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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05951049




Registration number
NCT05951049
Ethics application status
Date submitted
29/06/2023
Date registered
18/07/2023
Date last updated
30/01/2024

Titles & IDs
Public title
A Study of AT-02 in Subjects With Systemic Amyloidosis.
Scientific title
A Phase 2, Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of AT-02
Secondary ID [1] 0 0
AT02-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyloidosis; Systemic 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AT02

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


Treatment: Drugs: AT02
Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
Up to 112 weeks
Primary outcome [2] 0 0
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
Timepoint [2] 0 0
Up to 112 weeks
Secondary outcome [1] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [1] 0 0
Up to 112 weeks
Secondary outcome [2] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [2] 0 0
Up to 112 weeks
Secondary outcome [3] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [3] 0 0
Up to 112 weeks
Secondary outcome [4] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [4] 0 0
Up to 112 weeks
Secondary outcome [5] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [5] 0 0
Up to 112 weeks
Secondary outcome [6] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [6] 0 0
Up to 112 weeks
Secondary outcome [7] 0 0
To assess PK of AT-02 during long-term administration
Timepoint [7] 0 0
Up to 112 weeks
Secondary outcome [8] 0 0
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
Timepoint [8] 0 0
Up to 112 weeks
Secondary outcome [9] 0 0
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Timepoint [9] 0 0
Up to 112 weeks
Secondary outcome [10] 0 0
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Timepoint [10] 0 0
Up to 112 weeks
Secondary outcome [11] 0 0
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Timepoint [11] 0 0
Up to 112 weeks
Secondary outcome [12] 0 0
Serial cardiac magnetic resonance assessments of systemic amyloidosis
Timepoint [12] 0 0
Up to 112 weeks

Eligibility
Key inclusion criteria
1. Subject understands the study procedures and can give signed informed consent.
2. Subject is willing and able to comply with this protocol and will be available for the entire duration of the study.
3. Subject must have a confirmed diagnosis of SA per the diagnostic criteria specified in the parent study protocol.
4. Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02.
5. 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.
6. 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.
7. Must continue to satisfy the eligibility criteria in the parent study protocol for WOCBP, WONCBP, or male participants
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period.
2. Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis.
3. Has acquired any new, clinically significant underlying illness since enrollment in the parent study.
4. 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.
5. Estimated glomerular filtration (eGFR) =30 mL/min/1.73 m2.
6. Currently using any prohibited concomitant medications.
7. Any contraindication to MRI or MRI contrast.
8. 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/02/2026
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [2] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [3] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [4] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [2] 0 0
5042 - Bedford Park
Recruitment postcode(s) [3] 0 0
3128 - Box Hill
Recruitment postcode(s) [4] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Kansas
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United Kingdom
State/province [6] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Attralus, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Novotech (Australia) Pty Limited
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05951049
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Scott Stephens
Address 0 0
Country 0 0
Phone 0 0
+1-321-228-7400
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05951049