Amyotrophic Lateral SclerosisPhase 1NCT05974579

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

A Phase 1 study testing 89Zr-DFO-AP-101 in people with Amyotrophic Lateral Sclerosis, run by Université de Sherbrooke at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and olderHealthy volunteers can take part
Conditions the study is for: Amyotrophic Lateral Sclerosis
What is being tested?: 89Zr-DFO-AP-101
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 8 participants
Where: 1 siteCanada
Study dates: 23 Nov 2023to 31 Mar 2026
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

Aged of:
For healthy participants: Male or female subjects aged 50 years or older
For ALS patients: Male or female subjects aged 18 years and older
Able to remain in a lying position for up to 45 minutes without respiratory support.
A) For ALS patients, confirmed diagnostic of definitive ALS according to El-Escorial criteria14 B) for healthy participants: no neurologic condition (confirmed by physical exam)
Have venous access sufficient to allow for blood sampling
Are reliable and willing to make themselves available for the duration of the study and are willing to follow CRCHUS-specific study procedures.

Who cannot join

Are currently enrolled or were enrolled in the last 12 weeks in any other clinical trial involving a study drug or off label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study.
Female participants who are pregnant or breast feeding; or women of childbearing potential (<50 years old) and men who are sexually active who are not willing to use an accepted effective contraceptive method.
Plan to have surgery or other invasive procedure during the course of the study (up to 14 days post-injection)
Have a progressive medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric or neurological disease, convulsions, or any clinically significant laboratory abnormality at screening and at first visit (D0) that, in the judgment of the medical doctor, indicate a medical problem that would preclude study participation.
Have one of these conditions (for both patient groups):
hepatic disorder such as hepatic encephalopathy, hepatic laboratory abnormalities (ALT or AST ≥3 × ULN or total bilirubin ≥2 × ULN) and hematology abnormalities at screening.

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severe chronic kidney disease (eg, an estimated glomerular filtration rate [eGFR] <30 mL/min/1.73m or requires chronic dialysis) at screening.
Have severe active psychiatric illness.
Have a diagnosis of another neurodegenerative disease (e.g. Parkinson disease, Alzheimer's disease, etc).
Have a significant infection or known inflammatory process on screening or at Day 0.
Alcohol or drug abuse based on patient auto-report
Have a history of relevant atopy or drug hypersensitivity or allergy to antibodies;
Have an abnormal blood pressure (supine) defined as a diastolic blood pressure >90 or <45 mmHg and/or a systolic blood pressure >160 or <90 mmHg. Re-testing may occur once during the screening visit within 2 hours of the initial abnormal blood pressure measurement at the discretion of the investigator.
For ALS patients:
Have undergone a tracheostomy for ALS symptoms.
Are on nasal intermittent positive pressure ventilation (NIPPV) >4h during the day, while awake for the treatment of ALS related symptoms.
Have other causes of neuromuscular weakness.
Have received treatment with biologic agents (such as monoclonal antibodies, including marketed drugs and AP-101) within 3 months or 5 half-lives (whichever is longer) prior to study drug injection.
Have received any blood or blood products within the 3 months prior to screening.
Cannot communicate reliably with the investigator.
Are unwilling or unable to give written informed consent.
In the opinion of the medical doctor or his/her delegate, are unsuitable for inclusion in the study.

Trial locations and contacts

1 site in 1 country.

Canada1 site

CIUSSS de l'Estrie-CHUS Hospital
Sherbrooke, Quebec, J1H 5N4

Other trials in Canada

About this study

Official title: 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

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.

Read the 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
Being tested: 89Zr-DFO-AP-101
Keywords: PET imaging, CT imaging, AP-101, 89-Zirconium, DFO
Medical subject headings: Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

89Zr-DFO-AP-101Drug
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.

Study groups in NCT05974579
Study group	Role	What they receive
Healthy participants	Experimental	Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0. Receives: Drug: 89Zr-DFO-AP-101
Patients with ALS	Experimental	Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0. Receives: Drug: 89Zr-DFO-AP-101

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of adverse events (AEs) and serious adverse events (SAEs)
Number of adverse events (AEs) and serious adverse events following administration of [89Zr]Zr-DFO-AP-101 that are new or worsened (compared to baseline/pre-dose)
Measured: day 0 (post-injection) to day 14 (end of study)
Biodistribution of [89Zr]Zr-DFO-AP-101
Assessed by whole-body PET imaging
Measured: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Dosimetry of [89Zr]Zr-DFO-AP-101 in human
Organ activity concentration (in liver, kidneys, blood, spleen, ...) measured by drawing region of interests on the PET images.
Measured: Pre-Dose and at 2 hours, 1, 3, 7, 10 days post-dose

Secondary outcomes (4)

Cmax
Maximal concentration of [89Zr]Zr-DFO-AP-101 in plasma over time after injection
Measured: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Area under the curve (AUC)
AUC of [89Zr]Zr-DFO-AP-101 in plasma over time after injection
Measured: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
residence time
Time (1/2) of residence of [89Zr]Zr-DFO-AP-101 in plasma
Measured: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Excretion
Concentration of [89Zr]Zr-DFO-AP-101 urine over time
Measured: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

23 Nov 202331 Mar 2026

First posted3 Aug 2023
Study start23 Nov 2023
Primary completion30 Dec 2024
Study completion31 Mar 2026

