Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis
AT02-001
A Three-part, Phase 1, Single-ascending, and Multiple-ascending Dose Escalation Study in Healthy Volunteers and Subjects With Systemic Amyloidosis to Assess the Safety, Tolerability, and Pharmacokinetics of AT-02
1 other identifier
interventional
100
2 countries
11
Brief Summary
This is a multicenter, international, three-part, Phase 1 study designed to evaluate the safety, tolerability, and PK of rising single doses of AT-02 in healthy volunteers and in subjects with systemic amyloidosis and to assess the safety, tolerability, and PK of multiple doses of AT-02 in subjects with systemic amyloidosis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Sep 2022
Typical duration for phase_1
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 23, 2022
CompletedFirst Posted
Study publicly available on registry
August 30, 2022
CompletedStudy Start
First participant enrolled
September 1, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2025
CompletedApril 3, 2024
April 1, 2024
2.5 years
August 23, 2022
April 2, 2024
Conditions
Outcome Measures
Primary Outcomes (4)
Incidence and severity of treatment-emergent adverse events (TEAEs) from Day 1 to end of study (EOS).
Safety will be assessed by review of clinical laboratory parameters and incidence and severity of TEAEs (graded using Common Terminology Criteria for Adverse Events (CTCAE) version 5).
Up to 57+/-7 days
Incidence of dose-limiting toxicities (DLTs) in subjects with systemic amyloidosis.
A DLT is defined as any related TEAE with a National Cancer Institute (NCI) CTCAE version 5.0 Grade ≥3 which also represents a shift from Baseline clinical status of \>1 NCI CTCAE Grade.
Up to 85+/-7 Days
Incidence and frequency of abnormal and clinically significant abnormal clinical laboratory parameter values.
Up to 85+/-7 Days
Incidence of treatment-emergent anti-drug antibodies (ADA)
The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
Up to 85+/-7 Days
Secondary Outcomes (6)
To determine the plasma pharmacokinetics (PK) profile of AT-02
Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02
Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02
Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02
Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02
Up to 85+/-7 Days
- +1 more secondary outcomes
Study Arms (4)
Part 1 AT-02
EXPERIMENTALPart 1 enrolling Healthy Volunteers (Randomised, Double-blind) Drug: AT-02 Dosage: 30mg to 1000mg Dosage Form \& Route of Admin: Solution for IV Infusion
Part 1 Placebo
PLACEBO COMPARATORPart 1 enrolling Healthy Volunteers (Randomised, Double-blind) Dosage Form \& Route of Admin: Normal Saline Solution for IV Infusion
Part 2 AT-02
EXPERIMENTALPart 2 enrolling Systemic Amyloidosis Participants (Single-Arm, Open-label) Drug: AT-02 Dosage: 300mg to 4000mg Frequency: Single Dose Dosage Form \& Route of Admin: Solution for IV Infusion
Part 3 AT-02
EXPERIMENTALPart 3 enrolling Systemic Amyloidosis Participants (Single-Arm, Open-label) Drug: AT-02 Dosage: Dose levels will be determined by the SRC. The starting dose in Part 3 will be determined by the SRC based on all available safety, tolerability, PK, and PD data from all prior cohorts Frequency: Multiple Doses Dosage Form \& Route of Admin: Solution for IV Infusion
Interventions
Eligibility Criteria
You may qualify if:
- Eligibility Criteria for Healthy Volunteers:
- Healthy volunteers are eligible to be included in the study only if all the following criteria apply:
- Understands the study procedures and can give signed informed consent
- Male or female between \>18 and \<56 years of age.
- Willing and able to comply with this protocol and informed consent and be available for the entire duration of the study.
- Willing to abstain from alcohol and strenuous physical activity (i.e., strenuous or unaccustomed weightlifting, running, bicycling, etc.) from 48 hours prior to study treatment administration until discharge from the clinical unit and prior to each outpatient visit.
- In good general health, determined by no clinically significant findings in the opinion of the Investigator from medical history, physical examination, 12-lead electrocardiogram (ECG), clinical laboratory findings, and vital signs at Screening and Phase 1 unit Check-in.
- Has body mass index (BMI) of 18 to 32 kg/m2, inclusive.
- Women of childbearing potential (WOCBP)
- WOCBP must have a negative serum or urine pregnancy test within 24 hours prior to the start of study drug.
- Must not be breastfeeding, lactating, or planning a pregnancy during the study period.
- WOCBP who are not exclusively in same-sex relationships must agree to remain abstinent (complete avoidance of heterosexual intercourse) or use adequate contraceptive methods, defined as use of a condom by the male partner combined with use of a highly effective method of contraception by the female partner, during the treatment period and for at least 105 days after the last dose of study intervention.
- Postmenopausal females:
- a) Postmenopausal females under the age of 55 years must have a documented serum follicle stimulating hormone (FSH) level \>40 mIU/mL to confirm menopause
- Women of non-childbearing potential (WONCBP) and female participants with vasectomized male partners:
- +53 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Attralus, Inc.lead
- Novotech (Australia) Pty Limitedcollaborator
Study Sites (11)
Midwest Heart and Vascular
Overland Park, Kansas, 66211, United States
Johns Hopkins
Baltimore, Maryland, 21287, United States
St. Luke's Hospital of Kansas City
Kansas City, Missouri, 64111, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
OHSU (Oregon Health & Science University)
Portland, Oregon, 97239, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104, United States
Q-Pharm Pty Ltd
Herston, Queensland, 4006, Australia
Princess Alexandra Hospital
Woolloongabba, Queensland, 4102, Australia
Flinders Medical Centre
Bedford Park, South Australia, 5042, Australia
Box Hill Hospital
Box Hill, Victoria, 3128, Australia
Royal Perth Hospital
Perth, Western Australia, 6000, Australia
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Masking Details
- Double blind (Part 1) Open Label (Part 2) Open Label (Part 3)
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 23, 2022
First Posted
August 30, 2022
Study Start
September 1, 2022
Primary Completion
March 1, 2025
Study Completion
March 1, 2025
Last Updated
April 3, 2024
Record last verified: 2024-04