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Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis

PHASE1RECRUITING

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.

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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.

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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

Study type

TREATMENT

phase

Phase

    PHASE1

trial

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

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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/ArmIntervention/Treatment
EXPERIMENTAL: Part 1 AT-02
  • Part 1 enrolling Healthy Volunteers (Randomised, Double-blind) Drug: AT-02 Dosage: 30mg to 1000mg Dosage Form \& Route of Admin: Solution for IV Infusion
DRUG: AT-02
  • AT-02 via IV infusion
PLACEBO_COMPARATOR: Part 1 Placebo
  • Part 1 enrolling Healthy Volunteers (Randomised, Double-blind) Dosage Form \& Route of Admin: Normal Saline Solution for IV Infusion
OTHER: AT-02 (Placebo)
  • Normal saline solution via IV infusion
EXPERIMENTAL: Part 2 AT-02
  • Part 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
DRUG: AT-02
  • AT-02 via IV infusion
EXPERIMENTAL: Part 3 AT-02
  • Part 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
DRUG: AT-02
  • AT-02 via IV infusion

What is the study measuring?

Primary outcome

Primary Outcome MeasurePrimary Outcome DescriptionPrimary 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 SpecifiedUp 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 MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: time to maximum observed AT-02 concentration (Tmax).Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: maximum observed concentration of AT-02 (Cmax).Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: area under the plasma concentration versus time curve (AUC).Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: volume of distribution at steady state (Vss).Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: total body clearance (CL) of AT-02.Up to 85+/-7 Days
To determine the plasma pharmacokinetics (PK) profile of AT-02Parameter: AT-02 half-life (t1/2).Up to 85+/-7 Days

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References

Clinical Trials Gov: Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis

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