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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
Help: Registration number
NCT05951049
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
29/06/2023
Date registered
Help: Date registered
18/07/2023
Date last updated
Help: Date last updated
6/05/2026

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
Additional trial identifiers [1] 0 0
AT02-003
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Amyloidosis; Systemic 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or 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
Description of the comparator or control
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. Has a confirmed diagnosis of AL amyloidosis and meets the criteria below:

1. Histologic confirmation with a biopsy containing deposits of apple-green birefringent, Congophilic material or other amyloid staining (i.e., thioflavin T or sulfated alcian blue) with confirmatory immunohistochemistry or mass spectrometry, AND
2. May be receiving maintenance daratumumab and must have achieved and maintained a hematologic very good partial response (VGPR) or complete response (CR), have completed chemotherapy therapy (ie, melphalan, bortezomib, thalidomide, lenalidomide, or cyclophosphamide) and be at least 6 months from first hematologic response (CR or VGPR), AND
3. Either

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1. Screening eGFR =20 and =75 mL/min/1.73m2 based on CKD-EPI equation, OR
2. Proteinuria that is not improving (e.g., <25% reduction in urine protein creatinine ratio (UPCR) in the last 12 months or since hematologic response, whichever is shorter) with screening urine albumin creatinine ratio (UACR) >700 mg/g based on central lab assessment of first morning void urine collected at screening and confirmed by a separate Screening 24-hr urine protein >1.0gm/day; 24-hr urine protein may be repeated once during Screening.

Note: Participants may meet both eGFR and proteinuria criteria
4. Women of childbearing potential (WOCBP):

1. WOCBP must have a negative serum or urine pregnancy test within 24 hours prior to the start of study treatment.
2. Must not be breastfeeding, lactating, or planning a pregnancy during the study period.
3. 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.
5. Women of non-childbearing potential (WONCBP), and female participants with vasectomized male partners:

1. WONCBP must agree to remain abstinent (complete avoidance of intercourse) or the male partners of WONCBP participants must wear a condom to protect against the transfer of study intervention through bodily fluids during the treatment period and for at least 105 days after the last dose of study intervention.
2. Postmenopausal females must have a documented serum FSH level >40 mIU/mL at Screening to confirm menopause.
6. Male participants:

1. Male participants must inform their female sexual partners who are WOCBP of the contraceptive requirements of the protocol and are expected to adhere to using contraception with their partner.
2. Male participants with female sexual partners who are WOCBP 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 165 days after the last dose of study intervention.
3. Male participants must not donate sperm for at least 165 days after the last dose of study intervention.
4. Male participants in same-sex relationships or in relationships with WONCBP, must agree to remain abstinent (complete avoidance of intercourse) or use a condom to prevent exposure of the partner to study intervention through ejaculate/seminal fluid during the treatment period and for at least 165 days after the last dose of study intervention.
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
1. Receiving hemodialysis or peritoneal dialysis.
2. Myocardial infarction within 3 months of Screening.
3. New York Heart Association Class IV heart failure.
4. Kidney disease not caused by AL amyloidosis.
5. Respiratory insufficiency requiring oxygen therapy.
6. Currently receiving: melphalan, bortezomib, thalidomide, lenalidomide, or cyclophosphamide.
7. Currently receiving unfractionated heparin or heparin analogs (e.g., enoxaparin, dalteparin).
8. Active malignancy with exception of basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, curatively treated in situ cervical cancer, non-metastatic prostate adenocarcinoma stably managed on hormonal therapy by medical oncologist or for which appropriate management is observation alone.
9. Uncontrolled or active infection.
10. Autoimmune disease requiring treatment with immunosuppressive/modulating treatment in the last year.
11. History of solid organ transplantation.
12. Suspected or known substance abuse disorder(s), serious psychiatric or any other medical condition, which, in the opinion of the Investigator, makes the participant unsuitable.
13. Has any concurrent illness that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the participant.
14. Screening alanine transaminase (ALT) or aspartate transaminase (AST) is >2.5x upper limit of normal (ULN).
15. Screening estimated creatinine clearance/eGFR using the CKD-EPI equation is <20 mL/min/1.73 m2.
16. Currently participating in an interventional clinical study or has participated in another interventional clinical study within the last four (4) weeks or within five (5) half-lives of the prior study treatment, whichever is longer. This exclusion does not apply to clinical studies in which the only investigational product is a diagnostic agent.
17. Any contraindication to MRI.
18. Hypersensitivity to AT-02 or any of its excipients.
19. Brain MRI performed within 6 months of Screening, or during the Screening period, that shows evidence of any of the following:

1. Acute or sub-acute hemorrhage.
2. Prior macrohemorrhage (defined as >1 cm in diameter on T2* sequence or susceptibility-weighted imaging (SWI)) or prior subarachnoid hemorrhage unless it can be documented that the finding is not due to an underlying structural or vascular abnormality (i.e., finding does not suggest participant is at risk of recurrent hemorrhage).
3. Greater than 4 microhemorrhages (defined as <1 cm in diameter on T2* sequence or SWI).
4. Cortical infarct (defined as >1.5 cm in diameter; irrespective of anatomic location).
5. >1 lacunar infarct (defined as <1.5 cm in diameter).
6. Superficial siderosis.
7. Any finding that, in the opinion of the Investigator, might pose a risk to the participant.

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
Active, not 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
28/09/2026
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
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
Ohio
Country [3] 0 0
United States of America
State/province [3] 0 0
Oregon
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania

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
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05951049