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8 Diseases |  |
1 Trial |
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1 Trial |  |
52 News |  |
- || Onpattro (patisiran) / Alnylam, Sanofi, revusiran (ALN-TTRsc) / Alnylam, Sanofi
Review, Journal: Safety and effectiveness of interference RNA (RNAi) based therapeutics in cardiac failure: A systematic review. (Pubmed Central) - Oct 13, 2024
RNAi therapeutics are novel treatment option for improving cardiac function because their high target specificity, ability to target genes that conventional drugs struggle to reach and potential for long-lasting effects. Further research on optimizing delivery methods, improving target specificity, evaluating long-term safety profiles and cost-effectiveness to fully realize their potential.
- | Journal: Targeted Therapeutics for Transthyretin Amyloid Cardiomyopathy. (Pubmed Central) - Sep 15, 2023
Evidence from the pooled analysis revealed targeted therapeutics improve survival and are well-tolerated. These findings suggest a potential role for targeted therapeutics in the treatment of patients with ATTR-CM.
- || Review, Journal: Targeted Therapies for Hereditary Peripheral Neuropathies: Systematic Review and Steps Towards a 'treatabolome'. (Pubmed Central) - Nov 17, 2021
These findings suggest a potential role for targeted therapeutics in the treatment of patients with ATTR-CM. The 'treatable' variants highlighted in this project will be flagged in the treatabolome database to alert clinicians at the time of the diagnosis and enable timely treatment of patients with hereditary peripheral neuropathies.
- || revusiran (ALN-TTRsc) / Alnylam, Sanofi
Clinical, Journal: Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis. (Pubmed Central) - Jun 10, 2021
There was no evidence of genotoxicity in vitro or in vivo at limit doses or carcinogenicity in a 2-year study in rats at doses up to 100 mg/kg. Overall, these results demonstrate that revusiran had a favorable nonclinical safety profile.
- ||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Clinical, P3 data, Journal: Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR). (Pubmed Central) - Oct 1, 2020
P3
Causes for the observed mortality imbalance associated with revusiran were thoroughly investigated and no clear causative mechanism could be identified. Although the results suggest similar progression of cardiac parameters in both treatment arms, a role for revusiran cannot be excluded.
- | vutrisiran (ALN-TTRsc02) / Alnylam, revusiran (ALN-TTRsc) / Alnylam, Sanofi
Clinical, Journal: Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates. (Pubmed Central) - Oct 29, 2019
No apparent functional impact on mitochondria and no significant accumulation of 2'-F-monomers were observed after weekly administration of two GalNAc-siRNA conjugates in rats for ∼2 years. Taken together, the results support the conclusion that 2'-F nucleotides can be safely applied for the design of metabolically stabilized therapeutic GalNAc-siRNAs with favorable potency and prolonged duration of activity allowing for low dose and infrequent dosing.
- Targeted Therapeutics for Transthyretin amyloid cardiomyopathy: Time to Move Forward () - Sep 9, 2019 - Abstract #HFSA2019HFSA_68;
Targeted therapeutics (TT) for ATTR-CA include TTR stabilizers (tafamidis, dilfunisal) and oligonucleotide drugs (Revusiran, Patisiran and Inotersen).TTR stabilizers prevent dissociation of amyloid tetramers and thereby limit the deposition of amyloid fibrils. sIn patients with ATTR-CA, TT reduced all-cause mortality and were well-tolerated as compared to placebo.
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Equally well except for revusiran. (Twitter) - Jun 22, 2019
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Clinical, Journal: Demyelinating Neuropathy in a Patient Treated With Revusiran for Transthyretin (Thr60Ala) Amyloidosis. (Pubmed Central) - Jun 15, 2019
A sural nerve biopsy demonstrated demyelination with substantial axonal loss in the absence of histologically detectable endoneurial amyloid deposition. This case expands the clinicopathologic spectrum of transthyretin amyloidosis and may represent complex disease and treatment effects.
