Exploratory Evaluation of [11C]MPC6827

Exploratory Evaluation of [11C]MPC6827 Pharmacokinetics With Positron Emission Tomography (PET)

This is a phase 0 study that will enable an assessment of biodistribution and estimation of absorbed dose in humans based on data collected from five healthy volunteers, which is typically the minimum number required by the FDA for first-in-human studies to assess dosimetry of a new tracer. The evaluation of the brain imaging of thirty additional subjects in the 2nd part of the study will lead to a descriptive assessment of the targeting and pharmacokinetics of MPC6827 in the brain and between normal and diseased brain.

Study Overview

• Status: Recruiting
• Conditions: Neurodegenerative Diseases; Amyotrophic Lateral Sclerosis; Alzheimer Disease
• Intervention / Treatment: Drug: [11C]MPC6827

Detailed Description

Neurodegenerative diseases are conditions where the brain cells break down, causing mental and/or physical impairment. Alzheimer's Disease is a neurodegenerative disorder that affects millions of individuals and causes irreversible memory loss and cognitive impairment. Amyotrophic lateral sclerosis is also and incurable disorder that causes patients irreversible paralysis, which results in death due to inability to breath and suffocation. These disease have been shown to be associated with abnormalities in an important scaffolding called microtubules, a cellular structure that help support the shape of the cells. This study will explore an experimental imaging test to see if it can be used to help doctors identify early microtubule changes. The test involves the injection of a radioactive compound that has been shown to go to cells and bind to microtubules. Special cameras called Positron Emission Tomography (PET)/CT cameras will be used to allow doctors to view where the radioactive compound goes in the body, as this is the first time this radiolabeled agent is being used in humans. In this study, doctors will give the new compound to up to five healthy volunteers to see where it goes in the body of people who presumable have normal microtubule function. Then doctors will give [11C]MPC6827 to up to 30 additional subjects (healthy controls and patients with Alzheimers Disease or ALS) to focus on imaging the brain for extended times. The research-imaging drug in this study is [11C]MPC6827. The radioactive drug in this study is experimental, meaning it is not approved by the FDA and can only be used in research studies.

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Rodolfo Arevalo, BA; Phone Number: 212-342-1683; Email: ra2874@cumc.columbia.edu

Study Locations

• United States
  • New York
    • New York, New York, United States, 10032
      • Recruiting
      • Cuimc / Nyp
      • Contact: Rodolfo Arevalo, BA; Email: ra2874@cumc.columbia.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria (Healthy Volunteers)

1. All volunteers must be 18 years of age or older, able to read, understand and voluntarily sign and informed consent document.
2. Volunteers must have no current medical history of brain disease
3. Negative pregnancy test if female of childbearing potential.
4. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of study participation.

Inclusion Criteria (Alzheimer's Disease or ALS)

1. All volunteers must be 18 years of age or older, able to read, understand and voluntarily sign and informed consent document.
2. Subjects must have a diagnosis of Alzheimer's Disease or ALS for which they are under a physician's care.
3. Subjects must have a negative pregnancy test if female of childbearing potential
4. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of the study participation.

Exclusion Criteria:

1. Participants with evidence of brain disease other than ALS or Alzheimer's Disease at the time of enrolment up to agent administration are to be excluded from the study.
2. Concomitant medication use that, in the judgement of the investigator, would make the participant inappropriate for enrolment.
3. Severe concurrent disease, infection, or medical co-morbidity that, in the judgement of the investigator, would make the participant inappropriate for enrolment.
4. Participants who are receiving other investigational radiation drugs.
5. Women who are pregnant or breast feeding.
6. Subjects who are unable to tolerate PET/CT imaging

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Health Volunteers; In the first stage, five healthy human subjects will receive a microdose (10 µg) of [11C]MPC6827, immediately followed by whole body PET/CT to determine dosimetry and perform an initial safety evaluation of the radiotracer. A dose of 20 mCi [11C]MPC6827 will be administered and serial whole body PET scans will be acquired up to 2 hours post injection.	Drug: [11C]MPC6827; Subjects will receive a microdose ( 10 µg) of [11C]MPC6827 (dose of 20 mCi); Other Names: [11C]MPC
Experimental: Patients with Neurodegenerative Disorders; Up to 30 patients with neurodegenerative disorders will receive a microdose (10 µg) of [11C]MPC6827 and be imaged dynamically for up to 90 minutes using PET/CT for research purposes.	Drug: [11C]MPC6827; Subjects will receive a microdose ( 10 µg) of [11C]MPC6827 (dose of 20 mCi); Other Names: [11C]MPC

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biodistribution of [11C]MPC6827	Total body residence time and visual examination of whole body PET/CT images will be used to determine biodistribution of [11C]MPC6827.	up to 48 hours from injection
Estimation of clearance of [11C]MPC6827	Serial venous blood draws (about 5 ml per sample) will be taken for estimation of clearance of [11C]MPC6827.	Up to 48 hours from injection

Collaborators and Investigators

• Sponsor: Columbia University
• Investigators: Principal Investigator: Akiva Mintz, MD, Columbia University

Study record dates

Study Major Dates

• Study Start (Actual): January 8, 2021
• Primary Completion (Estimated): September 1, 2024
• Study Completion (Estimated): September 1, 2025

Study Registration Dates

• First Submitted: September 29, 2020
• First Submitted That Met QC Criteria: October 2, 2020
• First Posted (Actual): October 5, 2020

Study Record Updates

• Last Update Posted (Actual): October 26, 2023
• Last Update Submitted That Met QC Criteria: October 24, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: [11C]MPC6827
• Additional Relevant MeSH Terms: Mental Disorders; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Neuromuscular Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Dementia; Tauopathies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Alzheimer Disease; Neurodegenerative Diseases
• Other Study ID Numbers: AAAS4926

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Final data analysis and conclusion will be submitted to scientific journals and made available for publication.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No