89Zr-DFO-AP-101 Imaging for ALS

Overseen By Sarah Côté-Bigras, MSc

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Université de Sherbrooke

Must not be taking: Biologics, Monoclonal antibodies

Disqualifiers: Pregnancy, Surgery, Psychiatric illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 7

Duration: 10 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new imaging tracer, 89Zr-DFO-AP-101, to help diagnose and track ALS, a disease affecting nerve cells in the brain and spinal cord. The researchers aim to determine the tracer's safety and distribution in the body during PET scans. The study includes two groups: healthy individuals and those with ALS. Individuals diagnosed with ALS who experience difficulty moving or breathing, but can lie down for 45 minutes, might be suitable for this trial. As a Phase 1 trial, this research focuses on understanding how the new tracer works in people, offering participants the opportunity to be among the first to use this innovative diagnostic tool.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What prior data suggests that this imaging technique is safe for ALS patients?

Research shows that the investigational imaging agent, 89Zr-DFO-AP-101, is safe and well-tolerated. In earlier studies with a similar compound, AP-101, used in patients with various types of ALS, safety goals were met, indicating it was generally well-received by patients. No serious side effects were reported, suggesting it does not cause significant harm.

This trial is in an early stage, so detailed safety information might be limited. However, the compound's success in passing safety checks in previous studies is encouraging. Participants in this trial will receive the treatment only once, reducing the risk of side effects. The safety record remains positive, but this study aims to gather more information. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for ALS, which focus mainly on symptom management with medications like riluzole or edaravone, the new approach with 89Zr-DFO-AP-101 involves imaging. This treatment is unique because it uses a zirconium-89-labeled compound to potentially enhance the visualization of disease activity in the brain. Researchers are excited about this technique because it could provide better insights into the progression of ALS, helping to tailor treatments more effectively and possibly lead to new therapeutic strategies.

What evidence suggests that this imaging technique is effective for ALS?

Research has shown that the radiotracer 89Zr-DFO-AP-101 could help detect ALS early. In studies with mice, this tracer successfully identified the misfolded proteins associated with ALS. This could aid in diagnosing the disease sooner and monitoring its progression. In this trial, both healthy participants and patients with ALS will receive 89Zr-DFO-AP-101 to evaluate its effectiveness as an imaging tool for detecting changes in the brain and spinal cord of people with ALS. By using this tracer, doctors might detect the disease before symptoms become severe. ^{4 5 6 7 8}

Who Is on the Research Team?

Eric E Turcotte, MD

Principal Investigator

Estrie University Integrated Health and Social Services Center - University Hospital of Sherbrooke

Brigitte Guérin, PhD

Principal Investigator

Estrie University Integrated Health and Social Services Center - University Hospital of Sherbrooke

Are You a Good Fit for This Trial?

This trial is for healthy adults over 50 and ALS patients aged 18 or older who can lie down for up to 45 minutes without help. Participants must not have other neurodegenerative diseases, severe psychiatric illnesses, recent blood transfusions, or be on certain respiratory support. Pregnant women and those not using contraception are excluded.

Inclusion Criteria

I do not have any neurological conditions.

Have venous access sufficient to allow for blood sampling

I am committed to following the study's procedures and can participate for its full duration.

Exclusion Criteria

I have had a tracheostomy for my ALS symptoms.

You have a history of allergic reactions to certain medications or antibodies.

I have conditions that cause muscle weakness.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

1 visit (in-person)

Treatment

Participants receive a single intravenous dose of [89Zr]Zr-DFO-AP-101 and undergo PET/CT imaging

10 days

5 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• 89Zr-DFO-AP-101

Trial Overview The study tests a new PET scan radiotracer called [89Zr]Zr-DFO-AP-101 in both healthy volunteers and ALS patients to evaluate its safety and how it spreads throughout the body. This could potentially help in early detection of ALS.

How Is the Trial Designed?

Group I: Patients with ALS Experimental Treatment 1 Intervention

Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0.

Group II: Healthy participants Experimental Treatment 1 Intervention

Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0.

Who Is Running the Clinical Trial?

Université de Sherbrooke

Lead Sponsor

Trials

317

Recruited

79,300+

Chorus Wellness Inc.

Industry Sponsor

Trials

2

Recruited

160+

AL-S Pharma AG

Collaborator

Trials

1

Recruited

10+

Eli Lilly and Company

Industry Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Citations

1. jnm.snmjournals.org jnm.snmjournals.org/content/66/1/130

Antibody-Based PET Imaging of Misfolded Superoxide ... [ 89 Zr]Zr-DFO-α-miSOD1 PET can detect misSOD1 in transgenic mice, paving the way for application in early diagnosis of ALS and therapeutic monitoring.

2. trialx.com trialx.com/clinical-trials/listings/241337/safety-and-dosimetry-of-a-new-radiotracer-to-detect-misfolded-sod1-associated-with-amyotrophic-lateral-sclerosis/

Safety and Dosimetry of a New Radiotracer to Detect Misfolded ... 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 ...

3. ctv.veeva.com ctv.veeva.com/study/safety-and-dosimetry-of-a-new-radiotracer-to-detect-misfolded-sod1-associated-with-amyotrophic-later

Safety and Dosimetry of a New Radiotracer to Detect Misfolded ... 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.

4. neurimmune.com neurimmune.com/news/al-s-pharma-announces-positive-topline-results-from-phase-2-study-of-ap-101-for-the-treatment-of-amyotrophic-lateral-sclerosis-als

AL-S Pharma announces positive topline results from ... Phase 2 of AP-101 in sporadic ALS and mutant SOD1-ALS met its primary safety and tolerability endpoint. Clinically meaningful changes in ...

5. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT05974579/

Trial | NCT05974579 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 ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05039099

NCT05039099 | A Study to Evaluate, Safety, Tolerability ... The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

7. synapse.patsnap.com synapse.patsnap.com/drug/a5ec4b09c7994f9cac0ed0bcde380b11

89Zr-DFO-AP-101 - Drug Targets, Indications, Patents A Single Center, Open Label Study to Evaluate Biodistribution, Pharmacokinetics and Safety of [89Zr]Zr-DFO-AP-101 PET (Positron Emission Tomography) in ...

8. jnm.snmjournals.org jnm.snmjournals.org/content/early/2024/12/12/jnumed.124.268343

Antibody-Based PET Imaging of Misfolded Superoxide ... [ 89 Zr]Zr-DFO-α-miSOD1 PET can detect misSOD1 in transgenic mice, paving the way for application in early diagnosis of ALS and therapeutic monitoring.