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Amyloidosis, Hereditary, Transthyretin-Related clinical trials

The registry lists 82 studies for Amyloidosis, Hereditary, Transthyretin-Related across more than 10 countries. 28 are recruiting or about to start. 64 of them are mainly about Amyloidosis, Hereditary, Transthyretin-Related (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-13.

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Status

Completed
34
Recruiting
22
Active, not recruiting
7
Withdrawn
5
Status unknown
4
Terminated
3

Phase

Phase 3
14
Phase 2
6
Phase 1
5
Early Phase 1
4
Phase 4
4

Where

United States
37
France
19
Italy
18
Germany
17
Spain
16
Portugal
15

Who runs them

Alnylam Pharmaceuticals (12) · AstraZeneca (6) · Eidos Therapeutics, a BridgeBio company (6) · The Cleveland Clinic (4) · Pfizer (4) · Akcea Therapeutics (3) · Steen Hvitfeldt Poulsen (2) · CENTOGENE GmbH Rostock (2)

Who develops what

Save this set

Every programme with a company-led study here in the last two years, whatever its size: 1 programme from 1 company, 1 with this as their studies’ main aim; 1 with a live phase 3 study; 0 primary completions in the next 12 months.

Comparator & background therapies

What other companies’ phase 2+ studies give as the comparator or the background here (its leading trial sponsor and partners left out): a sign of what new drugs are measured against, not by itself the standard of care, its prescriptions or its market.

None given by two companies or more yet.

Programmes

“Meets, by arms” reads each study’s arms: vs its arm against the therapy’s; + X vs a combination against it; added to it plus the therapy against the therapy; studied with given together only. The next primary completion is the next phase-3 study’s when there is one.

The 1 programme — sign in to see the whole list, free: filter by phase and aim, sort by late development, direct comparisons or the next primary completion.

Primary completion in the next 12 months (0)

Live phase 2 and 3 studies of these programmes. Primary completion is when the main measure’s data collection ends, as the registry states it (most are estimates); results come later, if they are announced.

None in the next 12 months.

Read from ClinicalTrials.gov, industry-led studies. Main aim: this is the study’s judged main condition; named, not the main aim: the sponsor names it, the study is about something else; registry mapping only: only the registry’s mapping put it here. Leading trial sponsor is the company leading the most industry studies of a drug, a measure of trial activity, not of rights. US and EU rights, and Approved: from the regulators’ records (FDA’s Drugs@FDA via openFDA; EMA’s medicines data) — the holder of a marketed or authorised original, generics and other companies’ versions; not rights elsewhere, nor approval for this condition. Phase 4 trial recorded, where neither lists it: a phase 4 study exists somewhere. Not read yet: labels by indication, exclusivity, licences, line of therapy, academic comparators, sales.

What is being tested here

Drugs under test in Amyloidosis, Hereditary, Transthyretin-Related studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Acoramidis356
Vutrisiran134
NTLA-2001122
Patisiran034
Doxycycline003
Ursodoxicoltaurine002
Tafamidis001
Flutemetamol001
Tolcapone001
Revusiran011

Coming up

6phase 3 studies under way
6studies 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 6, phase 3 first

Who these studies take

Of the 22 Amyloidosis, Hereditary, Transthyretin-Related studies recruiting now, 15 (68%) set no upper age limit and 1 (5%) stop at 75 or younger. 2 (9%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 4 (18%) take healthy volunteers.

Recruiting studies that take each age, of 22
AgeWomenMen
102 (9%)2 (9%)
1817 (77%)17 (77%)
3019 (86%)19 (86%)
4020 (91%)20 (91%)
5020 (91%)20 (91%)
6522 (100%)22 (100%)
7522 (100%)22 (100%)
8518 (82%)18 (82%)

Across all 82 studies, recruiting or not: 2% stop at 75 or younger and 1% at 65; 9% take children; 1% are for women only and 0% for men only; 13% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Amyloidosis, Hereditary, Transthyretin-Related studies take

Of 25 observational studies under Amyloidosis, Hereditary, Transthyretin-Related, half reached primary completion within 93 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Observational93 mo29–97 · 25 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 82. Browse all 82 → or search them.