ATTR Amyloidosis trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in ATTR Amyloidosis. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Upcoming readout windows
next 180 days · by month2 studies across 2 companies, in the next 180 days.
November 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| AZN | AstraZeneca | OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR AmyloidosisNCT06355934observationalHealth Care Resource Utilization (HCRU) - Outpatient visits | — | Recruiting | 2026-11-02 | in 74 d | 2026-08-11 |
December 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| PFE | Pfizer | Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid CardiomyopathyNCT04801329observationalThe incidence of Adverse Events (AEs) | — | Active, not recruiting | 2026-12-31 | in 133 d | 2026-03-30 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Every registered study in ATTR Amyloidosis
most recently updated first57 studies match.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| AZN | AstraZeneca | — | Non-interventional Study of Patients With Transthyretin (ATTR) AmyloidosisNCT06465810observationalDemographic characteristics (overall and in patients initiating a treatment with eplontersen) | — | Recruiting | Drug: Treatment of transthyretin (ATTR) amyloidosis in observational study setting | Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR | 1,850 | 2024-06-25 | 2031-12-29 | in 1,957 d | 2026-08-19 |
| NVO | Novo Nordisk A/S | — | CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) AmyloidosisNCT07207811Composite Outcome of cardiovascular (CV) deaths and recurrent CV events (CV hospitalisations and urgent heart failure [HF] visits) | Phase 3 | Recruiting | Drug: NNC6019-0001, Placebo (NNC6019-0001) | Transthyretin Amyloid Cardiomyopathy (ATTR CM) | 1,280 | 2025-10-02 | 2029-06-29 | in 1,044 d | 2026-08-19 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT07052903Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits) | Phase 3 | Recruiting | Drug: Nucresiran, Sterile Normal Saline (0.9% NaCl) | Transthyretin Amyloidosis With Cardiomyopathy | 1,750 | 2025-07-02 | 2030-05-28 | in 1,377 d | 2026-08-14 |
| AZN | AstraZeneca | — | OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR AmyloidosisNCT06355934observationalHealth Care Resource Utilization (HCRU) - Outpatient visits | — | Recruiting | Other: no intervention | ATTR Amyloidosis | 52,121 | 2023-08-21 | 2026-11-02 | in 74 d | 2026-08-11 |
| AZN | AstraZeneca | — | Implementation of Standardized Early Identification and Diagnosis for Transthyretin Amyloidosis (ATTR) in High-Risk PopulationsNCT07338942Proportion of patients diagnosed with ATTR in high-risk population. | Not applicable | Active, not recruiting | Behavioral: implementation of standard early identification and diagnosis of ATTR | ATTR-CM | 4,000 | 2026-04-21 | 2027-11-20 | in 457 d | 2026-08-07 |
| NTLA | Intellia Therapeutics | $1.6B | Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001NCT05697861observationalIncidence of Treatment-related SAEs and Protocol-specified AESIs | — | Enrolling by invitation | — | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis | 72 | 2023-07-10 | Mar 2038 | ≤ 4,241 d | 2026-08-03 |
| AZN | AstraZeneca | — | Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CMNCT07690436observationalComparison of per-participant confidence scores distributions between ATTR-CM cases and HF controls without ATTR-CM | — | Not yet recruiting | — | Transthyretin Amyloid Cardiomyopathy | 500 | 2026-07-02 | 2027-06-03 | in 287 d | 2026-07-08 |
| AZN | AstraZeneca | — | The Eplontersen Pregnancy and Lactation Outcomes StudyNCT07205666observationalMajor congenital malformations | — | Recruiting | — | Transthyretin Amyloidosis | 10 | 2025-11-30 | 2035-12-30 | in 3,419 d | 2026-06-22 |
| AZN | AstraZeneca | — | Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired SubpopulationsNCT07654855observationalDemographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment | — | Not yet recruiting | Other: None ( observational study ) | Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy | 320 | 2026-09-01 | 2032-01-29 | in 1,988 d | 2026-06-17 |
