Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (TRAMA)

Pfizer

Status: Completed

Conditions: Polyneuropathy; Hereditary Transthyretin Amyloidosis (ATTRv)

Treatments: Drug: Tafamidis

Study type: Observational

Funder types: Industry

Identifiers

NCT05560555

TRAMA (Other Identifier)

B3461104

Details and patient eligibility

About

A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months.

Enrollment: 5 patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Treatment with tafamidis 61 mg ≥ 12 months
• Neurological follow up ≥ 12 months
• Diagnosis of transthyretin amyloidosis with polyneuropathy (ATTR-PN) based on one of the following:
• Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudo scan, RR Interval analysis, etc..)

Exclusion criteria

• Treatment with tafamidis 61 mg < 12 months
• Neurological follow up < 12 months
• Other diagnosis for polyneuropathy

Trial design

5 participants in 1 patient group

Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain

Treatment:

Drug: Tafamidis