Clinical trials: ATTR
Transthyretin-Mediated Amyloidosis
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
- Trial name
- NCT05661916
- Testing
- ALN-TTRSC04
- Where
- United Kingdom
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Healthy Volunteers, hATTR Amyloidosis
Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
To evaluate the safety and tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of single and multiple doses of Eplontersen administered subcutaneously to healthy volunteers and patients with Hereditary…
- Trial name
- NCT03728634
- Testing
- ION-682884, Vitamin A
- Where
- Canada
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
TTR-mediated Amyloidosis
Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
- Trial name
- NCT01559077
- Testing
- ALN-TTR02, Sterile Normal Saline (0.9% NaCl)
- Where
- 2 sites in United Kingdom
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR…
- Trial name
- NCT06360289
- Testing
- Standard of Care
- Where
- France
- Volunteers
- 346 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Amyloid Cardiomyopathy, Transthyretin-Related
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
- Trial name
- NCT03294707
- Testing
- AG10 oral tablet
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin-mediated Amyloidosis
A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
This is a Phase 1 study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of eplontersen following single dosing in healthy Chinese participants.
- Trial name
- NCT06527755
- Testing
- Eplontersen Solution for Injection
- Where
- China
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Familial Amyloid Polyneuropathy (FAP)
Study of SOM0226 in Familial Amyloid Polyneuropathy
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
- Trial name
- NCT02191826
- Testing
- SOM0226
- Where
- Spain
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- SOM Innovation Biotech SA
Healthy Participants
A First-In-Human Study of the Study Medicine, Called PF-07291177, in Healthy Adult Participants
The purpose of the study is to learn about the safety, tolerability (the extent to which side effects can be tolerated), and plasma pharmacokinetics (PK) (PK helps us understand how the drug is changed and eliminated from body…
- Trial name
- NCT05434091
- Testing
- PF-07291177
- Volunteers
- healthy volunteers welcome
- Sponsor
- Pfizer
Transthyretin (TTR)-Mediated Amyloidosis
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
- Trial name
- NCT02053454
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals