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Amyotrophic Lateral SclerosisPhase 1NCT04749433

Study of [11C]CPPC to Assess the Safety and Tolerability in Patients With ALS

A Phase 1 study testing [11C]CPPC Injection in people with Amyotrophic Lateral Sclerosis, run by Johns Hopkins University at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The goal of this study is to evaluate the safety of using the [5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide] ([11C]CPPC) radiotracer in positron emission tomography (PET) imaging of people with amyotrophic lateral sclerosis (ALS). 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?
[11C]CPPC Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
4 participants
Where
1 siteUnited States
Study dates
1 Sep 2021to 30 Mar 2023
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

  • 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.
  • 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).
  • 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
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  • 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 30 days of administration of study compound.
  • Use of anti-inflammatory medications, immunosuppressants, or benzodiazepines.
  • 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

1 site in 1 country.

United States1 site

  • Johns Hopkins Outpatient Center

    Baltimore, Maryland, 21287

Other trials in United States

About this study

Official title: A Phase 1 Study to Assess the Safety and Tolerability of PET Imaging With [11C]CPPC [5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide] Radioligand in Patients With Amyotrophic Lateral Sclerosis

The goal of this study is to evaluate the safety of using the [5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide] ([11C]CPPC) radiotracer in positron emission tomography (PET) imaging of people with amyotrophic lateral sclerosis (ALS). The investigators are also interested to see whether use of this radiotracer reveals imaging differences between patients with ALS and healthy patients.

Read the detailed description

This is a Phase 1 study of safety and tolerability of an investigational radiotracer drug called [5-cyano-N-(4-(4-[11C]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide] ([11C]CPPC). A radiotracer is a substance that chemically marks certain structures in the body. In this case, [11C]CPPC highlights structures expressing colony stimulating factor receptor (CSF1R), a receptor that is expressed on microglial cells. A safety and tolerability study is looking to see if there are any unanticipated, possibly harmful, effects of the use of the radiotracer in humans. However, ultimately, the investigators would like to know if this drug can be used to make better images of the brain for patients with amyotrophic lateral sclerosis (ALS), which could help doctors better understand the disease and help take care of patients with ALS. This study will use a radiotracer to look for a chemical receptor which ALS patients have more of in the brain. After receiving the radiotracer, participants' brains will be scanned with a positron emission tomography (PET) imaging machine.

Being tested
[11C]CPPC Injection
Keywords
Positron Emission Tomography
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.

  • [11C]CPPC InjectionDrug

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

    A single dose of [11C]CPPC (370 megabecquerel (MBq) (X±1 mCi)) intravenously and subsequent positron emission tomography (PET) scan.

Study groups in NCT04749433
Study groupRoleWhat they receive
Participants with diagnosis of ALSExperimentalParticipants with a diagnosis of ALS will receive a single dose of [11C]CPPC (370 megabecquerel (MBq) (X±1 mCi)) intravenously and subsequent positron emission tomography (PET) scan.
Receives: Drug: [11C]CPPC Injection
Healthy Participants without a diagnosis of ALSExperimentalHealthy participants (without a diagnosis of ALS) will receive a single dose of [11C]CPPC (370 megabecquerel (MBq) (X±1 mCi)) intravenously and subsequent positron emission tomography (PET) scan.
Receives: Drug: [11C]CPPC Injection

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 10 days after the injection.

    Measured: Up to 10 days follow up 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 10 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 10 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 10 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: 1 day

Timeline

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

  1. First posted11 Feb 2021
  2. Study start1 Sep 2021
  3. Primary completion30 Mar 2023
  4. Study completion30 Mar 2023

Why it stopped: study stopped to enhance protocol and resume under different sponsor

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
Primary purpose
Diagnosis
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
There will be two groups of participants in this study, healthy control participants and participants with a diagnosis of ALS. All patients will receive the same radiotracer and scan.
Masking
None (open label)
Enrolment
4 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04749433
Sponsor's protocol ID
IRB00263068
Status last verified
Oct 2024
First submitted
29 Jan 2021
Last updated
8 Oct 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Horti AG, Naik R, Foss CA, Minn I, Misheneva V, Du Y, Wang Y, Mathews WB, Wu Y, Hall A, LaCourse C, Ahn HH, Nam H, Lesniak WG, Valentine H, Pletnikova O, Troncoso JC, Smith MD, Calabresi PA, Savonenko AV, Dannals RF, Pletnikov MV, Pomper MG. PET imaging of microglia by targeting macrophage colony-stimulating factor 1 receptor (CSF1R). Proc Natl Acad Sci U S A. 2019 Jan 29;116(5):1686-1691. doi: 10.1073/pnas.1812155116. Epub 2019 Jan 11.

    PubMed 30635412

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing [11C]CPPC Injection in people with Amyotrophic Lateral Sclerosis, run by Johns Hopkins University at 1 site in United States.

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 9 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: stopped early, as of the sponsor's last update on 8 Oct 2024. 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 United States. 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 was stopped early (reason given: study stopped to enhance protocol and resume under different sponsor). Results are not available in the source record.

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?

Safety of use of [11C]CPPC in patients with ALS as assessed by adverse events (time frame: Up to 10 days follow up 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 NCT04749433, 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 8 Oct 2024.

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Clinical Trial NCT04749433 | AzTrials