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ALSPhase 1/2NCT05602142

Study of [11C]CPPC as a Clinical PET Radioligand Biomarker of Microglial Activation in ALS

A Phase 1/2 study testing [11C]CPPC PET ligand in people with ALS, run by Johns Hopkins University.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

1. Establish the safety and tolerability of the 5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide ([11C]CPPC) PET radioligand in ALS patients and controls 2. Read more about this study

Who is it for?
Ages 18 years and olderHealthy volunteers can take part
Conditions the study is for
ALS
What is being tested?
[11C]CPPC PET ligand
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
No sites listed yet
Study dates
Nov 2026to Jul 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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

  • Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) [per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling] and comply with the study procedures.
  • Men and women at least 18 years old.
  • Male patients, who have not had a vasectomy and a confirmed zero sperm count, must agree for the duration of the study to:
  • use a condom during sexual intercourse with female partners who are of reproductive potential AND to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide, or oral contraceptives) OR
  • male patient must agree to abstain from sexual intercourse during the study
  • Women must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or who have undergone hysterectomy, oophorectomy or surgical sterilization). Women must not be breastfeeding.
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  • Geographic accessibility to the study center and the ability to travel to the clinic for study visits.
  • Presence of a willing and able caregiver.
  • Diagnosis of ALS based on examination by the site PI, meeting El Escorial criteria for possible, laboratory-supported probable, probable or definite ALS or be a person without a diagnosis of ALS disorder.
  • Vital capacity ≥ 50% of predicted normal for age, height and gender measured in the seated position and the ability to lie supine for a period of 1 hour.
  • Agrees to the visit schedule as outlined in the informed consent.
  • Pre-study labs within normal range, or if abnormal, deemed not clinically significant by the site investigator.

Who cannot join

  • Weakness due to causes other than ALS.
  • Receipt of any investigational drug, device or biologic within 10 days of administration of study compound.
  • Use of anti-inflammatory medications, immunosuppressants, or benzodiazepines within 7 days of administration of study compound.
  • Any concomitant medical disease or condition limiting the safety to participate including, but not limited to:
  • Coagulopathy
  • Active infection
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  • Any condition that the site PI feels may interfere with participation in the study.
  • Inability to provide informed consent as determined by the site PI.
  • Known clinical evidence of frontotemporal dementia.
  • Inadequate family or caregiver support as determined by the site PI.
  • Presence of any of the following conditions:
  • Current drug abuse or alcoholism
  • Unstable medical conditions
  • Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

JinAe Arneklev, CRNP contact

410-502-2932

jarnekl1@jhu.edu

About this study

Official title: A Phase 1/2 Study of [11C]CPPC as a Clinical PET Radioligand Biomarker of Microglial Activation in ALS

  1. Establish the safety and tolerability of the 5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide ([11C]CPPC) PET radioligand in ALS patients and controls
  2. Examine whether [11C]CPPC PET uptake is elevated in brains of ALS patients and whether there is a correlation with clinical phenotype.
  3. Correlate [11C]CPPC PET imaging with other ALS outcome measures and biofluid biomarkers
  4. Examine longitudinal changes in [11C]CPPC PET imaging during disease course.
Read the detailed description

There are a paucity of reliable serum and cerebrospinal (CSF) biomarkers and validated neuroimaging techniques to aid in amyotrophic lateral sclerosis (ALS) diagnosis, prognosis, or pharmacodynamic insight. Positron emission tomography (PET) imaging is a technique that uses radioactive molecules attached to a ligand of interest which localizes to the desired target, allowing for visualization of the three dimensional distribution of the ligand's target receptor.

One of the upstream processes that are thought to lead to motor neuron degeneration in ALS is microglial dysfunction, resulting in the initiation of neuroinflammatory cascades. Macrophage colony stimulating factor 1 receptor (CSF1R) is found on microglia predominately in the brain, with low levels of expression in neurons and other neural cells, making it a promising target for studying microglial activation.