| AZN | AstraZeneca | — | EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid CardiomyopathyNCT06194825Percent change of serum TTR concentration from baseline | Phase 3 | Active, not recruiting | Drug: Eplontersen, Placebo | Transthyretin Amyloid Cardiomyopathy | 64 | 2023-12-01 | 2026-05-13actual | — | 2026-06-09 |
| AZN | AstraZeneca | — | Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in RussiaNCT06414746observationalTo define the prevalence of ATTR PN in patients diagnosed with CTS and having bilateral involvement in routine clinical practice in the Russian Federation. | — | Completed | — | Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis | 721 | 2023-12-29 | 2025-06-12actual | — | 2026-06-04 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)NCT04561518observationalIncidence of Adverse Events | — | Recruiting | — | Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis | 1,500 | 2020-11-23 | 2033-12-31 | in 2,690 d | 2026-05-29 |
| PFE | Pfizer | $155.3B | A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in GermanyNCT07494656observationalOverall Survival in Individuals With Incident Transthyretin Amyloid Cardiomyopathy (ATTR-CM), Overall and in Those With at Least One Tafamidis Prescription | — | Active, not recruiting | Other: tafamidis 61mg (based on prescription data) | ATTR-CM (Transthyretin Amyloid Cardiomyopathy) | 1 | 2026-04-02 | 2026-06-30 | — | 2026-05-22 |
| PFE | Pfizer | $155.3B | Vyndaqel Capsules Special Investigation (ATTR-CM)NCT04108091results2026-05-18observationalNumber of Participants With Adverse Drug Reactions | — | Completed | Drug: Treatment for TTR amyloidosis, Treatment for TTR amyloidosis | Transthyretin (TTR) Amyloid Cardiomyopathy | 1,214 | 2019-10-03 | 2025-03-27actual | — | 2026-05-18 |
| PFE | Pfizer | $155.3B | Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid CardiomyopathyNCT06393465observationalRate of neurologic disease progression | — | Completed | Drug: Tafamidis | Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) | 50 | 2024-06-15 | 2026-04-07actual | — | 2026-04-29 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World SettingNCT07358078observationalNew York Heart Association (NYHA) Class | — | Recruiting | — | Transthyretin Amyloidosis With Cardiomyopathy | 2,000 | 2026-01-09 | 2030-10-23 | in 1,525 d | 2026-04-24 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)NCT03997383results2023-10-18Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) | Phase 3 | Completed | Drug: Placebo, Patisiran | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 360 | 2019-09-04 | 2022-06-20actual | — | 2026-04-20 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)NCT05667493Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) | Phase 3 | Enrolling by invitation | Drug: Eplontersen | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | 1,400 | 2022-11-30 | Aug 2029 | ≤ 1,107 d | 2026-04-17 |
| NVO | Novo Nordisk A/S | — | A Research Study to Look at the Distribution and Effects of Coramitug on Amyloid Deposits in Heart Tissue Using PET/CT Imaging in People With ATTR Amyloidosis.NCT07448623Mean Standard Uptake Value Ratio (SUVRmean) in myocardium | Phase 1 | Recruiting | Drug: Coramitug | Transthyretin Amyloid Cardiomyopathy | 14 | 2026-03-02 | 2027-05-30 | in 283 d | 2026-04-13 |
| PFE | Pfizer | $155.3B | Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid CardiomyopathyNCT04801329observationalThe incidence of Adverse Events (AEs) | — | Active, not recruiting | Drug: Vyndamax (tafamidis 61mg) | ATTR-CM (Transthyretin Amyloid Cardiomyopathy) | 110 | 2021-06-29 | 2026-12-31 | in 133 d | 2026-03-30 |
| NTLA | Intellia Therapeutics | $1.6B | MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)NCT06128629Composite outcome of cardiovascular (CV) mortality and CV events | Phase 3 | Recruiting | Biological: NTLA-2001 · Drug: Placebo | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 1,200 | 2023-12-13 | Dec 2027 | ≤ 498 d | 2026-03-27 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT06679946Frequency of Adverse Events (AEs) | Phase 3 | Enrolling by invitation | Drug: Vutrisiran | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 700 | 2024-12-03 | 2028-03-31 | in 589 d | 2026-02-20 |
