NCT03536767

Brief Summary

This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of AG10 administered on a background of stable heart failure therapy.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
55

participants targeted

Target at P25-P50 for phase_2

Timeline
12mo left

Started Aug 2018

Longer than P75 for phase_2

Geographic Reach
1 country

9 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress88%
Aug 2018Jul 2027

First Submitted

Initial submission to the registry

May 11, 2018

Completed
14 days until next milestone

First Posted

Study publicly available on registry

May 25, 2018

Completed
3 months until next milestone

Study Start

First participant enrolled

August 8, 2018

Completed
8.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2027

Last Updated

March 13, 2025

Status Verified

March 1, 2025

Enrollment Period

8.9 years

First QC Date

May 11, 2018

Last Update Submit

March 11, 2025

Conditions

Amyloid Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

  • Assessment of long-term safety and tolerability: Incidence of each treatment-emergent adverse events

    Incidence of each treatment-emergent adverse events

    up to 60 Months or study completion by recommendation from safety monitoring committee

Secondary Outcomes (4)

  • AG10 Pharmacokinetics AUC

    up to 60 Months or study completion by recommendation from safety monitoring committee

  • AG10 Pharmacodynamic Assessments of TTR stabilization by Fluorescent Polarization Exclusion Assay

    up to 60 Months or study completion by recommendation from safety monitoring committee

  • AG10 Pharmacodynamic Assessments of TTR stabilization by Western Blot

    up to 60 Months or study completion by recommendation from safety monitoring committee

  • AG10 Pharmacodynamic Assessments: prealbumin

    up to 60 Months or study completion by recommendation from safety monitoring committee

Study Arms (1)

Open-Label

EXPERIMENTAL
Drug: AG10

Interventions

AG10DRUG

AG10 800mg twice daily, oral administration

Also known as: TTR Stabilizer
Open-Label

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Completed participation in study AG10-201.
  • Willing and able to comply with the study medication regimen and all study requirements.
  • The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation.
  • Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use effective method(s) of contraception.

You may not qualify if:

  • Hemodynamic instability that would pose too great a risk to the subject.
  • Abnormalities in clinical laboratory tests that would pose too great a risk to the subject.
  • Any clinically significant ongoing medical condition or any laboratory abnormality or condition that could adversely affect the safety of the patient.
  • Known hypersensitivity to study drug (AG10), its metabolites, or formulation excipients.
  • Likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year.
  • Receiving current treatment with diflunisal, tafamidis, green tea, doxycycline, TUDCA/Ursodiol, patisiran, inotersen or any other investigational ATTR agent within 14 days or 5 half-lives of the prior investigational agent (whichever is longer) prior to dosing with study drug.
  • Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before the study drug is administered.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Stanford University

Palo Alto, California, 94304, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29424, United States

Location

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesAmyloid NeuropathiesPeripheral Nervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmyloidosis, FamilialMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis Deficiencies

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 11, 2018

First Posted

May 25, 2018

Study Start

August 8, 2018

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

July 1, 2027

Last Updated

March 13, 2025

Record last verified: 2025-03

Data Sharing

IPD Sharing
Will not share

Locations

US(9)