Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10

NCT03294707 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: AG10-001
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects

Conditions

Amyloid Cardiomyopathy, Transthyretin-Related

Outcome Measures

Primary Outcomes (1)

• Safety & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment.

  To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects

  30 days

Secondary Outcomes (12)

• Pharmacokinetic Assessments: T1/2

  30 days

• Pharmacokinetic Assessments: Tmax

  30 days

• Pharmacokinetic Assessments: Cmax

  30 days

• Pharmacokinetic Assessments: Cmin

  30 days

• Pharmacokinetic Assessments: AUC

  30 days

Study Arms (2)

AG10 single oral dose

EXPERIMENTAL

AG10 oral tablet, administered by mouth, once

Drug: AG10 oral tablet

Placebo single oral dose

PLACEBO COMPARATOR

Placebo Oral Tablet, administered by mouth, once

Drug: Placebo Oral Tablet

Interventions

AG10 oral tablet DRUG

Active single ascending dose

Also known as: Eidos Therapeutics AG10

AG10 single oral dose

Placebo Oral Tablet DRUG

Placebo single dose

Also known as: Matching placebo

Placebo single oral dose

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Weight between \>50 kg and ≤110 kg;
• BMI of 18 to 32 kg/m2;
• Subjects who are healthy as determined by medical history, physical examination, 12 lead ECG and standard laboratory tests;
• Subjects who are negative for drugs of abuse and alcohol tests;
• Subjects who are non-smokers;

You may not qualify if:

• Subjects who have used prescription drugs within 4 weeks of first dosing;
• Subjects who have a prior cholecystectomy;
• Subjects who have used any over-the-counter medications within 7 days prior to Day -1;
• Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, or connective tissue diseases or disorders;
• Subjects who have an abnormal screening ECG;

Sponsors & Collaborators

• Eidos Therapeutics, a BridgeBio company: lead
• Celerion: collaborator

Study Sites (1)

Celerion

Tempe, Arizona, 85283, United States

Location

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Officials

• Terry O'Reilly, M.D.

  Celerion

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Subjects will be randomized with an overall ratio of 3:1 to AG10: placebo within each cohort
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2017
• First Posted: September 27, 2017
• Study Start: September 11, 2017
• Primary Completion: February 5, 2018
• Study Completion: May 18, 2018
• Last Updated: September 19, 2024

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)