| NVO | Novo Nordisk A/S | — | A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) AmyloidosisNCT05442047Change in 6-minute walk test (6-MWT) | Phase 2 | Completed | Drug: NNC6019-0001, Placebo (NNC6019-0001) | Transthyretin Amyloid Cardiomyopathy (ATTR CM) | 105 | 2022-08-02 | 2025-02-17actual | — | 2026-02-17 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy SubjectsNCT05661916Frequency of Adverse Events | Phase 1 | Completed | Drug: ALN-TTRSC04, Placebo | Transthyretin-Mediated Amyloidosis | 96 | 2023-01-16 | 2025-09-30actual | — | 2026-02-17 |
| NTLA | Intellia Therapeutics | $1.6B | Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)NCT04601051Number of Participants with Treatment-Emergent Adverse Events | Phase 1 | Completed | Biological: NTLA-2001 | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis | 72 | 2020-11-05 | 2025-09-12actual | — | 2026-01-30 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)NCT03759379results2022-08-11Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)] | Phase 3 | Completed | Drug: Patisiran, Vutrisiran | Amyloidosis, Hereditary, Transthyretin Amyloidosis | 164 | 2019-02-14 | 2020-11-10actual | — | 2026-01-12 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT04153149results2025-10-21Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population | Phase 3 | Active, not recruiting | Drug: Vutrisiran, Sterile Normal Saline (0.9% NaCl) | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 655 | 2019-11-26 | 2024-05-08actual | — | 2026-01-12 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid PolyneuropathyNCT05071300Change From Baseline in Platelet Count | Phase 3 | Active, not recruiting | Drug: Eplontersen | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | 151 | 2022-01-04 | Aug 2029 | ≤ 1,107 d | 2025-12-04 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyNCT06073574observationalChange in the Percent of ECV in Participants Receiving ION-682884 vs. Placebo According to ION-682884-CS2 Treatment Groups | — | Active, not recruiting | Diagnostic test: Cardiac MRI | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | 150 | 2021-03-31 | Mar 2026 | — | 2025-11-28 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | The CARDIO-TTRansform Scintigraphy Sub-studyNCT06073587observationalChanges From Baseline in Perugini Grading Score From Scintigraphy Scan Images at Week 140 | — | Active, not recruiting | Diagnostic test: Scintigraphy scan | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | 150 | 2023-04-04 | Mar 2026 | — | 2025-11-28 |
| NVO | Novo Nordisk A/S | — | A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin AmyloidosisNCT06260709Number of Treatment Emergent Adverse Events | Phase 2 | Active, not recruiting | Drug: NNC6019-0001 | Transthyretin Amyloid Cardiomyopathy (ATTR CM) | 80 | 2024-02-20 | 2028-02-29 | in 558 d | 2025-11-25 |
| PFE | Pfizer | $155.3B | A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in IndiaNCT06086353observationalNumber of Adverse events with Tafamidis | — | Completed | Drug: Tafamidis | Transthyretin Amyloid Cardiomyopathy | 6 | 2024-03-25 | 2025-06-06actual | — | 2025-11-20 |
| PFE | Pfizer | $155.3B | A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)NCT06321523observationalNumber of Participants With Change From Baseline in New York Heart Association (NYHA) Classification | — | Completed | Drug: Tafamidis 61 milligrams | Transthyretin Amyloid Cardiomyopathy | 13 | 2024-03-19 | 2025-10-13actual | — | 2025-11-20 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)NCT04136171Composite Outcome of Cardiovascular (CV) Mortality and Recurrent CV Clinical Events up to Week 140 | Phase 3 | Active, not recruiting | Drug: Eplontersen, Placebo | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | 1,438 | 2020-03-13 | Apr 2026 | — | 2025-08-08 |
| AZN | AstraZeneca | — | hATTR Polyneuropathy in RussiaNCT06365593observationalIn order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Mean age at the hATTR-PN or CIAP diagnosis information will be collected | — | Completed | — | Hereditary Transthyretin Amyloidosis | 100 | 2023-12-29 | 2024-05-30actual | — | 2025-05-25 |
| PFE | Pfizer | $155.3B | Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)NCT02146378results2025-02-03observationalPercentage of Participants With ADRs in This Study | — | Completed | Drug: Vyndaqel | Transthyretin Familial Amyloid Polyneuropathy | 500 | 2014-01-10 | 2023-03-10actual | — | 2025-02-03 |
