NCT03678259

124I-p5+14 Injection Safety in Subjects With Systemic Amyloidosis

Evaluation of 124I-p5+14 Injection as an Imaging Agent for the Detection of Systemic Amyloidosis

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: University of Tennessee Graduate School of Medicine · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a single-center, exploratory, Phase 1 Positron Emission Tomography/x-ray Computed Tomography (PET/CT) imaging study to detect amyloidosis that will enroll patients with a confirmed diagnosis of systemic amyloidosis. The purpose of this exploratory trial is to assess the safety and efficacy of 124I-p5+14 Injection at a single-injection dose adequate for imaging amyloid deposits by using PET/CT imaging in subjects with confirmed systemic Immunoglobulin Light Chain-associated Amyloidosis (AL), Transthyretin-associated Amyloidosis (ATTR), Leukocyte Chemotactic Factor 2-associated Amyloidosis (ALect2) as well as other types.

Detailed description

The rationale for this study is the discovery of a synthetic polypeptide, designated p5+14, a synthetic 45 amino acid peptide that binds many forms of amyloid, including human AL-, ATTR- and ALect2-associated amyloid, as well as human and murine serum amyloid protein A-associated (AA) amyloid. In preclinical studies, using SPECT and PET imaging, as well as microautoradiography, it has been shown that radioiodinated p5+14 binds rapidly and specifically to all amyloid deposits in abdominothoracic organs and tissues. This is a single site, exploratory, open-label Phase I PET/CT imaging and dosimetry study. The investigational drug product (designated 124I-p5+14 Injection) is an amyloid-reactive synthetic peptide, p5+14 (also known as APi1832), radiolabeled with iodine-124 (I-124 or 124I). All patients enrolled in this exploratory trial will be outpatients with a confirmed diagnosis of systemic amyloidosis. The first three patients enrolled in the trial (Part 1) will take part in a dose-escalation dosimetry study and will receive a single intravenous (IV) dose of 11.1 Megabecquerel (MBq) (0.3 millicuries (mCi); n = 1), 37 MBq (1 mCi; n = 1) or 74 MBq (2 mCi; n = 1) of 124I-p5+14 Injection for the purpose of determining estimates of organ-associated and whole body radioactive dosimetry. The trial then will be opened to include another 54 patients who will receive a single IV bolus injection of 2 mCi 124I-p5+14 Injection. Every patient participating will receive \< 2 mg of peptide p5+14. The study comprises five parts. In Part 2, the trial will enroll SA patients with confirmed AL, ATTR, ALECT2, or other forms of amyloidosis. In Part 3, the trial will study asymptomatic subjects with genetically confirmed mutation of the transthyretin gene. Part 4 will include five healthy control subjects. Part 5 will recruit previously enrolled subjects from Parts 2 or 3 who received a 2 mC dose of 124I-p5+14 Injection and had images confirming the presence of abnormal amyloid deposits for a second exposure to 124I p5+14 Injection and second PET/CT scan.

Conditions

• Systemic Amyloidosis

Interventions

Type	Name	Description
DRUG	124I-p5+14 Injection	124I-p5+14 Injection which is a formulation of a synthetic, all natural, 45 amino acid peptide (MW = 4766.4) with a net +12 positive charge

Timeline

• Start date: 2018-10-01
• Primary completion: 2021-10-01
• Completion: 2021-10-01
• First posted: 2018-09-19
• Last updated: 2022-03-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study