NCT05974579

Safety and Dosimetry of a New Radiotracer to Detect Misfolded SOD1 Associated With Amyotrophic Lateral Sclerosis

A Single Center, Open Label Study to Evaluate Biodistribution, Pharmacokinetics and Safety of [89Zr]Zr-DFO-AP-101 PET (Positron Emission Tomography) in Healthy Volunteers and Amyotrophic Lateral Sclerosis (ALS) Patients

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Université de Sherbrooke · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Accepted

Summary

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurodegenerative disease resulting in loss, primarily, of the motor neurons in the motor cortex, brainstem and spinal cord. It currently affects 3 of every 100,000 people in the US. Currently, there is no diagnostic tool for ALS, resulting in misdiagnosis and significant disease progression before formal diagnosis. An imaging test for early detection of ALS and for monitoring disease progression would have significant diagnostic and prognostic value. PET imaging with an appropriate radiotracer has great potential as a biomarker for ALS given that it would permit visualization of central nervous system (CNS) pathology in individuals living with the disease. To that extent, the primary goal of this phase I study is evaluating the safety and biodistribution of the new tracer \[89Zr\]Zr-DFO-AP-101 in healthy volunteers and ALS patients.

Detailed description

Background: Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurodegenerative disease resulting in loss, primarily, of the motor neurons in the motor cortex, brainstem and spinal cord. It currently affects 3 of every 100,000 people in the US. Currently, there is no diagnostic tool for ALS, resulting in misdiagnosis and significant disease progression before formal diagnosis. Design: This is a phase I clinical trial and a 2-part, single center, open label study in healthy volunteers (Part A) and confirmed ALS patients (Part B). The primary goal is evaluating the safety and biodistribution of the radiotracer \[89Zr\]Zr-DFO-AP-101 in healthy volunteers and ALS patients via PET/CT imaging. Objectives: The primary objectives of this study are: (Part A) To evaluate, by PET imaging, the safety, biodistribution and dosimetry of \[89Zr\]Zr-DFO-AP-101 in healthy men and women and in ALS patients Intervention and Follow-up: Following a screening visit, eligible participants will come to the research center for all study assessments. * On Day 0, a single intravenous dose of \[89Zr\]Zr-DFO-AP-101 40 MBq will be administrated and a 45 min whole body PET/CT acquisition will be performed at two hours post injection. Physical exam, ECG, vital signs and blood/urine samples will be collected. * Further PET acquisitions and same data/samples collection will be repeated at days 1, 3, 7 and 10 post-injection. * Participants will be contacted for a final follow-up visit approximately 14 days after injection.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	89Zr-DFO-AP-101	At Day 0, patients will receive one dose of the radiotracer. A PET/CT scan will be done 2h post-dose. At 1, 3, 7 and 10 days post-dose, a PET/CT scan will be repeated.

Timeline

• Start date: 2023-11-23
• Primary completion: 2024-12-30
• Completion: 2025-03-31
• First posted: 2023-08-03
• Last updated: 2025-01-28

Locations

1 site across 1 country: Canada