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
More trials run by Université de SherbrookeThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Diagnosis
Allocation: Non-randomised
Intervention model: Parallel groups
Model description: Two groups will be enrolled: 8 healthy subjects and 4 patients with ALS This is non-randomized, all participants will receive the same dose of radiotracer
Masking: None (open label)
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05974579
Sponsor's protocol ID: 2024-5148
Other IDs: 2024-5148
Status last verified: May 2026
First submitted: 11 Jul 2023
Last updated: 1 Jun 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Croteau E, Rousseau E, Tremblay S, Rousseau JF, Espinosa-Betancourt E, Lavallee E, Dubreuil S, Ait-Mohand S, Lareau-Trudel E, Gosselin S, Carrier V, Cote-Bigras S, Allard C, Maier M, Salzmann M, Tetu A, Houde MP, Turcotte E, Guerin B. A phase I study to evaluate the dosimetry and safety of [89Zr]Zr-DFO-AP-101, a new antibody-based radiopharmaceutical to detect misfolded SOD1 in amyotrophic lateral sclerosis. Eur J Nucl Med Mol Imaging. 2026 Jun;53(7):4639-4651. doi: 10.1007/s00259-026-07853-y. Epub 2026 Mar 24.
PubMed 41872337

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
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All Amyotrophic Lateral Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT05974579
Acronym / sponsor ID: 2024-5148
Conditions: Amyotrophic Lateral Sclerosis
Keywords: PET imaging, CT imaging, AP-101, 89-Zirconium, DFO
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 8 participants (actual)
Lead sponsor: Université de SherbrookeUniversity, hospital or other
Collaborators: Eli Lilly and Company, Chorus Wellness Inc.
Study start: 23 Nov 2023 (actual)
Primary completion: 30 Dec 2024 (actual)
Study completion: 31 Mar 2026 (actual)
First posted: 3 Aug 2023 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

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.

Study design

Primary purpose: Diagnosis
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2
Model description: Two groups will be enrolled: 8 healthy subjects and 4 patients with ALS This is non-randomized, all participants will receive the same dose of radiotracer

Arm	Type	Receives
Healthy participants	Experimental	Drug: 89Zr-DFO-AP-101Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0.
Patients with ALS	Experimental	Drug: 89Zr-DFO-AP-101Will receive 40MBq of 89Zr-DFO-AP-101, once, at Day 0.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Aged of:
Able to remain in a lying position for up to 45 minutes without respiratory support.
A) For ALS patients, confirmed diagnostic of definitive ALS according to El-Escorial criteria14 B) for healthy participants: no neurologic condition (confirmed by physical exam)
Have venous access sufficient to allow for blood sampling
Are reliable and willing to make themselves available for the duration of the study and are willing to follow CRCHUS-specific study procedures.

Key exclusion criteria (22 listed)

Are currently enrolled or were enrolled in the last 12 weeks in any other clinical trial involving a study drug or off label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study.
Female participants who are pregnant or breast feeding; or women of childbearing potential (<50 years old) and men who are sexually active who are not willing to use an accepted effective contraceptive method.
Plan to have surgery or other invasive procedure during the course of the study (up to 14 days post-injection)
Have a progressive medical illness including, but not limited to, any cardiovascular, hepatic, respiratory, hematological, endocrine, psychiatric or neurological disease, convulsions, or any clinically significant laboratory abnormality at screening and at first visit (D0) that, in the judgment of the medical doctor, indicate a medical problem that would preclude study participation.
Have one of these conditions (for both patient groups):

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
89Zr-DFO-AP-101At 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.	Drug	Healthy participants, Patients with ALS

Primary outcome

Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: day 0 (post-injection) to day 14 (end of study)
Number of adverse events (AEs) and serious adverse events following administration of [89Zr]Zr-DFO-AP-101 that are new or worsened (compared to baseline/pre-dose)
Biodistribution of [89Zr]Zr-DFO-AP-101
Time frame: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Assessed by whole-body PET imaging
Dosimetry of [89Zr]Zr-DFO-AP-101 in human
Time frame: Pre-Dose and at 2 hours, 1, 3, 7, 10 days post-dose
Organ activity concentration (in liver, kidneys, blood, spleen, ...) measured by drawing region of interests on the PET images.

4 secondary outcomes

Cmax
Time frame: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Area under the curve (AUC)
Time frame: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
residence time
Time frame: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose
Excretion
Time frame: Pre-dose and at 2 hours, 1, 3, 7, 10 days post-dose

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2026.
Derived from this study: PubMed 41872337Croteau E, Rousseau E, Tremblay S, Rousseau JF, Espinosa-Betancourt E, Lavallee E, Dubreuil S, Ait-Mohand S, Lareau-Trudel E, Gosselin S, Carrier V, Cote-Bigras S, Allard C, Maier M, Salzmann M, Tetu A, Houde MP, Turcotte E, Guerin B. A phase I study to evaluate the dosimetry and safety of [89Zr]Zr-DFO-AP-101, a new antibody-based radiopharmaceutical to detect misfolded SOD1 in amyotrophic lateral sclerosis. Eur J Nucl Med Mol Imaging. 2026 Jun;53(7):4639-4651. doi: 10.1007/s00259-026-07853-y. Epub 2026 Mar 24.
Individual participant dataSharing planned: No

Related trials for Amyotrophic Lateral Sclerosis

RecruitingPhase 1/2
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Eric E Turcotte, MD study director

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

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing 89Zr-DFO-AP-101 in people with Amyotrophic Lateral Sclerosis, run by Université de Sherbrooke at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 1 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (AEs) and serious adverse events (SAEs) (time frame: day 0 (post-injection) to day 14 (end of study)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05974579, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2026.