Clinical trials: ATTR-Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study…
- Trial name
- LTSAFE-ALXN (NCT07789483)
- Testing
- ALXN2220
- Where
- 195 sites in United States, Argentina and 25 more
- Volunteers
- 1,180 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Symptomatic Transthyretin Amyloid Cardiomyopathy
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with…
- Trial name
- NCT04622046
- Testing
- ALXN2060
- Where
- 11 sites in Japan
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures