NCT04882735

A Phase 3, Open-Label, Multicenter, Single-Arm Study to Evaluate the Efficacy and Safety of Acoramidis in Subjects with Symptomatic Transthyretin Amyloid Polyneuropathy (ATTRibute-PN Study)

Withdrawn Phase 3 Last updated 10 September 2024

What this trial tests

Phase 3 trial testing Acoramidis in Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy. Withdrawn.

Timeline

8 September 2021

Primary endpoint
1 September 2026

1 October 2026

Quick facts

Lead sponsor	Eidos Therapeutics, a BridgeBio company
Phase	Phase 3
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Start date	8 September 2021
Primary completion	1 September 2026
Estimated completion	1 October 2026

Drugs / interventions tested

• Acoramidis (ACORAMIDIS) — full drug profile →

Conditions studied

• Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy — all drugs for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy →

Sponsor

Eidos Therapeutics, a BridgeBio company — full company profile →

Who can join

Adults 18 to 90, any sex, with Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Change from baseline to Month 18 in mNIS+7
  Time frame: 18 Months
  To determine the efficacy of acoramidis in subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN) by evaluating the change in Modified Neuropathy Impairment Score +7 (mNIS+7) from baseline to 18 months.
• Safety: TESAEs will be used to evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic polyneuropathy (ATTR-PN)
  Time frame: 60 Months
  To evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)
• Safety: Adverse Events leading to treatment discontinuation will be used to evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic polyneuropathy (ATTR-PN)
  Time frame: 60 Months
  To evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)
• Safety: Adverse Events will be used to evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic polyneuropathy (ATTR-PN)
  Time frame: 60 Months
  To evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)
• Safety: Incidence of abnormal physical exam will be used to evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic polyneuropathy (ATTR-PN)
  Time frame: 60 Months
  To evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)
• Safety: Incidence of abnormal vital signs will be used to evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic polyneuropathy (ATTR-PN)
  Time frame: 60 Months
  To evaluate the long-term safety and tolerability of acoramidis administered to subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)

Sponsor's own description

Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis.
   Carroll A, Dyck PJ, de Carvalho M, Kennerson M, et al · · 2022 · cited 79× · PMID 35256455 · DOI 10.1136/jnnp-2021-327909
2. Current and Emerging Therapies for Hereditary Transthyretin Amyloidosis: Strides Towards a Brighter Future.
   Obici L, Mussinelli R. · · 2021 · cited 17× · PMID 34850359 · DOI 10.1007/s13311-021-01154-y
3. Transthyretin Kinetic Stabilizers for ATTR Amyloidosis: A Narrative Review of Mechanisms and Therapeutic Benefits.
   Powers ET, Amass L, Baylor L, Fernández-Arias I, et al · · 2025 · cited 4× · PMID 40730935 · DOI 10.1007/s40119-025-00423-7