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Home › Conditions › Transthyretin Amyloidosis

Transthyretin Amyloidosis

0 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Transthyretin Amyloidosis

5 trials tracked for Transthyretin Amyloidosis: 3 in phase 3 or 4 and 3 with published results. The most-cited published study has 379 citations.

5Trials tracked
3Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 3 3 Phase 2 1 Other / NA 1
  1. Phase 3 HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) Completed · 379 cited
  2. Phase 3 Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant Completed · 55 cited
  3. Phase 3 Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy Completed · 4 cited
  4. Phase 2 Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR Completed
  5. N/A Transthyretin Amyloidosis Outcome Survey (THAOS) Completed

Showing the 5 most-cited and recently-updated of 5 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Transthyretin Amyloidosis: what the trials found

Patisiran has demonstrated significant improvements in clinical outcomes for patients with transthyretin amyloidosis. In a Phase 3 trial, Patisiran showed statistically significant improvements in the Modified Neurologic Impairment Score (mNIS) 1, while another Phase 3 study reported a 91.0% average reduction in serum Transthyretin (TTR) and improvements in both the Neuropathy Impairment Score (NIS) and Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) scores 2.

Acoramidis demonstrated significant efficacy in a Phase 3 trial, showing a statistically significant improvement in a hierarchical combination of all-cause mortality, cumulative frequency of CV-related hospitalization, change from baseline in NT-proBNP, and change in the 6-minute walk test (6MWT) 3. Additionally, patients treated with acoramidis showed significant improvements in 6MWT distance and Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at month 30 3.

Clinical assessments using Patisiran also demonstrated statistically significant improvements in the Timed 10-Meter Walk Test (10-MWT) compared to placebo 1.

Recent results — preliminary, needs further review

  • I 124-Evuzamitide showed a median change in uptake as measured by percentage change in injected dose of 0.2, 0.8, 1.3, and 0.3 in a Phase 2 study 4.

For the clinician treating this condition

  • Patisiran is associated with significant reductions in serum TTR levels and improvements in neurological impairment scores (mNIS, NIS) and quality of life metrics [1, 2].
  • Acoramidis significantly improves cardiovascular outcomes, including reduced CV-related hospitalizations and improved functional capacity as measured by the 6MWT and KCCQ-OS 3.
  • Patisiran has been shown to improve mobility as measured by the Timed 10-Meter Walk Test 1.

AI synthesis of 4 cited trials, updated Jul 8, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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