- Conditions
- Hereditary Amyloidosis, Transthyretin-Related
- Interventions
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Diagnostic Test
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 30 Years to 90 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
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Showing 25–48
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- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
- Interventions
- Not listed
- Lead sponsor
- xCures
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Los Altos, California
- Conditions
- Transthyretin Amyloidosis
- Interventions
- Cardiac MRI
- Diagnostic Test
- Lead sponsor
- The Christ Hospital
- Other
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 131 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Amyloidogenic Transthyretin (ATTR) Amyloidosis
- Interventions
- PRX004
- Drug
- Lead sponsor
- Prothena Biosciences Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Rochester, Minnesota • Portland, Oregon + 1 more
- Conditions
- Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis
- Interventions
- Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 206 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 41
- States / cities
- Bakersfield, California • Beverly Hills, California • La Mesa, California + 30 more
- Conditions
- Amyloid, Amyloidosis, Spinal Stenosis, Transthyretin Amyloidosis, Primary Amyloidosis of Light Chain Type
- Interventions
- Biopsy
- Procedure
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 200 participants
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
- Interventions
- Not listed
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2033
- U.S. locations
- 13
- States / cities
- La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more
- Conditions
- Amyloid Cardiomyopathy, Transthyretin-Related
- Interventions
- Acoramidis (AG10)
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 389 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 26
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
- Interventions
- Tc99-PYP or Tc99-HDMP Scan
- Diagnostic Test
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 1,663 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2028
- U.S. locations
- 6
- States / cities
- Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
- Conditions
- Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
- Interventions
- tafamidis
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Interventions
- Eplontersen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 151 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 8
- States / cities
- Scottsdale, Arizona • Indianapolis, Indiana • Baltimore, Maryland + 5 more
- Conditions
- Aortic Stenosis, Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
- Interventions
- 99mTc-PYP
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 75 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Tafamidis, Placebo
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 441 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 50
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 30 Years to 80 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Transthyretin Cardiac Amyloidosis
- Interventions
- Tafamidis 61 MG
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 4
- States / cities
- Beverly Hills, California • New York, New York • Cleveland, Ohio + 1 more
- Conditions
- Familial Amyloid Polyneuropathy, Familial Amyloidosis
- Interventions
- diflunisal, placebo
- Drug · Other
- Lead sponsor
- Boston University
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 130 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2012
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Rochester, Minnesota • New York, New York
- Conditions
- Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
- Interventions
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
- Drug
- Lead sponsor
- AstraZeneca
- Industry
- Eligibility
- 18 Years to 130 Years
- Enrollment
- 1,850 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 33
- States / cities
- La Jolla, California • Los Angeles, California • San Francisco, California + 24 more
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Revusiran (ALN-TTRSC)
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • New York, New York • Cleveland, Ohio
- Conditions
- Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
- Interventions
- Beta blocker
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 65 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Amyloidosis, Hereditary, Transthyretin Amyloidosis
- Interventions
- Patisiran, Vutrisiran
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 164 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 15
- States / cities
- La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 12 more
- Conditions
- Hereditary Transthyretin Amyloidosis With Polyneuropthy
- Interventions
- TEGSEDI
- Drug
- Lead sponsor
- Akcea Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Rosedale, New York
- Conditions
- Familial ATTR-CM (ATTRm-CM, or FAC), Wild-type ATTR-CM (ATTRwt-CM)
- Interventions
- AG10, Placebo Oral Tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 49 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 13
- States / cities
- Beverly Hills, California • Palo Alto, California • San Francisco, California + 9 more
- Conditions
- Transthyretin Amyloid Cardiomyopathy (ATTR CM)
- Interventions
- NNC6019-0001, Placebo (NNC6019-0001)
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1,280 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 63
- States / cities
- Phoenix, Arizona • La Jolla, California • Los Angeles, California + 49 more
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- Interventions
- Eplontersen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 1,438 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 51
- States / cities
- Phoenix, Arizona • La Jolla, California • Los Angeles, California + 39 more