Clinical trial NCT05951049: 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
Overall status: RECRUITING
Phase: PHASE2
Conditions: Amyloidosis; Systemic
Lead sponsor: Attralus, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Novotech (Australia) Pty Limited
First posted: 2023-07-18
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

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.

Primary outcomes:
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)
timeFrame: Up to 112 weeks
measure: To assess the safety and tolerability of AT-02 through change from baseline in clinical laboratory results
timeFrame: Up to 112 weeks
Secondary outcomes:
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: To assess PK of AT-02 during long-term administration
timeFrame: Up to 112 weeks
measure: Incidence of treatment-emergent Anti-drug antibodies (ADAs)
timeFrame: Up to 112 weeks
measure: To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
timeFrame: Up to 112 weeks
measure: To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
timeFrame: Up to 112 weeks
measure: To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers
timeFrame: Up to 112 weeks
measure: Serial cardiac magnetic resonance assessments of systemic amyloidosis
timeFrame: Up to 112 weeks
Other outcomes:
Locations:
city: Overland Park
country: United States
facility: Midwest Heart and Vascular
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: OHSU (Oregon Health & Science University)
state: Oregon
city: Philadelphia
country: United States
facility: Penn Presbyterian Medical Center
state: Pennsylvania
city: Woolloongabba
country: Australia
facility: Princess Alexandra Hospital
state: Queensland
city: Bedford Park
country: Australia
facility: Flinders Medical Centre
state: South Australia
city: Box Hill
country: Australia
facility: Box Hill Hospital
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: London
country: United Kingdom
facility: Royal Free London Nhs Foundation Trust Royal Free Hospital
Study officials:
References: