Clinical trial NCT03294707: Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
Official title: A Phase 1 Randomized, Placebo-controlled, Single and Multiple Ascending Dose Study of the Tolerability, Pharmacokinetics and Pharmacodynamics of AG10 in Healthy Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Amyloid Cardiomyopathy, Transthyretin-Related
Lead sponsor: Eidos Therapeutics, a BridgeBio company
Sponsor class: INDUSTRY
Collaborators: name: Celerion
First posted: 2017-09-27
Enrollment: 56
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: Up to 48 healthy volunteers will be given a single dose of AG10 or placebo and be monitored for safety and tolerability over a 5-day period. Up to 48 healthy volunteers will be given multiple doses of AG10 or placebo and be monitored for safety and tolerability over a 15-day period.

Eligibility criteria: Inclusion Criteria:

* 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;

Exclusion Criteria:

* 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;

Primary outcomes:
measure: 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.
timeFrame: 30 days
Secondary outcomes:
measure: Pharmacokinetic Assessments: T1/2
timeFrame: 30 days
measure: Pharmacokinetic Assessments: Tmax
timeFrame: 30 days
measure: Pharmacokinetic Assessments: Cmax
timeFrame: 30 days
measure: Pharmacokinetic Assessments: Cmin
timeFrame: 30 days
measure: Pharmacokinetic Assessments: AUC
timeFrame: 30 days
measure: Pharmacokinetic Assessments: Clearance
timeFrame: 30 days
measure: Pharmacokinetic Assessments: volume of distribution
timeFrame: 30 days
measure: Pharmacodynamic Assessments: Assessments of TTR stabilization will be listed and summarized by part, treatment, and time point using appropriate descriptive statistics.
timeFrame: 30 days
measure: Pharmacodynamic Assessments: Western blot
timeFrame: 30 days
measure: Pharmacodynamic Assessments: prealbumin
timeFrame: 30 days
measure: Food effect: AUC
timeFrame: 30 days
measure: Food effect: Cmax
timeFrame: 30 days
Other outcomes:
Locations:
city: Tempe
country: United States
facility: Celerion
state: Arizona
Study officials:
affiliation: Celerion
name: Terry O'Reilly, M.D.
role: PRINCIPAL_INVESTIGATOR
References: