Amyloidosis, Familial clinical trials
The registry lists 28 studies for Amyloidosis, Familial across more than 10 countries. 10 are recruiting or about to start. 7 of them are mainly about Amyloidosis, Familial (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05. Counting its 3 narrower conditions too, such as Amyloid Neuropathies, Familial: 253 studies, 117 open.
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Status
Who runs them
Alnylam Pharmaceuticals (10) · Eidos Therapeutics, a BridgeBio company (3) · Boston University (2) · Intellia Therapeutics (2) · Brigham and Women's Hospital (1) · Centre Hospitalier Universitaire de Nice (1) · Rennes University Hospital (1) · Ionis Pharmaceuticals, Inc. (1)
What is being tested here
Drugs under test in Amyloidosis, Familial studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
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, phase 3 first
- Jul 2027A Phase 3 Study of NTLA-2001 in ATTRv-PN · Phase 3 · Intellia Therapeutics
- Jun 2027Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers · University of Texas Southwestern Medical Center
Who these studies take
Of the 7 Amyloidosis, Familial studies recruiting now, 4 (57%) set no upper age limit and 0 (0%) stop at 75 or younger. 2 (29%) take children under 18. 1 (14%) are for women only and 0 (0%) for men only; 1 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (29%) | 1 (14%) |
| 18 | 6 (86%) | 5 (71%) |
| 30 | 7 (100%) | 6 (86%) |
| 40 | 7 (100%) | 6 (86%) |
| 50 | 7 (100%) | 6 (86%) |
| 65 | 7 (100%) | 6 (86%) |
| 75 | 7 (100%) | 6 (86%) |
| 85 | 6 (86%) | 5 (71%) |
Across all 28 studies, recruiting or not: 7% stop at 75 or younger and 4% at 65; 7% take children; 4% are for women only and 0% for men only; 7% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 28 of 28. search them.
- MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)RecruitingNCT06128629Phase 326 countries
- Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family RegistryNot yet recruitingNCT059746441 country
- ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)RecruitingNCT0456151812 countries
- Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance ProgramRecruitingNCT050403737 countries
- Non-interventional Study of Patients With Transthyretin (ATTR) AmyloidosisRecruitingNCT064658106 countries
- Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR CarriersRecruitingNCT054895491 country
- To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)Not yet recruitingNCT07116473Phase 3
- Comprehensive Program for Hereditary Transthyretin AmyloidosisRecruitingNCT072132971 country
- ATTR Amyloid Cardiomyopathy: Characterization of Extracellular Vesicles as Potential Disease Stratifiers and Prognostic BiomarkersRecruitingNCT073142681 country
- RWE-based Treatment Patterns and Outcomes in CIDPNot yet recruitingNCT059409221 country
- A Phase 3 Study of NTLA-2001 in ATTRv-PNActive, not recruitingNCT06672237Phase 36 countries
- HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyActive, not recruitingNCT04153149Phase 333 countries
- HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)CompletedNCT03759379Phase 322 countries
- Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or VutrisiranActive, not recruitingNCT058738681 country
- Prospective Evaluation of NfL as a Biomarker in ATTRvActive, not recruitingNCT058798741 country
- Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)WithdrawnNCT04882735Phase 3
- Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid PolyneurophathyWithdrawnNCT04418024Phase 31 country
- Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)Approved for marketingNCT02939820
- APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated AmyloidosisCompletedNCT01960348Phase 321 countries
- Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver TransplantCompletedNCT03862807Phase 37 countries
- Effect of Psyllium (Plantago Ovata) on Digestive Disorders in Familial AmyloidosisWithdrawnNCT04695340Not applicable1 country
- A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A MutationCompletedNCT042014181 country
- Doxycycline and TUDCA in Patients With Transthyretin Amyloid CardiomyopathyCompletedNCT01855360Phase 1/21 country
- ATTR Expanded Access Program (EAP) by IonisApproved for marketingNCT03400098
- A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy SubjectsCompletedNCT02797847Phase 11 country
- ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)CompletedNCT02319005Phase 39 countries
- Safety and Effect of Doxycycline in Patients With AmyloidosisCompletedNCT01677286Phase 21 country
- The Effect of Diflunisal on Familial AmyloidosisCompletedNCT00294671Phase 2/35 countries