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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04749433
Recruitment Status : Recruiting
First Posted : February 11, 2021
Last Update Posted : September 20, 2021
Sponsor:
Collaborator:
Precision Molecular
Information provided by (Responsible Party):
Johns Hopkins University

Brief 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). The investigators are also interested to see whether use of this radiotracer reveals imaging differences between patients with ALS and healthy patients.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: [11C]CPPC Injection Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention 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)
Primary Purpose: Diagnostic
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
Actual Study Start Date : September 1, 2021
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : June 30, 2022


Arm Intervention/treatment
Experimental: Participants with diagnosis of ALS
Participants 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.
Drug: [11C]CPPC Injection
A single dose of [11C]CPPC (370 megabecquerel (MBq) (X±1 mCi)) intravenously and subsequent positron emission tomography (PET) scan.
Other Name: [5-cyano-N-(4-(4-[11C]methylpiperazin-1-yl)-2-(piperidin-1-yl)phenyl)furan-2-carboxamide]

Experimental: Healthy Participants without a diagnosis of ALS
Healthy 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.
Drug: [11C]CPPC Injection
A single dose of [11C]CPPC (370 megabecquerel (MBq) (X±1 mCi)) intravenously and subsequent positron emission tomography (PET) scan.
Other Name: [5-cyano-N-(4-(4-[11C]methylpiperazin-1-yl)-2-(piperidin-1-yl)phenyl)furan-2-carboxamide]




Primary Outcome Measures :
  1. 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 ]
    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.

  2. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in neurological status [ Time Frame: Baseline and 10 days after scan ]
    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.

  3. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in complete blood count (CBC) test [ Time Frame: Baseline and 10 days after scan ]
    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.

  4. Safety of use of [11C]CPPC in patients with ALS as assessed by a change in complete metabolic panel (CMP) test [ Time Frame: Baseline and 10 days after scan ]
    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.


Secondary Outcome Measures :
  1. Sensitivity of use of [11C]CPPC as assessed by a radiologist [ Time Frame: 1 day ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  1. 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.
  2. Men and women at least 18 years old.
  3. 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).
  4. Geographic accessibility to the study center and the ability to travel to the clinic for study visits.
  5. Presence of a willing and able caregiver.
  6. 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
  7. 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.
  8. Agrees to the visit schedule as outlined in the informed consent.
  9. Pre-study labs within normal range, or if abnormal, deemed not clinically significant by the site investigator.

Exclusion Criteria:

  1. Weakness due to causes other than ALS.
  2. Receipt of any investigational drug, device or biologic within 30 days of administration of study compound.
  3. Use of anti-inflammatory medications, immunosuppressants, or benzodiazepines.
  4. Any concomitant medical disease or condition limiting the safety to participate including, but not limited to:

    1. Coagulopathy
    2. Active infection
  5. Any condition that the site PI feels may interfere with participation in the study
  6. Inability to provide informed consent as determined by the site PI.
  7. Known clinical evidence of frontotemporal dementia
  8. Inadequate family or caregiver support as determined by the site PI.
  9. Presence of any of the following conditions:

    1. Current drug abuse or alcoholism
    2. Unstable medical conditions
    3. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04749433


Contacts
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Contact: Caroline F Plott, MS 4106149874 cplott1@jhmi.edu

Locations
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United States, Maryland
Johns Hopkins Outpatient Center Recruiting
Baltimore, Maryland, United States, 21287
Contact: Nicholas J Maragakis, MD    410-614-9874      
Sponsors and Collaborators
Johns Hopkins University
Precision Molecular
Investigators
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Principal Investigator: Nicholas J Maragakis, MD Johns Hopkins University
Publications:
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Responsible Party: Johns Hopkins University
ClinicalTrials.gov Identifier: NCT04749433    
Other Study ID Numbers: IRB00263068
First Posted: February 11, 2021    Key Record Dates
Last Update Posted: September 20, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Johns Hopkins University:
Positron Emission Tomography
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases