Clinical trials: TTR-mediated Amyloidosis
TTR-mediated Amyloidosis
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.
- Trial name
- NCT02292186
- Testing
- Revusiran (ALN-TTRSC)
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis
A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC (revusiran) in healthy volunteer subjects.
- Trial name
- NCT01814839
- Testing
- ALN-TTRSC (revusiran), Sterile Normal Saline (0.9% NaCl)
- Where
- 2 sites in United Kingdom
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
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 Amyloid Cardiomyopathy (ATTR CM)
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC).
- Trial name
- NCT04136171
- Testing
- Eplontersen
- Where
- 139 sites in United States, Argentina and 19 more
- Volunteers
- 1,438 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Amyloidosis, Familial, Transthyretin Amyloidosis
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver…
- Trial name
- NCT03862807
- Testing
- Patisiran
- Where
- 9 sites in France, Germany and 5 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The CARDIO-TTRansform Scintigraphy Sub-study
The purpose of this study is to examine the changes in amyloid myocardial burden in a subset of the population participating in the ION682884-CS2 (NCT04136171) study, up to 150 participants, after treatment with eplontersen or…
- Trial name
- NCT06073587
- Testing
- Scintigraphy scan
- Where
- 5 sites in United States, Spain
- Volunteers
- 163 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in participants with ATTR amyloidosis with cardiomyopathy.
- Trial name
- NCT04153149
- Testing
- Vutrisiran, Sterile Normal Saline (0.9% NaCl)
- Where
- 119 sites in United States, Argentina and 30 more
- Volunteers
- 655 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-Mediated Amyloid Cardiomyopathy (ATTR-CM)
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
The main purpose of this study is to measure the amyloid burden, defined as extracellular volume (ECV) assessed by Magnetic Resonance Imaging (MRI) over time in a subset of up to 150 participants enrolled in ION-682884-CS2…
- Trial name
- NCT06073574
- Testing
- Cardiac MRI
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 160 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin Amyloidosis, Amyloidosis, Hereditary
Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran
ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).
- Trial name
- MyocardON-TTR (NCT05873868)
- Testing
- Six minutes walk test, Kansas City questionnaire, COMPASS31 self questionnaire
- Where
- 5 sites in France
- Volunteers
- 20 people
- Sponsor
- Rennes University Hospital
Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy
Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).
- Trial name
- NCT05505838
- Testing
- Patisiran
- Where
- 20 sites in United States
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary…
- Trial name
- NCT04136184
- Testing
- Inotersen, Eplontersen
- Where
- 45 sites in United States, Argentina and 14 more
- Volunteers
- 168 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM
The purpose of this randomised, double-blind, placebo-controlled, multicenter study is to evaluate the efficacy and safety of concomitant use of eplontersen and ALXN2220 compared with eplontersen and placebo in adult participants…
- Trial name
- ATTRiumph (NCT07608354)
- Testing
- Eplontersen, ALXN2220
- Where
- 76 sites in United States, Canada and 8 more
- Volunteers
- 326 people
- Sponsor
- AstraZeneca
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
Hereditary Transthyretin Amyloidosis With Polyneuropthy
A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN
The objective of the study was to characterize adverse events (AEs) occurring within one day of TEGSEDI administration to adult participants with hATTR-PN overall and in individual participants with respect to time course of AE…
- Trial name
- NCT04306510
- Testing
- TEGSEDI
- Where
- 2 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- Akcea Therapeutics
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy.
- Conditions
- Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies
- Trial name
- NCT02319005
- Testing
- Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
- Where
- 61 sites in United States, Belgium and 7 more
- Volunteers
- 206 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01617967
- Testing
- Patisiran
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 29 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01961921
- Testing
- ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 27 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Mediated Amyloidosis (ATTR)
Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01148953
- Testing
- ALN-TTR01, Sterile Normal Saline (0.9% NaCl)
- Where
- 4 sites in France, Portugal and 2 more
- Volunteers
- 32 people
- Sponsor
- Alnylam Pharmaceuticals