Clinical trials: Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, open to volunteers
- Condition Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
TEGSEDI Pregnancy Surveillance Program
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
- Trial name
- NCT04270058
- Testing
- inotersen
- Volunteers
- 20 people
- Sponsor
- Akcea Therapeutics
Transthyretin Amyloidosis, ATTR-CM
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes.
- Conditions
- Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR
- Trial name
- MaesTTRo (NCT06465810)
- Testing
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
- Where
- 89 sites in United States, Canada and 4 more
- Volunteers
- 1,850 people
- Sponsor
- AstraZeneca