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Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis
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.
Study details:
Systemic amyloidosis is an incurable disease, and about 20% of patients with cardiac or advanced kidney involvement experience early deaths (\<1 year). Despite recent progress in proteasome inhibitors, chemotherapies, and immunotherapies that target plasma cells have greatly improved the prognosis of patients with systemic amyloidosis, median survival remains low at approximately five years. AT-02 (INN: not yet available) is a full-length, humanized, recombinant immunoglobulin 1 (IgG1)-like glycoprotein monoclonal antibody (mAb) that is being developed to treat systemic amyloidosis.
This is a three-part, Phase 1 study designed to evaluate the safety, tolerability, and PK of rising single doses of AT-02 in healthy volunteers (HV) and in subjects with systemic amyloidosis (SA) and to assess the safety, tolerability, and PK of multiple doses of AT-02 in subjects with systemic amyloidosis. Part 1 is a double-blind, single-center, single-ascending dose escalation study in HV to assess the safety, tolerability, and PK of AT-02. Healthy volunteers between 18 to 56 years of will be enrolled in the Part 1 study.
Part 2 is an open-label, single-ascending dose escalation study in subjects with systemic amyloidosis to assess the safety, tolerability, and PK of AT-02 and to identify a maximum tolerated dose (MTD). Subjects with SA over 18 years of age will be involved in the Part 2 study. Part 3 is an open-label, multiple-ascending dose, dose escalation study in subjects with systemic amyloidosis to assess the safety, tolerability, PK, PD, and clinical activity of multiple doses of AT-02.
Subjects with SA ≥18 and ≤85 years of age will be involved in the Part 3 study.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Eligibility
Age eligible for study : 18 and older
Healthy volunteers accepted : Yes
Gender eligible for study: All
Things to know
Study dates
Study start: 2022-09-01
Primary completion: 2025-03-01
Study completion finish: 2025-03-01
Study type
TREATMENT
Phase
PHASE1
Trial ID
NCT05521022
Intervention or treatment
DRUG: AT-02
OTHER: AT-02 (Placebo)
Conditions
- • Amyloidosis; Systemic
Find a site
Closest Location:
Princess Alexandra Hospital
Research sites nearby
Select from list below to view details:
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Flinders Medical Centre
Bedford Park, South Australia, Australia
Box Hill Hospital
Box Hill, Victoria, Australia
Royal Perth Hospital
Perth, Western Australia, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: Part 1 AT-02
| DRUG: AT-02
|
PLACEBO_COMPARATOR: Part 1 Placebo
| OTHER: AT-02 (Placebo)
|
EXPERIMENTAL: Part 2 AT-02
| DRUG: AT-02
|
EXPERIMENTAL: Part 3 AT-02
| DRUG: AT-02
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| 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. | Not Specified | 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 outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: time to maximum observed AT-02 concentration (Tmax). | Up to 85+/-7 Days |
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: maximum observed concentration of AT-02 (Cmax). | Up to 85+/-7 Days |
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: area under the plasma concentration versus time curve (AUC). | Up to 85+/-7 Days |
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: volume of distribution at steady state (Vss). | Up to 85+/-7 Days |
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: total body clearance (CL) of AT-02. | Up to 85+/-7 Days |
| To determine the plasma pharmacokinetics (PK) profile of AT-02 | Parameter: AT-02 half-life (t1/2). | Up to 85+/-7 Days |
Frequently Asked Questions
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