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

Relapsing Remitting Multiple Sclerosis Clinical Trial

— VISTA  

Official title:

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Multicenter Study to Evaluate the Safety and Efficacy of Oral PIPE-307 as an Adjunctive Treatment in Subjects With Relapsing-Remitting Multiple Sclerosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06083753
• Other study ID #: PIPE 307-201
• Status: Recruiting
• Phase: Phase 2
• Start date: November 6, 2023
• Est. completion date: September 2025

Study information

• Verified date: April 2024
• Source: Contineum Therapeutics
• Contact: Jules Lee
• Email: jlee[@]contineum-tx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 168
• Est. completion date: September 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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.

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.

Related Conditions & MeSH terms

• Multiple Sclerosis
• Multiple Sclerosis, Relapsing-Remitting
• Relapsing Remitting Multiple Sclerosis
• Sclerosis

Intervention

Drug:
• PIPE-307 Dose A
Subjects will receive daily oral doses of PIPE-307
• PIPE-307 Dose B
Subjects will receive daily oral doses of PIPE-307
• Placebo
Subjects will receive daily oral matching dose of Placebo

Locations

Country	Name	City	State
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	Shepherd Center	Atlanta	Georgia
United States	Sutter East Bay Medical Foundation	Berkeley	California
United States	MS and Neuromuscular Center of Excellence	Clearwater	Florida
United States	Colorado Springs Neurological Associates	Colorado Springs	Colorado
United States	Michigan Institute for Neurological Disorders (MIND)	Farmington Hills	Michigan
United States	Neurology Center of New England P.C.	Foxboro	Massachusetts
United States	Clinical Trial Network	Houston	Texas
United States	Indiana University Health Neuroscience Center, Adult Neurology Center	Indianapolis	Indiana
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Sibyl Wray Neurology PC	Knoxville	Tennessee
United States	Bhupesh Dihenia, MD, PA	Lubbock	Texas
United States	Aqualane Clinical Research	Naples	Florida
United States	Oklahoma Research Foundation - MS Center of Excellence	Oklahoma City	Oklahoma
United States	Arizona Neuroscience Research, LLC	Phoenix	Arizona
United States	Xenosciences	Phoenix	Arizona
United States	Accel Research Sites Network - Brain & Spine Institute	Port Orange	Florida
United States	Velocity Clinical Research, Raleigh Neurology	Raleigh	North Carolina
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	Velocity Clinical Research, Savannah Neurology Specialists	Savannah	Georgia
United States	UW Medicine MS Center	Seattle	Washington
United States	Virginia Mason Medical Center	Seattle	Washington
United States	Multicare Neuroscience Center of Washington	Tacoma	Washington
United States	Vero Beach Neurology And Research Institute	Vero Beach	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Contineum Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment-emergent adverse events (TEAE)	Number of participants with TEAEs	From baseline to week 26 (end of treatment period)
Primary	Change in binocular 2.5% low contrast letter acuity (LCLA)		From baseline to week 26 (end-of-study)
Secondary	Percentage of subjects with >/=5-letter gain in binocular 2.5% LCLA		From baseline to week 26
Secondary	Change in monocular 2.5% LCLA		From baseline to week 26
Secondary	Number of subjects with at least a 15% change in disability with the Timed 25-Foot Walk Test (T25WT)		From baseline to week 26
Secondary	Number of subjects with at least a 15% change in disability with the Nine-Hole Peg Test (9HPT)		From baseline to week 26
Secondary	Number of subjects with at least a 15% change in disability with the Symbol Digital Modality Test (SDMT)		From baseline to week 26
Secondary	Change in magnetic resonance imaging (MRI) measures of myelination and MS disease activity		From baseline to week 26
Secondary	Change in serum neurofilament light chain (NfL)		From baseline to week 26
Secondary	Pharmacokinetics: Change in blood concentration levels of PIPE-307		From baseline to week 30