Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Respons… (NCT05023889) | Clinical Trial Compass
Active — Not RecruitingEarly Phase 1
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
United States10 participantsStarted 2022-08-03
Plain-language summary
To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Male or female \>18
✓. Diagnosis of symptomatic polyneuropathy
✓. wtATTR based on cardiac biopsy or Tc99m PYP
✓. Negative hATTR sequencing
✓. 0 to 0.5 gram/dl serum monoclonal protein.
✓. No history of other secondary causes of neuropathy.
✓. Have adequate complete blood counts and liver function tests
✓. Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)
Exclusion criteria
✕. Other Causes of neuropathy as determined by the principle investigator.
✕. Has known human immunodeficiency virus (HIV) infection;
✕. Primary AL.
✕. NYHA Class IV at the Screening visit. 5. Has any of the following laboratory parameter assessments at screening:
✕. Aspartate transaminase (AST) or alanine transaminase (ALT) levels ˃2.0 × the upper limit of normal (ULN).
✕. Total bilirubin ˃ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if total bilirubin \<2 × ULN.