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This is a single center Phase 1 study is to evaluate the biodistribution of radiolabeled AT-03 in patients with systemic amyloidosis.
Eligible subjects will receive a single dose of radiolabeled AT-03, a fusion protein of serum amyloid P and a single chain Fc receptor. Repeat Positron Emission Tomography/Computed Tomography (PET/CT) scans will be performed over a ~7 day period to assess the biodistribution of AT-03 to amyloid-containing tissues in patients with systemic amyloidosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Injection of I-124 AT03. | Experimental | Single arm only, no placebo or comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 124I-AT03 | Drug | IV injection of the radiolabeled compound on day 1 followed by Positron Emission Tomography/Computed Tomography (PET/CT) scanning on Days 2, 4 and 8 to. The first 3 subjects enrolled will have two additional scans on Day 1. |
| Measure | Description | Time Frame |
|---|---|---|
| To measure the tissue distribution of 124I-AT-03 in select organs in patients with systemic amyloidosis as shown in the Positron Emission Tomography/Computed Tomography (PET/CT scans) series. | Visual determination of 124I-AT-03 uptake in the heart, kidneys, liver and spleen (positive or negative) at Days 4 and 6. | Day 4. |
| To measure the tissue distribution of 124I-AT-03 in select organs in patients with systemic amyloidosis as shown in the Positron Emission Tomography/Computed Tomography (PET/CT scans) series. | Visual determination of 124I-AT-03 uptake in the heart, kidneys, liver and spleen (positive or negative) at Days 4 and 6. | Day 6. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-related adverse events. | Incidence of treatment-emergent adverse events from Day 1 to Day 28. | Assessed from time of consent through the day 28 follow-up phone call. |
| Number of participants with abnormal laboratory test results. |
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Inclusion Criteria:
Understands the study procedures and capable of giving signed informed consent, as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Male or female ≥18 years of age.
Has a confirmed diagnosis of Amyloid Light-Chain (AL), Transthyretin Amyloidosis (ATTR), or Leukocyte chemotactic factor 2 amyloidosi (ALECT2), or any other forms of systemic amyloidosis based on any one of the following:
Has known involvement of at least one thoracoabdominal organ (excluding peripheral nervous system) by clinical history (e.g., imaging consistent with amyloid deposition, organ biopsy, elevated amyloid-related biomarkers, etc.).
For women of childbearing potential: agreement to remain as abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of <1% per year during the treatment period and for at least 90 days after the last dose of study intervention.
For men: agreement to remain abstinent or use contraceptive measures and agreement to refrain from donating sperm, as defined below:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gregory Bell, MD | Attralus, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Tennessee Medical Center | Knoxville | Tennessee | 37920 | United States |
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| ID | Term |
|---|---|
| D000075363 | Immunoglobulin Light-chain Amyloidosis |
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| ID | Term |
|---|---|
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D000686 | Amyloidosis |
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Change from Baseline in clinical laboratory values at Days 4 and 6. |
| Assessed from time of consent through Day 6. |
| Whole blood radioactivity of 124I-AT-03 in subjects with systemic amyloidosis. | Whole body radiation dose by dosimetry in all enrolled subjects. Whole blood radioactivity (corrected for radioactive decay). | Days 1, 4, and 6. |
| Selected tissue-bound half-life of 124I-AT-03 in subjects with systemic amyloidosis. | Organ specific radioactivity for the heart, kidneys, liver, and spleen at Days 4 and 6. | Days 4 and 6. |
| D057165 |
| Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D010265 | Paraproteinemias |