- |||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (clinicaltrials.gov) - Dec 12, 2017
P3, N=206, Completed, Sponsor: Alnylam Pharmaceuticals
This case expands the clinicopathologic spectrum of transthyretin amyloidosis and may represent complex disease and treatment effects. Trial primary completion date: Oct 2016 --> Mar 2017
- revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Dec 12, 2017
P2, N=25, Completed, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Oct 2016 --> Mar 2017 Trial primary completion date: Oct 2016 --> Feb 2017
- revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant (clinicaltrials.gov) - Dec 12, 2017
P2, N=12, Completed, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Oct 2016 --> Feb 2017 Trial primary completion date: Oct 2016 --> Feb 2017
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial completion: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant (clinicaltrials.gov) - May 24, 2017
P2, N=12, Completed, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Oct 2016 --> Feb 2017 Active, not recruiting --> Completed
- ||||||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial completion: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (clinicaltrials.gov) - May 24, 2017
P3, N=206, Completed, Sponsor: Alnylam Pharmaceuticals
Active, not recruiting --> Completed Active, not recruiting --> Completed
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial completion: A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - May 24, 2017
P2, N=25, Completed, Sponsor: Alnylam Pharmaceuticals
Active, not recruiting --> Completed Active, not recruiting --> Completed
- |||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (clinicaltrials.gov) - Oct 11, 2016
P3, N=206, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Active, not recruiting --> Completed Trial primary completion date: Feb 2018 --> Oct 2016
- revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Oct 11, 2016
P2, N=25, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Feb 2018 --> Oct 2016 Trial primary completion date: Feb 2017 --> Oct 2016
- revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial primary completion date: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant (clinicaltrials.gov) - Oct 11, 2016
P2, N=12, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Feb 2017 --> Oct 2016 Trial primary completion date: Dec 2017 --> Oct 2016
- ||||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment closed, Trial primary completion date: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (clinicaltrials.gov) - Aug 15, 2016
P3, N=206, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Trial primary completion date: Dec 2017 --> Oct 2016 Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2018 --> Feb 2018
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment closed, Trial primary completion date: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant (clinicaltrials.gov) - Jul 28, 2016
P2, N=12, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2018 --> Feb 2018 Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2016 --> Dec 2017
- ||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial completion: 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 (clinicaltrials.gov) - Feb 5, 2016
P1, N=85, Completed, Sponsor: Alnylam Pharmaceuticals
Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2016 --> Dec 2017 Active, not recruiting --> Completed
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
New P2 trial: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant (clinicaltrials.gov) - Nov 5, 2015
P2, N=12, Recruiting, Sponsor: Alnylam Pharmaceuticals
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment closed: A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Jun 30, 2015
P2, N=25, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Active, not recruiting --> Completed Recruiting --> Active, not recruiting
- || revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment closed: 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 (clinicaltrials.gov) - May 22, 2015
P1, N=96, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- || revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment change, Trial primary completion date: 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 (clinicaltrials.gov) - Feb 27, 2015
P1, N=96, Recruiting, Sponsor: Alnylam Pharmaceuticals
Recruiting --> Active, not recruiting N=76 --> 96 | Trial primary completion date: Dec 2014 --> Jun 2015
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Trial completion: Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Feb 27, 2015
P2, N=25, Completed, Sponsor: Alnylam Pharmaceuticals
N=76 --> 96 | Trial primary completion date: Dec 2014 --> Jun 2015 Active, not recruiting --> Completed
- |||||||||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
New P3 trial: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (clinicaltrials.gov) - Dec 19, 2014
P3, N=200, Recruiting, Sponsor: Alnylam Pharmaceuticals
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
New P2 trial: A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Nov 17, 2014
P2, N=25, Recruiting, Sponsor: Alnylam Pharmaceuticals
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment closed: Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Oct 31, 2014
P2, N=25, Active, not recruiting, Sponsor: Alnylam Pharmaceuticals
Active, not recruiting --> Completed Recruiting --> Active, not recruiting
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment change: Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Aug 26, 2014
P2, N=25, Recruiting, Sponsor: Alnylam Pharmaceuticals
Recruiting --> Active, not recruiting N=15 --> 25
- || revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment change: 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 (clinicaltrials.gov) - Apr 21, 2014
P1, N=76, Recruiting, Sponsor: Alnylam Pharmaceuticals
N=15 --> 25 N=40 --> 76
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
Enrollment open: Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Jan 23, 2014
P2, N=15, Recruiting, Sponsor: Alnylam Pharmaceuticals
N=40 --> 76 Not yet recruiting --> Recruiting
- | revusiran (ALN-TTRsc) / Alnylam, Sanofi
New P2 trial: Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis (clinicaltrials.gov) - Nov 11, 2013
P2, N=15, Recruiting, Sponsor: Alnylam Pharmaceuticals
- ||| revusiran (ALN-TTRsc) / Alnylam, Sanofi
New P1 trial: 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 (clinicaltrials.gov) - Mar 18, 2013
P1, N=76, Recruiting, Sponsor: Alnylam Pharmaceuticals