Exploratory Evaluation of [11C]MPC6827

Early Phase 1

Completed

• Conditions: Alzheimer Disease; Neurodegenerative Diseases; Amyotrophic Lateral Sclerosis
• Interventions: Drug: [11C]MPC6827

• Registration Number: NCT04575727
• Lead Sponsor: Columbia University

Brief Summary

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.

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 Timeline

• Study First Submitted: 2020-09-29
• Study First Submitted QC: 2020-10-02
• Study First Posted: 2020-10-05
• Study Start: 2021-01-08
• Primary Completion: 2024-06-21
• Study Completion: 2024-06-21
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-09
• Last Update Posted: 2025-04-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

Not provided

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Health Volunteers	[11C]MPC6827	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.
Patients with Neurodegenerative Disorders	[11C]MPC6827	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.

Primary Outcome Measures

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

Trial Locations

Locations (1)

Cuimc / Nyp; 🇺🇸 New York, New York, United States