Trial Search Results

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

To evaluate the efficacy of AKCEA-TTR-LRx compared to placebo for 120 weeks in patients with ATTR-CM receiving available standard of care (SoC). For more information, please visit

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Ionis Pharmaceuticals, Inc.

Collaborator: Akcea Therapeutics

Stanford Investigator(s):


  • Drug: AKCEA-TTR-LRx
  • Drug: Placebo


Phase 3


Inclusion Criteria:

   - Females must be non-pregnant and non-lactating, and either surgically sterile or
   post-menopausal or abstinent. If engaged in sexual relations of child-bearing
   potential, agree to use 1 highly effective contraceptive method

   - Males must be surgically sterile or, abstinent or, if engaged in sexual relations with
   a woman of child-bearing potential, the participant or the participant's non-pregnant
   female partner must be using a highly effective contraceptive method

   - Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or
   equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2-
   propanodicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99m
   Tc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the
   absence of abnormal light chains ratio, centrally confirmed

   - End-diastolic interventricular septum thickness of > 12 mm on Screening echocardiogram

   - New York Heart Association (NYHA) class I-III

Exclusion Criteria:

   - Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA),
   coronary revascularization, cardiac device implantation, cardiac valve repair, or
   major surgery within 3 months of Screening

   - Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to
   hypertension, valvular heart disease, or ischemic heart disease

   - Monoclonal gammopathy of undetermined significance (MGUS) and/or immunoglobulin free
   light chain ratio < 0.26 and > 1.65, unless fat, bone marrow, or heart biopsy
   confirming the absence of light chain by mass spectrometry or immunoelectron

   - Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or
   anticipated liver transplant or LVAD within 1 year after randomization

   - Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or
   other oligonucleotide or RNA therapeutic (including siRNA)

   - Current treatment with diflunisal, doxycycline, and/or calcium-channel blocker.
   Participants receiving any of these agents must respect a wash-out period of 14 days
   before randomization.

Ages Eligible for Study

18 Years - 90 Years

Genders Eligible for Study


Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305