Pfizer is conducting a new study on individuals with hereditary transthyretin amyloidosis (hATTR) who have participated in 2 other clinical trials in Spain.

Inclusion criteria are that patients have received 61 mg of tafamidis (as given in the 2 Spanish studies) for at least 12 months and followed up for at least 12 months.

The primary outcome measure of the trial is the change from baseline in neurological impairment score; secondary measures include extracardiac clinical features and changes in quality of life, autonomic symptoms, body mass index, and sudomotor response.

Tafamidis is an oral medication known as a transthyretin stabilizer. It is designed to treat adults with cardiomyopathy and peripheral neuropathy due to hATTR by preventing deposits of transthyretin from forming in cardiac tissue and nerves. It is usually taken orally by capsule once a day. Tafamidis was approved by the European Medicines Agency in November 2011, and in 2019, the US Food and Drug Administration granted tafamidis priority review, fast track, and breakthrough therapy designations.

Read more about hATTR therapies

The observational, retrospective study plans to enroll 10 participants aged 18 years and above, and it was launched on September 30, 2022. The estimated completion date is November 30, 2022.

hATTR is a progressive hereditary condition in which abnormal deposits of amyloid protein accumulate in the body’s organs and tissues. Most often, the deposits are in the peripheral nervous system and therefore affect the nerves connecting the brain and spinal cord to muscles and sensory cells. The protein deposits can also affect the autonomic nervous system, interfering with blood pressure, heart rate, and digestion. The disease can also affect the central nervous system.

The 3 main forms of hATTR are (1) neuropathic, affecting the peripheral and autonomic nervous systems, (2) leptomeningeal, which affects the central nervous system, and (3) cardiac, which affects the heart.

Reference

Retrospective study collecting neurological follow-up of hereditary transthyretin amyloidosis (ATTRv) patients included in B3461028 and B3461045. (TRAMA). ClinicalTrials.gov. September 29, 2022. Accessed October 10, 2022.