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Homozygous Familial Hypercholesterolemia clinical trials

The registry lists 68 studies for Homozygous Familial Hypercholesterolemia across more than 10 countries. 13 are recruiting or about to start. 59 of them are mainly about Homozygous Familial Hypercholesterolemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-03.

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Status

Completed
41
Recruiting
12
Active, not recruiting
5
Terminated
4
Withdrawn
3
Status unknown
2

Phase

Phase 3
31
Phase 2
20
Phase 1
2
Early Phase 1
1

Where

United States
36
Canada
28
Netherlands
25
South Africa
19
Italy
15
France
13

Who runs them

Aegerion Pharmaceuticals, Inc. (6) · Novartis Pharmaceuticals (6) · Regeneron Pharmaceuticals (5) · Kastle Therapeutics, LLC (4) · REGENXBIO Inc. (3) · Arrowhead Pharmaceuticals (3) · Beijing Suncadia Pharmaceuticals Co., Ltd (3) · Amgen (3)

What is being tested here

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

DrugOpenPhase 3All
Inclisiran256
SHR-1918113
Zodasiran122
EDP167101
BMS201038056
Evinacumab045
Mipomersen024
Evolocumab033
Rosuvastatin033
Tafolecimab022

Drug classes tested here PCSK9 inhibitors 6 · Statins 3

Coming up

5phase 3 studies under way
7studies finish main data collection within a year1 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 7, phase 3 first

Who these studies take

Of the 12 Homozygous Familial Hypercholesterolemia studies recruiting now, 6 (50%) set no upper age limit and 5 (42%) stop at 75 or younger. 8 (67%) 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 12
AgeWomenMen
105 (42%)5 (42%)
1810 (83%)10 (83%)
309 (75%)9 (75%)
409 (75%)9 (75%)
509 (75%)9 (75%)
658 (67%)8 (67%)
758 (67%)8 (67%)
857 (58%)7 (58%)

Across all 68 studies, recruiting or not: 22% stop at 75 or younger and 19% at 65; 56% take children; 0% are for women only and 0% for men only; 1% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Homozygous Familial Hypercholesterolemia studies take

Of 20 phase 3 interventional studies under Homozygous Familial Hypercholesterolemia, half reached primary completion within 22 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 322 mo16–34 · 20 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 68. Browse all 68 → or search them.