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A Study of AT-02 in Subjects With Systemic Amyloidosis.

Primary Purpose

Amyloidosis; Systemic

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AT02
Sponsored by
Attralus, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyloidosis; Systemic

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Subject understands the study procedures and can give signed informed consent. Subject is willing and able to comply with this protocol and will be available for the entire duration of the study. Subject must have a confirmed diagnosis of SA per the diagnostic criteria specified in the parent study protocol. Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02. AT02-001 Part 2: a. Subjects must have completed the last follow-up visit in AT02-001 Part 2 without significant adverse events, as determined by the Investigator. AT02-001 Part 3: a. Subjects must have completed the post-treatment imaging studies in AT02-001Part 3 (e.g., CMR, echocardiogram) without significant AEs in the parent study as determined by the Investigator. Must continue to satisfy the eligibility criteria in the parent study protocol for WOCBP, WONCBP, or male participants Exclusion Criteria: Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period. Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis. Has acquired any new, clinically significant underlying illness since enrollment in the parent study. Has any clinically significant worsening of organ function associated with underlying SA or clinically significant change in concomitant medications for the treatment of SA since enrollment in the parent study. Estimated glomerular filtration (eGFR) ≤30 mL/min/1.73 m2. Currently using any prohibited concomitant medications. Any contraindication to MRI or MRI contrast. Is currently participating in an interventional clinical study or has participated in another clinical study (other than AT02-001) within the last four (4) weeks or within five (5) half-lives of the prior study treatment, whichever is longer.

Sites / Locations

  • Midwest Heart and Vascular
  • Johns Hopkins
  • Cleveland Clinic
  • OHSU (Oregon Health & Science University)
  • Penn Presbyterian Medical Center
  • Princess Alexandra HospitalRecruiting
  • Flinders Medical CentreRecruiting
  • Box Hill HospitalRecruiting
  • Royal Perth Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

A (AT-02)

Arm Description

Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations).

Outcomes

Primary Outcome Measures

Incidence, frequency, and severity of Treatment-emergent adverse events (TEAEs) as assessed National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0)
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results

Secondary Outcome Measures

To assess PK of AT-02 during long-term administration
Parameter: maximum observed concentration of AT-02 (Cmax)
To assess PK of AT-02 during long-term administration
Parameter: time to maximum observed AT-02 concentration (Tmax)
To assess PK of AT-02 during long-term administration
Parameter: AUClast
To assess PK of AT-02 during long-term administration
Parameter: AUCinf
To assess PK of AT-02 during long-term administration
Parameter: volume of distribution at steady state (Vss)
To assess PK of AT-02 during long-term administration
Parameter: total body clearance (CL) of AT-02
To assess PK of AT-02 during long-term administration
Parameter: AT-02 half-life (t½)
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum N-terminal prohormone of brain natriuretic peptide (NT-proBNP)
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum High-sensitivity cardiac troponin T (hsTnT)
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Biomarkers include serum Urine albumin creatinine ratio (UACR)
Serial cardiac magnetic resonance assessments of systemic amyloidosis

Full Information

First Posted
June 29, 2023
Last Updated
October 19, 2023
Sponsor
Attralus, Inc.
Collaborators
Novotech (Australia) Pty Limited
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1. Study Identification

Unique Protocol Identification Number
NCT05951049
Brief Title
A Study of AT-02 in Subjects With Systemic Amyloidosis.
Official Title
A Phase 2, Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of AT-02
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 21, 2023 (Actual)
Primary Completion Date
February 28, 2026 (Anticipated)
Study Completion Date
February 28, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Attralus, Inc.
Collaborators
Novotech (Australia) Pty Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02. AT-02 is an investigational medicinal product being developed to treat systemic amyloidosis.
Detailed Description
The study will enroll subjects with systemic amyloidosis who have participated in AT02-001 study. The study includes screening period (56 days), treatment period (week 104), follow up (week 112). The total duration of participant in study is up to 120 weeks. A Safety Review Committee (SRC) will periodically convene and review all available clinical and laboratory data during the study. A single SRC will monitor safety across all AT-02 studies to ensure that safety signals are assessed in aggregate.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyloidosis; Systemic

