A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Alnylam Pharmaceuticals

Status: Completed

Conditions: Polyneuropathy; Hereditary Transthyretin-mediated (ATTRv) Amyloidosis

Treatments: Drug: Patisiran

Study type: Observational

Funder types: Industry

Identifiers

NCT04201418

ALN-TTR02-012

Details and patient eligibility

About

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

Enrollment: 67 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

67 participants in 3 patient groups

Patisiran Prospective Cohort

Description:

Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.

Treatment:

Drug: Patisiran

Patisiran Mixed Cohort

Description:

Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.

Treatment:

Drug: Patisiran

Patisiran Retrospective Cohort

Description:

Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Treatment:

Drug: Patisiran