| PFE | Pfizer | $155.3B | Long-term Safety of Tafamidis in Subjects With Transthyretin CardiomyopathyNCT02791230results2025-02-03Time to All-Cause Mortality: Cohort A | Phase 3 | Completed | Drug: Tafamidis | Transthyretin (TTR) Amyloid Cardiomyopathy | 1,733 | 2016-06-13 | 2023-10-26actual | — | 2025-02-03 |
| PFE | Pfizer | $155.3B | A Retrospective Validation Study To Identify Chart-Based Clinical Diagnosis Of Wild-Type Transthyretin Amyloid Cardiomyopathy (Attrwt-CM) And Non-Amyloid Heart Failure Among Patients With Heart Failure (HF).NCT06029452results2025-01-03observationalNumber of Participants According to Clinical Diagnosis Predicted Using the Machine Learning (ML) Algorithm | — | Completed | Other: Machine learning algorithm | ATTR-CM | 558 | 2023-09-01 | 2023-11-14actual | — | 2025-01-03 |
| PFE | Pfizer | $155.3B | A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)NCT04814186results2024-12-18Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) | Phase 4 | Completed | Drug: Tafamidis | Transthyretin Amyloid Cardiomyopathy | 53 | 2021-07-22 | 2023-10-16actual | — | 2024-12-18 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid PolyneuropathyNCT04136184results2024-12-10Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66 | Phase 3 | Completed | Drug: Inotersen, Eplontersen | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | 168 | 2019-12-11 | 2023-04-11actual | — | 2024-12-13 |
| PFE | Pfizer | $155.3B | Transthyretin Amyloidosis Outcome Survey (THAOS)NCT00628745results2024-11-22observationalNumber of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | — | Completed | Other: None. Observational Study. | Transthyretin Gene Mutations, Transthyretin Amyloidosis | 6,718 | 2008-01-04 | 2023-06-16actual | — | 2024-11-22 |
| AZN | AstraZeneca | — | A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese VolunteersNCT06527755PK parameters: Maximum Observed Concentration(Cmax) | Phase 1 | Completed | Drug: Eplontersen Solution for Injection | Transthyretin-mediated Amyloidosis | 12 | 2024-08-06 | 2024-09-11actual | — | 2024-11-21 |
| PFE | Pfizer | $155.3B | Global Prevalence of ATTR-CM in Participants With HFpEFNCT04424914results2024-07-19Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants | Not applicable | Terminatedsponsor's stated reason: Sponsor terminated study early to not prolong timelines and to enable sharing of data collected which has potential to better support understanding of the prevalence of ATTR-CM in patients with HFpEF. Decision was not based on any safety findings. | Diagnostic test: Scintigraphy | Transthyretin Amyloid Cardiomyopathy, Heart Failure With Preserved Ejection Fraction | 347 | 2020-12-30 | 2023-06-02actual | — | 2024-07-19 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)NCT02939820expanded access | — | Approved for marketing | Drug: patisiran (ALN-TTR02) | TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related | — | — | — | — | 2024-05-20 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated AmyloidosisNCT01960348results2018-09-06Modified Neuropathy Impairment Score +7 (mNIS+7) | Phase 3 | Completed | Drug: patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) | TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related | 225 | Nov 2013 | Aug 2017actual | — | 2024-04-22 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver TransplantNCT03862807results2021-12-21Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) | Phase 3 | Completed | Drug: Patisiran | Amyloidosis, Familial, Transthyretin Amyloidosis | 24 | 2019-03-27 | 2020-10-06actual | — | 2024-04-22 |
| PFE | Pfizer | $155.3B | Prevalence and Characteristics of Transthyretin Amyloidosis in Patients With Left Ventricular Hypertrophy of Unknown EtiologyNCT03842163results2024-02-12observationalPercentage of Participants With Cardiac Fixation at the Radionuclide Bone Scintigraphy and/or Single Photon Emission Computed Tomography (SPECT): FAS1 | — | Completed | Diagnostic test: Diagnosis of TTR amyloidosis cardiomyopathy | Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) | 812 | 2018-07-09 | 2022-06-08actual | — | 2024-02-12 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)NCT02175004results2023-02-09Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Phase 3 | Completed | Drug: Inotersen | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | 135 | 2014-06-26 | 2020-09-11actual | — | 2023-11-18 |
