Amyloidosis; SystemicPhase 2NCT05951049
A Study of AT-02 in Subjects With Systemic Amyloidosis.
A Phase 2 study testing AT02 in people with Amyloidosis; Systemic, run by Attralus, Inc. at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Amyloidosis; Systemic
- What is being tested?
- AT02
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 4 sitesUnited States
- Study dates
- 21 Sep 2023to 28 Sep 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Has a confirmed diagnosis of AL amyloidosis and meets the criteria below:
- 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
- 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
- Either
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- Screening eGFR ≥20 and ≤75 mL/min/1.73m2 based on CKD-EPI equation, OR
- 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
- Women of childbearing potential (WOCBP):
- WOCBP must have a negative serum or urine pregnancy test within 24 hours prior to the start of study treatment.
- Must not be breastfeeding, lactating, or planning a pregnancy during the study period.
- 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.
- Women of non-childbearing potential (WONCBP), and female participants with vasectomized male partners:
- 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.
- Postmenopausal females must have a documented serum FSH level >40 mIU/mL at Screening to confirm menopause.
- Male participants:
- 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.
- 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.
- Male participants must not donate sperm for at least 165 days after the last dose of study intervention.
- 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.
Who cannot join
- Receiving hemodialysis or peritoneal dialysis.
- Myocardial infarction within 3 months of Screening.
- New York Heart Association Class IV heart failure.
- Kidney disease not caused by AL amyloidosis.
- Respiratory insufficiency requiring oxygen therapy.
- Currently receiving: melphalan, bortezomib, thalidomide, lenalidomide, or cyclophosphamide.
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- Currently receiving unfractionated heparin or heparin analogs (e.g., enoxaparin, dalteparin).
- 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.
- Uncontrolled or active infection.
- Autoimmune disease requiring treatment with immunosuppressive/modulating treatment in the last year.
- History of solid organ transplantation.
- 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.
- 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.
- Screening alanine transaminase (ALT) or aspartate transaminase (AST) is >2.5x upper limit of normal (ULN).
- Screening estimated creatinine clearance/eGFR using the CKD-EPI equation is <20 mL/min/1.73 m2.
- 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.
- Any contraindication to MRI.
- Hypersensitivity to AT-02 or any of its excipients.
- Brain MRI performed within 6 months of Screening, or during the Screening period, that shows evidence of any of the following:
- Acute or sub-acute hemorrhage.
- 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).
- Greater than 4 microhemorrhages (defined as <1 cm in diameter on T2* sequence or SWI).
- Cortical infarct (defined as >1.5 cm in diameter; irrespective of anatomic location).
- >1 lacunar infarct (defined as <1.5 cm in diameter).
- Superficial siderosis.
- Any finding that, in the opinion of the Investigator, might pose a risk to the participant.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Cleveland Clinic
Cleveland, Ohio, 44195
Kansas City
Kansas City, Kansas, 64111
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104
OHSU (Oregon Health & Science University)
Portland, Oregon, 97239
About this study
Official title: A Phase 2, Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of AT-02
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.
Read the detailed description
The study will enroll subjects with systemic amyloidosis who have participated in AT02-001 study and will also directly enroll AL participants with renal disease who did not participate in study AT02-001.
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.
- Conditions
- Amyloidosis; Systemic
- Being tested
- AT02
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AT02Drug
Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use
| Study group | Role | What they receive |
|---|---|---|
| A (AT-02) | Experimental | Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations). Receives: Drug: AT02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Up to 112 weeks
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
Measured: Up to 112 weeks
Secondary outcomes (12)
To assess PK of AT-02 during long-term administration
Parameter: maximum observed concentration of AT-02 (Cmax)
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: time to maximum observed AT-02 concentration (Tmax)
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: AUClast
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: AUCinf
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: volume of distribution at steady state (Vss)
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: total body clearance (CL) of AT-02
Measured: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Parameter: AT-02 half-life (t½)
Measured: Up to 112 weeks
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
The number and percentage of subjects who develop detectable ADA will be summarized by dose cohort.
Measured: Up to 112 weeks
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)
Measured: Up to 112 weeks
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)
Measured: Up to 112 weeks
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)
Measured: Up to 112 weeks
Serial cardiac magnetic resonance assessments of systemic amyloidosis
Measured: Up to 112 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2023
- Study start21 Sep 2023
- Primary completion28 Sep 2026 (estimated)
- Study completion28 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloidosis; SystemicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Attralus, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05951049
- Sponsor's protocol ID
- AT02-003
- Status last verified
- May 2026
- First submitted
- 29 Jun 2023
- Last updated
- 6 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of AT-02
- ClinicalTrials.gov ID
- NCT05951049
- Acronym / sponsor ID
- AT02-003
- Conditions
- Amyloidosis; Systemic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Attralus, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Study start
- 21 Sep 2023 (actual)
- Primary completion
- 28 Sep 2026 (estimated)
- Study completion
- 28 Sep 2026 (estimated)
- First posted
- 18 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 and will also directly enroll AL participants with renal disease who did not participate in study AT02-001.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A (AT-02) | Experimental | Drug: AT02Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations). |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- 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.
- Has a confirmed diagnosis of AL amyloidosis and meets the criteria below:
- <!-- -->
- Note: Participants may meet both eGFR and proteinuria criteria
Key exclusion criteria (26 listed)
- Receiving hemodialysis or peritoneal dialysis.
- Myocardial infarction within 3 months of Screening.
- New York Heart Association Class IV heart failure.
- Kidney disease not caused by AL amyloidosis.
- Respiratory insufficiency requiring oxygen therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AT02Dosage Form: Solution for injection/infusion Dosage level: Different dose levels of AT02 Route of Administration: Intravenous use | Drug | A (AT-02) |
Primary outcome
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
To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
Time frame: Up to 112 weeks
12 secondary outcomes
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
To assess PK of AT-02 during long-term administration
Time frame: Up to 112 weeks
Incidence of treatment-emergent Anti-drug antibodies (ADAs)
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Time frame: Up to 112 weeks
To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
Time frame: Up to 112 weeks
Serial cardiac magnetic resonance assessments of systemic amyloidosis
Time frame: Up to 112 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2026.
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Amyloidosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AT02 in people with Amyloidosis; Systemic, run by Attralus, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2026.