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
120 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
A (AT-02)
Arm Type
Experimental
Arm Description
Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations).
Intervention Type
Drug
Intervention Name(s)
AT02
Intervention Description
Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use
Primary Outcome Measure Information:
Title
Incidence, frequency, and severity of Treatment-emergent adverse events (TEAEs) as assessed National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0)
Time Frame
Up to 112 weeks
Title
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
Time Frame
Up to 112 weeks
Secondary Outcome Measure Information:
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: maximum observed concentration of AT-02 (Cmax)
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: time to maximum observed AT-02 concentration (Tmax)
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: AUClast
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: AUCinf
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: volume of distribution at steady state (Vss)
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: total body clearance (CL) of AT-02
Time Frame
Up to 112 weeks
Title
To assess PK of AT-02 during long-term administration
Description
Parameter: AT-02 half-life (t½)
Time Frame
Up to 112 weeks
Title
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
Description
The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
Time Frame
Up to 112 weeks
Title
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Description
Biomarkers include serum N-terminal prohormone of brain natriuretic peptide (NT-proBNP)
Time Frame
Up to 112 weeks
Title
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Description
Biomarkers include serum High-sensitivity cardiac troponin T (hsTnT)
Time Frame
Up to 112 weeks
Title
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Description
Biomarkers include serum Urine albumin creatinine ratio (UACR)
Time Frame
Up to 112 weeks
Title
Serial cardiac magnetic resonance assessments of systemic amyloidosis
Time Frame
Up to 112 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subject understands the study procedures and can give signed informed consent. Subject is willing and able to comply with this protocol and will be available for the entire duration of the study. Subject must have a confirmed diagnosis of SA per the diagnostic criteria specified in the parent study protocol. Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02. AT02-001 Part 2: a. Subjects must have completed the last follow-up visit in AT02-001 Part 2 without significant adverse events, as determined by the Investigator. AT02-001 Part 3: a. Subjects must have completed the post-treatment imaging studies in AT02-001Part 3 (e.g., CMR, echocardiogram) without significant AEs in the parent study as determined by the Investigator. Must continue to satisfy the eligibility criteria in the parent study protocol for WOCBP, WONCBP, or male participants Exclusion Criteria: Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period. Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis. Has acquired any new, clinically significant underlying illness since enrollment in the parent study. Has any clinically significant worsening of organ function associated with underlying SA or clinically significant change in concomitant medications for the treatment of SA since enrollment in the parent study. Estimated glomerular filtration (eGFR) ≤30 mL/min/1.73 m2. Currently using any prohibited concomitant medications. Any contraindication to MRI or MRI contrast. Is currently participating in an interventional clinical study or has participated in another clinical study (other than AT02-001) within the last four (4) weeks or within five (5) half-lives of the prior study treatment, whichever is longer.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Scott Stephens
Phone
+1-321-228-7400
Email
sstephens@attralus.com
First Name & Middle Initial & Last Name or Official Title & Degree
Deepika Aggarwal
Phone
3 9960 7997
Ext
+61
Email
deepika.aggarwal@novotech-cro.com
Facility Information:
Facility Name
Midwest Heart and Vascular
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66211
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vasvi Singh, Dr
Email
Vasvi.singh@hcahealthcare.com
Facility Name
Johns Hopkins
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Joban Vaishnav, MD
Email
jvaishn1@jhmi.edu
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mazen Hanna, Dr
Email
hannam@ccf.org
Facility Name
OHSU (Oregon Health & Science University)
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ahmad Masri
Email
masria@ohsu.edu
Facility Name
Penn Presbyterian Medical Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Drachman, Dr
Email
drachman@pennmedicine.upenn.edu
Facility Name
Princess Alexandra Hospital
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dr Dariusz Korczyk
First Name & Middle Initial & Last Name & Degree
Dr Dariusz Korczyk
Facility Name
Flinders Medical Centre
City
Bedford Park
State/Province
South Australia
ZIP/Postal Code
5042
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Joseph Selvanayagam, Prof
Facility Name
Box Hill Hospital
City
Box Hill
State/Province
Victoria
ZIP/Postal Code
3128
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dr Simon Gibbs
First Name & Middle Initial & Last Name & Degree
Dr Simon Gibbs
Facility Name
Royal Perth Hospital
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Graham Hillis, Prof

12. IPD Sharing Statement

Learn more about this trial

A Study of AT-02 in Subjects With Systemic Amyloidosis.

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