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Condition (MeSH)

Amyloidosis

ClinicalTrials.gov lists 617 trials for Amyloidosis or a narrower condition beneath it. Alnylam Pharmaceuticals has the most among industry and government lead sponsors, with 20.

MeSH D000686 Nutritional and Metabolic Diseases C18 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 71 Phase 1/2 Phase 1/2 35 Phase 2 Phase 2 113 Phase 2/3 Phase 2/3 5 Phase 3 Phase 3 68 Phase 4 Phase 4 11 Not applicable Not applicable 100 Observational Observational 208 Phase 1 Phase 1 71 Phase 1/2 Phase 1/2 35 Phase 2 Phase 2 113 Phase 2/3 Phase 2/3 5 Phase 3 Phase 3 68 Phase 4 Phase 4 11 Not applicable Not applicable 100 Observational Observational 208

Trials per phase (log scale)

2 more sections with rows

251 of the 617 trials are tagged with Amyloidosis itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Amyloidosis trials start? (33)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational
  • Other (Expanded access, Unknown)

Top of scale: 52

1991: 1 trials (Phase 2 1) 1992: 1 trials (Phase 2 1) 1996: 2 trials (Phase 2 2) 1997: 5 trials (Phase 2 1, Phase 1/2 1, Phase 1 3) 1998: 6 trials (Phase 3 1, Phase 2 2, Phase 1 3) 1999: 13 trials (Not applicable 1, Phase 3 3, Phase 2 4, Phase 1 5) 2000: 11 trials (Observational 1, Not applicable 2, Phase 3 4, Phase 2 2, Phase 1/2 2) 2001: 6 trials (Observational 1, Phase 2/3 1, Phase 2 2, Phase 1 2) 2002: 5 trials (Not applicable 1, Phase 2 2, Phase 1 2) 2003: 4 trials (Observational 1, Phase 3 1, Phase 2 1, Phase 1 1) 2004: 4 trials (Observational 1, Phase 2 2, Phase 1 1) 2005: 10 trials (Observational 2, Phase 3 2, Phase 2 4, Phase 1/2 2) 2006: 6 trials (Not applicable 2, Phase 3 1, Phase 2/3 1, Phase 2 2) 2007: 13 trials (Other 1, Observational 2, Not applicable 1, Phase 3 2, Phase 2/3 1, Phase 2 4, Phase 1/2 1, Phase 1 1) 2008: 17 trials (Observational 3, Not applicable 1, Phase 3 2, Phase 2/3 1, Phase 2 5, Phase 1/2 2, Phase 1 3) 2009: 16 trials (Other 1, Observational 7, Not applicable 2, Phase 3 2, Phase 2 2, Phase 1/2 1, Phase 1 1) 2010: 9 trials (Observational 3, Not applicable 1, Phase 3 2, Phase 2 3) 2011: 14 trials (Observational 6, Not applicable 3, Phase 3 1, Phase 2 1, Phase 1 3) 2012: 10 trials (Observational 2, Not applicable 2, Phase 3 1, Phase 2 4, Phase 1/2 1) 2013: 17 trials (Observational 2, Not applicable 1, Phase 4 1, Phase 3 2, Phase 2/3 1, Phase 2 4, Phase 1/2 3, Phase 1 3) 2014: 12 trials (Observational 3, Not applicable 1, Phase 3 3, Phase 2 2, Phase 1/2 1, Phase 1 2) 2015: 10 trials (Observational 4, Not applicable 1, Phase 3 3, Phase 2 2) 2016: 21 trials (Observational 6, Not applicable 2, Phase 3 1, Phase 2 7, Phase 1 5) 2017: 21 trials (Observational 5, Not applicable 1, Phase 4 1, Phase 3 1, Phase 2 5, Phase 1/2 4, Phase 1 4) 2018: 25 trials (Observational 5, Not applicable 7, Phase 3 1, Phase 2 5, Phase 1/2 1, Phase 1 6) 2019: 36 trials (Observational 17, Not applicable 7, Phase 3 7, Phase 2 3, Phase 1 2) 2020: 42 trials (Observational 20, Not applicable 9, Phase 4 2, Phase 3 5, Phase 2 2, Phase 1/2 1, Phase 1 3) 2021: 46 trials (Observational 19, Not applicable 6, Phase 4 2, Phase 3 3, Phase 2 8, Phase 1/2 3, Phase 1 5) 2022: 40 trials (Observational 24, Not applicable 6, Phase 3 1, Phase 2 4, Phase 1/2 1, Phase 1 4) 2023: 45 trials (Observational 21, Not applicable 11, Phase 4 1, Phase 3 4, Phase 2 4, Phase 1 4) 2024: 52 trials (Observational 20, Not applicable 12, Phase 4 1, Phase 3 4, Phase 2 6, Phase 1/2 7, Phase 1 2) 2025: 46 trials (Observational 19, Not applicable 7, Phase 4 1, Phase 3 3, Phase 2 11, Phase 1/2 1, Phase 1 4)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 44
2025 46
2024 52
2023 45
2022 40
2021 46
2020 42
2019 36
2018 25
2017 21

