Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04201418
Other study ID # ALN-TTR02-012
Secondary ID
Status Completed
Phase
First received
Last updated
Start date December 18, 2019
Est. completion date May 24, 2022

Study information

Verified date June 2022
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.


Recruitment information / eligibility

Status Completed
Enrollment 67
Est. completion date May 24, 2022
Est. primary completion date May 24, 2022
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at baseline. - Exposure to commercial patisiran in one of the 3 cohorts: - Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. - Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment. - Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment. Exclusion Criteria: - New York Heart Association (NYHA) heart failure classification =3 - Karnofsky Performance Status (KPS) <60% - Unstable congestive heart failure (CHF) - Known primary amyloidosis (AL) or leptomeningeal amyloidosis - Prior major organ transplant - Previously received patisiran - Previous treatment with a TTR silencing therapy

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Patisiran
Patisiran-lipid complex injection, for intravenous use

Locations

Country Name City State
United States Clinical Trial Site Allentown Pennsylvania
United States Clinical Trial Site Aurora Colorado
United States Clinical Trial Site Austin Texas
United States Clinical Trial Site Bethlehem Pennsylvania
United States Clinical Trial Site Boston Massachusetts
United States Clinical Trial Site Braselton Georgia
United States Clinical Trial Site Chapel Hill North Carolina
United States Clinical Trial Site Charlotte North Carolina
United States Clinical Trial Site Chicago Illinois
United States Clinical Trial Site Chicago Illinois
United States Clinical Trial Site Columbus Ohio
United States Clinical Trial Site Detroit Michigan
United States Clinical Trial Site Farmington Connecticut
United States Clinical Trial Site Germantown Tennessee
United States Clinical Trial Site Jackson Mississippi
United States Clinical Trial Site Jacksonville Florida
United States Clinical Trial Site Kansas City Missouri
United States Clinical Trial Site Lancaster Pennsylvania
United States Clinical Trial Site Los Angeles California
United States Clinical Trial Site Macon Georgia
United States Clinical Trial Site Milwaukee Wisconsin
United States Clinical Trial Site Omaha Nebraska
United States Clinical Trial Site Pittsburgh Pennsylvania
United States Clinical Trial Site Pittsburgh Pennsylvania
United States Clinical Trial Site San Antonio Texas
United States Clinical Trial Site San Diego California
United States Clinical Trial Site Tampa Florida

Sponsors (1)

Lead Sponsor Collaborator
Alnylam Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden. Baseline, Month 12
See also
  Status Clinical Trial Phase
Completed NCT05560555 - Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
Recruiting NCT02033057 - Muscular Electrostimulation of the Sedated and Mechanically Ventilated Critically Ill Patient. Analysis of the Effect on Acquired Muscular Weakness and Its Clinical Consequences. Phase 4
Withdrawn NCT02566941 - Neuromuscular Electrical Stimulation in the Critically Ill N/A
Completed NCT02442986 - Neurological Outcome in Surgical and Non-surgical Septic Patients N/A
Completed NCT02706314 - Impact of Human Blood Serum From Critically Ill Patients on Human Colon Neuronal Networks.
Completed NCT01076478 - Asian Study on Cilostazol Effectivity in Neuropathies of Diabetes Mellitus Type 2-A Pilot Study in the Philippines Phase 4
Completed NCT03373370 - Early Diagnosis of TTR Amyloidosis by Use of Molecular Biology
Completed NCT02846844 - Patients With Chemotherapy-induced Polyneuropathy Are Treated With an Integrated Program Including Massage, Mobilization in Posture and Transport Layers, Physical Exercises or With Whole-body Vibration Platform Training N/A
Recruiting NCT05950867 - Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause. N/A
Terminated NCT00832572 - Study of Ranexa in Patients With Coronary Artery Disease and Painful Polyneuropathy Phase 4
Completed NCT00614562 - Neurally Adjusted Ventilatory Assist (NAVA) in Patients With Critical Illness Associated Polyneuropathy / or Polymyopathy (CIP/M) Phase 1
Completed NCT01302275 - Oxcarbazepine for the Treatment of Chronic Peripheral Neuropathic Pain Phase 4
Terminated NCT01088256 - Efficacy of Etoricoxib on Peripheral Hyperalgesia Phase 2
Not yet recruiting NCT01047488 - Imipramine and Pregabalin Combination in Painful Polyneuropathy Phase 4
Recruiting NCT06414746 - Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
Completed NCT01450163 - Evaluate The Efficacy and Safety Of Pregabalin In Prevention, Reduction of Oxaliplatin-Induced Painful Neuropathy Phase 3
Recruiting NCT05040373 - Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
Not yet recruiting NCT02666456 - The Influence of Sensory Phenotype on the Risk of Developing Neuropathic Pain N/A
Withdrawn NCT00723918 - Combination of an Investigational Cannabinoid and Methadone for HIV-associated Neuropathy Phase 2
Completed NCT00259974 - RIMAG Study: Trial of Rituximab Versus Placebo in Polyneuropathy Associated With Anti-MAG IgM Monoclonal Gammopathy Phase 3