Given CSF1's potential role in ALS disease progression, and that its receptor (CSF1R) can be directly targeted, ligands binding this receptor are an area of interest for imaging in ALS. [11C]CPPC [5-cyano-N-(4-(4-[11C]methylpiperazin-1-yl)-2-(piperidin-1-yl)phenyl)furan-2-carboxamide], is a positron-emitting, high-affinity ligand that is specific for CSF1R.

The aims of this study are as follows:

  1. Establish the safety and tolerability of the [11C]CPPC PET radioligand in ALS patients and controls
  2. Examine whether [11C]CPPC PET uptake is elevated in brains of ALS patients and whether there is a correlation with clinical phenotype.
  3. Correlate [11C]CPPC PET imaging with other ALS outcome measures and biofluid biomarkers
  4. Examine longitudinal changes in [11C]CPPC PET imaging during disease course.
Conditions
ALS
Being tested
[11C]CPPC PET ligand
Keywords
Biomarkers

What participants receive

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

  • [11C]CPPC PET ligandDrug

    Also called 5-cyano-N-(4-(4-[11C]methylpiperazin-1-yl)-2-(piperidin-1-yl)phenyl)furan-2-carboxamide

    Radioactive PET ligand to determine microglia expression of colony stimulating factor 1 receptor (CSF1R).

Study groups in NCT05602142
Study groupRoleWhat they receive
[11C]CPPCExperimentalAll participants will receive [11C]CPPC which is a radiotracer ligand that specifically binds to CSF1R.
Receives: Drug: [11C]CPPC PET ligand

What is being measured

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

  1. Safety of use of [11C]CPPC in patients with ALS as assessed by adverse events

    Safety of use of [11C]CPPC in positron emission tomography (PET) neuroimaging of patients with a diagnosis of ALS. Safety will be assessed by number of adverse events during and up to 180 days after the injection

    Measured: Up to 180 days after scan

  2. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in neurological status

    Safety of use of [11C]CPPC in positron emission tomography (PET) neuroimaging of patients with a diagnosis of ALS. Safety will be assessed by neurological physical exam to determine if there is a change in the findings from baseline.

    Measured: Baseline and 180 days after scan

  3. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in complete blood count (CBC) test

    Safety of use of [11C]CPPC in positron emission tomography (PET) neuroimaging of patients with a diagnosis of ALS. Safety will be assessed by monitoring of the complete blood count (CBC) for a change from baseline that is outside of the normal range.

    Measured: Baseline and 180 days after scan

  4. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in complete metabolic panel (CMP) test

    Safety of use of [11C]CPPC in positron emission tomography (PET) neuroimaging of patients with a diagnosis of ALS. Safety will be assessed by a change in the CMP from baseline that is outside of the normal range.

    Measured: Baseline and 180 days after scan

Secondary outcomes (1)

  1. Sensitivity of use of [11C]CPPC as assessed by a radiologist

    Sensitivity of use of [11C]CPPC in PET neuroimaging to detect ALS will be determined by comparing the PET images from patients with ALS with those from healthy controls.

    Measured: Up to 180 days after scan

Timeline

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

  1. First posted1 Nov 2022
  2. Study startNov 2026 (estimated)
  3. Primary completionJan 2027 (estimated)
  4. Study completionJul 2027 (estimated)

Resources

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 1/2
Primary purpose
Diagnosis
Allocation
Not applicable
Intervention model
Single group
Model description
Disease group and healthy controls will receive the same drug
Masking
None (open label)
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05602142
Sponsor's protocol ID
IRB00343494
Other IDs
W81XWH-22-1-0130
Status last verified
Aug 2026
First submitted
21 Oct 2022
Last updated
25 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing [11C]CPPC PET ligand in people with ALS, run by Johns Hopkins University.

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 12 inclusion and 14 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: opening soon, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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

Contact JinAe Arneklev, CRNP (410-502-2932, jarnekl1@jhu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety of use of [11C]CPPC in patients with ALS as assessed by adverse events (time frame: Up to 180 days after scan). The study lists 4 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 NCT05602142.

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 25 Aug 2026.

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