Showing 10 of 33 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 53 trials starting in 2026 and about 47 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 80 trials still running at the 2026-10-09 snapshot; registered median of 34 months across 319 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Amyloidosis trials in? (10)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 71
Phase 1/2 35
Phase 2 113
Phase 2/3 5
Phase 3 68
Phase 4 11
Not applicable 100
Observational 208
Expanded access 5
Unknown 1

How many Amyloidosis trials are recruiting? (11)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 27
Recruiting 133
Enrolling by invitation 11
Active, not recruiting 35
Available 1

Showing 5 of 11 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Amyloidosis trials? (7)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Alnylam Pharmaceuticals 20
Pfizer 12
Eidos Therapeutics, a BridgeBio company 10
GlaxoSmithKline 9
Ionis Pharmaceuticals, Inc. 8

Showing 5 of 7 sponsors.

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Which drugs are being tested for Amyloidosis? (7)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Melphalan 32
Cytoxan 30
POMALIDOMIDE 10
Acoramidis 10
Patisiran 9

Showing 5 of 7 drugs.

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Interventions as named in the registry (8)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Dexamethasone/Dexamethasone acetate 59
Bortezomib SC 28
Filgrastime (G-CSF) 18
Peripheral blood stem cell transplantation 16
Daratumumab SC 16

Showing 5 of 8 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Amyloidosis trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Cardiac failure 807 23
Pneumonia 338 34
Cardiac failure congestive 299 18
Atrial fibrillation 282 27
Cardiac failure acute 234 15

Showing 5 of 10 adverse events.

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Which publications cover Amyloidosis? (338)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Inhaled xenon modulates microglia and ameliorates disease in mouse models of amyloidosis and tauopathy. Science translational medicine 2025 Yes
Predictors of proteinuria, amyloidosis and kidney failure in familial Mediterranean fever: data from the International AIDA Network Registry. Rheumatology (Oxford, England) 2025 Yes
Atrial amyloidosis identified by biopsy in atrial fibrillation: prevalence and clinical presentation. European heart journal 2025 Yes
The Amyloidosis Intersection: Dual Amyloid Types in a Single Host. European journal of haematology 2025 Yes
Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy. The New England journal of medicine 2024 Yes

Showing 5 of 338 publications.

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Prevalence (6)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Amyloidosis Point prevalence Unknown Worldwide Not stated Exact
Amyloidosis Point prevalence Unknown Worldwide Not stated Unspecified
Amyloidosis Point prevalence 1-9 / 100 000 Korea, Republic of Not stated Unspecified
Amyloidosis Point prevalence 1-9 / 100 000 Korea, Republic of Not stated Exact
Amyloidosis Point prevalence Unknown Worldwide Not stated Exact

Showing 5 of 6 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Amyloidosis 69 Exact Orphanet Rare Disease Ontology (ORDO)
Amyloidosis 69 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (4)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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