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

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

Transthyretin Amyloidosis (ATTR)

Patisiran

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation

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.

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

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

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

Polyneuropathy

Hereditary Transthyretin-mediated (ATTRv) Amyloidosis

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

Clinical Trial Site

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.

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

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

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

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

About this trial

Study ID

Status

Type

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status