PET Study of PIPE-791 in Healthy Volunteers and Volunteers With PrMS and IPF

A Phase 1b, Open-label Study of PIPE-791 to Determine Brain and Lung Lysophosphatidic Acid Receptor 1 (LPA1) Occupancy, by [18F] PIPE-497 PET Imaging in Healthy Volunteers, Volunteers With Progressive Multiple Sclerosis, and Volunteers With Idiopathic Pulmonary Fibrosis

This is a study of PIPE-791, an investigational study drug to treat progressive multiple sclerosis (MS) and idiopathic pulmonary fibrosis (IPF). The purpose of this study is to find out how much of the study drug gets into the brain and lung, and what the side effects and blood levels of the study drug are in healthy volunteers and patients.

Participants will:

• Take a single dose of the study drug
• Give many samples of blood and urine
• Have multiple PET scans

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis; Healthy; Multiple Sclerosis, MS; Idiopathic Pulmonary Fibrosis; Healthy Volunteers; Multiple Sclerosis, Secondary Progressive; Idiopathic Pulmonary Fibrosis (IPF); Multiple Sclerosis, Primary Progressive; Multiple Sclerosis, Progressive
• Intervention / Treatment: Drug: PIPE-791

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom
    • Hammersmith Medicines Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Main Inclusion Criteria

• All parts: male volunteers or female volunteers of non-childbearing potential; agree to follow the contraception requirements of the trial, and able to give fully informed written consent.
• Part A: normotensive volunteers, deemed healthy on the basis of a clinical history, medical examinations, ECG, vital signs, and laboratory tests of blood and urine.
• Part B: volunteers with a diagnosis of PPMS or SPMS by a neurologist, according to the 2017 Revised McDonald Criteria.
• Part C: volunteers with a diagnosis of IPF by a pulmonologist, according to the 2018 American Thoracic Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Thoracic Society (ALAT) Clinical Practice Guideline, within 7 years before screening.

Main Exclusion Criteria

• Positive tests for hepatitis B and C, human immunodeficiency virus (HIV)
• Severe adverse reaction to any drug; sensitivity to trial medication
• Drug or alcohol abuse
• Smoking or use of tobacco or nicotine-containing products from 7 days before screening, until the final visit
• Use of a prescription medicine (except hormone replacement therapy [HRT] in women, and medications for participants with PrMS and IPF at the discretion of the investigator), or any substance known to interact with cytochrome p450 (CYP)3A enzymes (including St. John's wort and foodstuffs such as grapefruit juice) during the 28 days before the first dose of PIPE-791
• Use of any other over-the-counter medicine, with the exception of acetaminophen (paracetamol) during the 7 days before the first dose of PIPE-791, or received vaccine against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), during the 7 days before screening
• Participation in other clinical trials of unlicensed medicines, or loss of more than 400 mL blood, within the 3 months before the baseline PET scan
• Vital signs or ECGs outside the acceptable range at screening
• Clinically relevant abnormal findings at the screening assessments, including C-SSRS and MRI (Parts A [brain PET imaging arm] and B only)
• Acute or chronic illness (except PrMS and IPF in Parts B and C, respectively)
• Clinically relevant abnormal medical history or concurrent medical condition
• Possibility that volunteer will not cooperate
• Contraindications to MRI (Parts A [brain PET imaging arm] and B only), computed tomography (CT), PET, or arterial cannulation procedures
• Significant exposure to research-related radiation or other radiation exposure (exceeding 10 mSv when added to exposure from this study) within the previous 12 months

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Healthy volunteers, brain PET imaging	Drug: PIPE-791; Subjects will receive a single oral dose of PIPE-791.
Experimental: Healthy volunteers, lung PET imaging	Drug: PIPE-791; Subjects will receive a single oral dose of PIPE-791.
Experimental: Volunteers with PrMS	Drug: PIPE-791; Subjects will receive a single oral dose of PIPE-791.
Experimental: Volunteers with IPF	Drug: PIPE-791; Subjects will receive a single oral dose of PIPE-791.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
LPA1 occupancy as determined by regional total volume of distribution (VT) at each brain scan.	Baseline to up to 28 days post-dose
LPA1 occupancy as determined by regional total volume of distribution (VT) at each lung scan.	Baseline to up to 28 days post-dose

Secondary Outcome Measures

Outcome Measure	Time Frame
The relationship between PIPE-791 plasma concentration and brain and lung LPA1 occupancy.	Baseline to up to 28 days post-dose
The PIPE-791 EC50 (i.e., the plasma concentration of PIPE-791 associated with 50% occupancy of LPA1) in the brain and lungs.	Baseline to up to 28 days post-dose
Pharmacokinetics (PK): blood concentration levels of PIPE-791	Baseline to up to 28 days post-dose
Safety and tolerability: Treatment-Emergent Adverse Events (TEAE)	Baseline to up to 32 days post-dose

Collaborators and Investigators

• Sponsor: Contineum Therapeutics
• Investigators: Study Director: Stephen Huhn, MD, Contineum Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2024
• Primary Completion (Actual): June 4, 2025
• Study Completion (Actual): June 12, 2025

Study Registration Dates

• First Submitted: November 6, 2024
• First Submitted That Met QC Criteria: November 8, 2024
• First Posted (Actual): November 12, 2024

Study Record Updates

• Last Update Posted (Actual): August 6, 2025
• Last Update Submitted That Met QC Criteria: August 5, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: MS; secondary progressive MS; multiple sclerosis; nervous system diseases; idiopathic pulmonary fibrosis; PMS; remyelination; IPF; SPMS; autoimmune diseases; neuroinflammation; sclerosis; PPMS; secondary progressive multiple sclerosis; primary progressive multiple sclerosis; PIPE-791; pathologic processes; PrMS; primary progressive MS; LPA1; demyelinating diseases; demyelinating autoimmune diseases, CNS; autoimmune diseases of the nervous system; immune diseases
• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Neoplastic Processes; Lung Diseases, Interstitial; Multiple Sclerosis; Sclerosis; Neoplasm Metastasis; Pulmonary Fibrosis; Idiopathic Pulmonary Fibrosis; Fibrosis; Multiple Sclerosis, Chronic Progressive
• Other Study ID Numbers: CTX-791-102

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No