Patisiran

Patisiran in patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) disease progression post-liver transplant.

Patisiran is an RNA inhibitor TTR lowering drug, belonging to the small interfering RNA drug class. It is administered by intravenous infusion. Patisiran has been shown to reverse neuropathy in patients with hereditary ATTR amyloidosis and neuropathy and is approved by the regulatory authorities for this indication. This study will evaluate the efficacy and safety of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

What the study involves for patients

The study aims to enrol 20 participants to receive patisiran by intravenous infusion over a 12-month period.

Who can take part in the trial

Patients who received a liver transplant for treatment of hereditary ATTR amyloidosis at least 12 months before the study started, and who have an increase in polyneuropathy (PND) score after transplant may take part. Participants must have received stable immunosuppression treatment with prednisone for at least 3 months prior to starting the study. Patients may not take part in the study if they have previously received patisiran or inotersen, have clinically significant liver function test abnormalities, portal hypertension with ascites, severe renal failure, leptomeningeal amyloidosis or very poor performance status. For a full list of inclusion and exclusion criteria, see the www.clinicaltrials.gov website.

Outcomes

The primary outcome assessed will be:  

  • Average month 6 and month 12 percentage reduction from baseline in serum transthyretin (TTR).

Other outcomes assessed will include: 

  • Change from baseline to month 12 of treatment in the modified neurologic impairment score +7 (mNIS+7).
  • Change from baseline to month 12 of treatment in the Norfolk quality of life-diabetic neuropathy (Norfolk QoL-DN) total score.
  • Change from baseline in the Rasch-Built Overall Disability Scale (R-ODS) at month 12.
  • Change from baseline in the composite autonomic symptom score (COMPASS-31) at month 12.
  • Change from baseline in the modified body mass index (mBMI) at month 12.
  • Percentage of participants with adverse events.

Timing

This study has completed recruitment and is ongoing at the NAC. Overall, 24 participants from around the world were enrolled.