NCT05602142

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

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

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Johns Hopkins University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Accepted

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. 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.

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

Interventions

Type	Name	Description
DRUG	[11C]CPPC PET ligand	Radioactive PET ligand to determine microglia expression of colony stimulating factor 1 receptor (CSF1R).

Timeline

• Start date: 2026-08-01
• Primary completion: 2027-01-01
• Completion: 2027-07-01
• First posted: 2022-11-01
• Last updated: 2026-04-13

Regulatory

• FDA-regulated drug study