Clinical trials: Family Relationships
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
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)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Family Relations, Sibling Relations
SIB-Time Web-application Tool for Typically Developing Siblings
In the United States, over 32.7 million people have special health, developmental, and mental health concerns. Most of these people have typically developing brothers and sisters.
- Trial name
- SIBTime (NCT04633473)
- Testing
- SIB-Time Web-Application Tool
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Oregon Research Behavioral Intervention Strategies, Inc.
Amyloid Cardiomyopathy, Transthyretin-Related
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)
- Trial name
- AG10 (NCT04988386)
- Testing
- Acoramidis (AG10)
- Where
- 72 sites in United States, Australia and 15 more
- Volunteers
- 389 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
Amyloidosis, Amyloid Cardiomyopathy
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause…
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- NCT06563895
- Testing
- Acoramidis
- Where
- 104 sites in United States, Argentina and 22 more
- Volunteers
- 587 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company