| PFE | Pfizer | $155.3B | Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin AmyloidosisNCT00925002results2021-07-29Val30Met Group: Neuropathy Impairment Score Lower Limb (NIS-LL) Score at Baseline | Phase 3 | Completed | Drug: Tafamidis | ATTR-PN | 93 | 2009-08-05 | 2020-07-08actual | — | 2021-07-29 |
| PFE | Pfizer | $155.3B | Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid CardiomyopathyNCT00935012results2021-01-05Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 12 | Phase 3 | Completed | Drug: tafamidis | ATTR-CM, TTR-CM | 31 | 2009-09-30 | 2019-11-20actual | — | 2021-01-05 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | Efficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyNCT01737398results2019-01-23Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 | Phase 2/3 | Completed | Drug: Inotersen, Placebo | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | 173 | 2013-03-15 | 2017-03-03actual | — | 2019-07-17 |
| PFE | Pfizer | $155.3B | Safety and Efficacy of Tafamidis in Patients With Transthyretin CardiomyopathyNCT01994889results2019-04-03Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations | Phase 3 | Completed | Drug: Tafamidis, Tafamidis, Placebo | Transthyretin (TTR) Amyloid Cardiomyopathy | 441 | 2013-12-09 | 2018-02-07actual | — | 2019-04-24 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantNCT02595983results2019-03-28Percentage Change From Baseline in Serum TTR at Month 6 | Phase 2 | Completed | Drug: Revusiran | Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies | 12 | Oct 2015 | 2017-02-06actual | — | 2019-03-28 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)NCT02319005results2018-07-186 Minute Walk Distance (6-MWD) | Phase 3 | Completed | Drug: Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl) | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis | 206 | Dec 2014 | 2017-03-30actual | — | 2018-07-18 |
| PFE | Pfizer | $155.3B | The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M TransthyretinNCT01435655results2015-09-09Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | Phase 3 | Completed | Drug: tafamidis | Transthyretin Familial Amyloid Polyneuropathy | 10 | Nov 2011 | Feb 2014actual | — | 2015-09-09 |
| ALNY | Alnylam Pharmaceuticals | $30.6B | A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy VolunteersNCT02053454The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation | Phase 1 | Completed | Drug: patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) | Transthyretin (TTR)-Mediated Amyloidosis | 12 | Jan 2014 | Mar 2014actual | — | 2015-05-25 |
| PFE | Pfizer | $155.3B | An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid PolyneuropathyNCT00791492results2012-12-17Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 | Phase 2/3 | Completed | Drug: Fx-1006A | Familial Amyloid Polyneuropathy, ATTR-PN | 86 | Jul 2008 | Aug 2008actual | — | 2012-12-17 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
How ATTR Amyloidosis is matched — 23 exact registry spellings
A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.
- Transthyretin Amyloid Cardiomyopathy
- Transthyretin Amyloidosis
- ATTR-CM
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- ATTR Amyloidosis
- Amyloidosis, Hereditary, Transthyretin-Related
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Transthyretin Amyloid Cardiomyopathy (ATTR CM)
- Transthyretin Amyloidosis With Cardiomyopathy
- ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
- ATTR-PN
- Hereditary Transthyretin Amyloidosis
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Transthyretin
- Transthyretin (TTR)-Mediated Amyloidosis
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
- Transthyretin Familial Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
- Transthyretin-Mediated Amyloidosis
- Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
- Wild-Type Transthyretin Cardiac Amyloidosis
Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “ATTR Amyloidosis” (a text search, wider than this page)