Study to Evaluate the Safety and Efficacy of PIPE-307 in Subjects With Relapsing-Remitting Multiple Sclerosis

Phase 2

Active, not recruiting

• Conditions: Relapsing Remitting Multiple Sclerosis
• Interventions: Drug: PIPE-307 Dose A; Drug: PIPE-307 Dose B; Drug: Placebo

• Registration Number: NCT06083753
• Lead Sponsor: Contineum Therapeutics

Brief Summary

This is a randomized, double-blind study of PIPE-307 or placebo in subjects with relapsing-remitting multiple sclerosis. Subjects will be randomized into 1 of 3 separate cohorts (1:1:1 randomization ratio, PIPE-307 Dose A:PIPE-307 Dose B: Placebo) for a total duration of approximately 30 weeks.

Detailed Description

This is a randomized, double-blind study of PIPE-307 or placebo given to 168 subjects randomized into one of 3 separate cohorts. They will be randomized 1:1:1 (PIPE-307 Dose A:Pipe 307 Dose B: Placebo). There will be a 28-day screening period followed by a 26-week treatment period. Safety will be assessed by periodic measurements of vital signs (VS), physical (PE) and neurological examinations, electrocardiograms (ECG), blood laboratory analyses and occurrence of adverse events (AE).

Study Timeline

• Study First Submitted: 2023-10-09
• Study First Submitted QC: 2023-10-09
• Study First Posted: 2023-10-16
• Study Start: 2023-11-06
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-06
• Last Update Posted: 2025-01-07
• Primary Completion: 2025-08
• Study Completion: 2025-09

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 168

Inclusion Criteria

• Subject is fluent in English.
• Male or female 18 to 50 years of age, inclusive, at the first Screening visit.
• A diagnosis of relapsing-remitting multiple sclerosis (RRMS) according to the 2017 Revised McDonald Criteria.
• Expanded Disability Status Scale (EDSS) and retinal nerve fiber layer within protocol requirements.
• Stable immunomodulatory treatment on no more than a single DMT for RRMS over the 6 months prior to Screening, as determined by the PI.
• Male or female subjects with reproductive potential agree to comply with a highly effective contraceptive method as per protocol through 1 month after last study drug administration as per protocol.
• General good medical health with no clinically significant or relevant abnormalities except those attributed to the underlying multiple sclerosis (MS), including medical history, physical exam, vital signs, ECG and laboratory evaluations, as assessed by the Investigator.

If enrolled in the visual evoked potential (VEP) sub-study, an additional inclusion criterion includes:

• Screening VEP P100 latency greater than the upper limit of normal (as defined in the protocol) in at least one eye, OR a protocol-defined difference in VEP P100 latency between eyes.

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Exclusion Criteria

• Diagnosis or history of symptoms of optic neuritis within 9 months prior to Screening in either eye.
• Diagnosis of MS more than 10 years prior to Screening.
• History of severe myopia, ophthalmologic or retinal disorder that would interfere with measurements of low contrast letter acuity (LCLA) or exam by optical coherence tomography (OCT), as determined by Investigator.
• Concurrent use of dalfampridine or other 4-aminopyridine or diamino-4-aminopyridine drugs.
• Clinical MS relapse or MS related treatment with corticosteroids within 6 months prior to or during Screening.
• History of treatment with bone marrow transplantation, mitoxantrone, cyclophosphamide, atacicept, or irradiation.
• Use of any daily or routine anticholinergic medications within 30 days of Screening or concurrent during the study.
• The presence of gadolinium enhancing lesions by MRI.
• Use of any drugs known to strongly or moderately induce or inhibit Cytochrome P450 3A4 (CYP3A4) enzyme activity within 30 days prior to Screening or concurrent during the study.
• Use of an investigational product, vaccine or intervention other than a non-interventional registry study within the greater of 30 days or 5 half-lives (if known) prior to Screening or expected during the study.
• History of malignancy under current active treatment or considered at substantial risk for progression or recurrence during the study interval, and/or significant cardiac disorder or dysrhythmia, as determined by the Investigator.
• History of a suicide attempt or suicidal behavior or considered at risk for suicide as judged by the PI using the Columbia-Suicide Severity Rating Scale (C-SSRS) as Screening.

If enrolled in the visual evoked potential (VEP) sub-study, an additional exclusion criterion includes:

• History of an ophthalmologic or retinal disorder that would interfere with measurements of VEP, as determined by the Investigator.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
PIPE-307 Dose A	PIPE-307 Dose A	-
PIPE-307 Dose B	PIPE-307 Dose B	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Change in binocular 2.5% low contrast letter acuity (LCLA)	From baseline to week 26 (end-of-study)
Treatment-emergent adverse events (TEAE)	From baseline to week 26 (end of treatment period)	Number of participants with TEAEs

Secondary Outcome Measures

Name	Time	Method
Change in monocular 2.5% LCLA	From baseline to week 26
Percentage of subjects with >/=5-letter gain in binocular 2.5% LCLA	From baseline to week 26
Number of subjects with at least a 15% change in disability with the Nine-Hole Peg Test (9HPT)	From baseline to week 26
Number of subjects with at least a 15% change in disability with the Timed 25-Foot Walk Test (T25WT)	From baseline to week 26
Number of subjects with at least a 15% change in disability with the Symbol Digital Modality Test (SDMT)	From baseline to week 26
Change in magnetic resonance imaging (MRI) measures of myelination and MS disease activity	From baseline to week 26
Change in serum neurofilament light chain (NfL)	From baseline to week 26
Pharmacokinetics: Change in blood concentration levels of PIPE-307	From baseline to week 30

Trial Locations

Locations (21)

Xenosciences; 🇺🇸 Phoenix, Arizona, United States

Arizona Neuroscience Research, LLC; 🇺🇸 Phoenix, Arizona, United States

Alta Bates Summit Medical Center; 🇺🇸 Berkeley, California, United States

Colorado Springs Neurological Associates; 🇺🇸 Colorado Springs, Colorado, United States

MS and Neuromuscular Center of Excellence; 🇺🇸 Clearwater, Florida, United States

Aqualane Clinical Research; 🇺🇸 Naples, Florida, United States

Shepherd Center; 🇺🇸 Atlanta, Georgia, United States

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States

Neurology Center of New England P.C.; 🇺🇸 Foxboro, Massachusetts, United States

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

University of New Mexico/Health Science Center/MIND Imaging Center/MS Specialty Clinic; 🇺🇸 Albuquerque, New Mexico, United States

Dent Neurologic Institute; 🇺🇸 Amherst, New York, United States

Neurological Associates of Long Island, P.C.; 🇺🇸 Lake Success, New York, United States

Oklahoma Research Foundation - MS Center of Excellence; 🇺🇸 Oklahoma City, Oklahoma, United States

Sibyl Wray Neurology PC; 🇺🇸 Knoxville, Tennessee, United States

University of Texas Health Science Center at Houston; 🇺🇸 Houston, Texas, United States

Clinical Trial Network; 🇺🇸 Houston, Texas, United States

Bhupesh Dihenia, MD, PA; 🇺🇸 Lubbock, Texas, United States

UW Medicine MS Center; 🇺🇸 Seattle, Washington, United States

Multicare Neuroscience Center of Washington; 🇺🇸 Tacoma, Washington, United States

Virginia Mason Medical Center; 🇺🇸 Seattle, Washington, United States