%)	0 (0%)

Across all 10 studies, recruiting or not: 40% stop at 75 or younger and 40% at 65; 70% take children; 0% are for women only and 0% for men only; 30% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 10 of 10. search them.