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

Primary Purpose

Relapsing Remitting Multiple Sclerosis

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
PIPE-307 Dose A
PIPE-307 Dose B
Placebo
Sponsored by
Pipeline Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Relapsing Remitting Multiple Sclerosis focused on measuring Relapsing Remitting Multiple Sclerosis, PIPE 307, Multiple Sclerosis

Eligibility Criteria

18 Years - 50 Years (Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Placebo Comparator

    Arm Label

    PIPE-307 Dose A

    PIPE-307 Dose B

    Placebo

    Arm Description

    Outcomes

    Primary Outcome Measures

    Treatment-emergent adverse events (TEAE)
    Number of participants with TEAEs
    Change in binocular 2.5% low contrast letter acuity (LCLA)

    Secondary Outcome Measures

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

    Full Information

    First Posted
    October 9, 2023
    Last Updated
    October 9, 2023
    Sponsor
    Pipeline Therapeutics, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06083753
    Brief Title
    Study to Evaluate the Safety and Efficacy of PIPE-307 in Subjects With Relapsing-Remitting Multiple Sclerosis
    Acronym
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    October 2023 (Anticipated)
    Primary Completion Date
    August 2025 (Anticipated)
    Study Completion Date
    September 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Pipeline Therapeutics, Inc.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    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).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Relapsing Remitting Multiple Sclerosis
    Keywords
    Relapsing Remitting Multiple Sclerosis, PIPE 307, Multiple Sclerosis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigator
    Allocation
    Randomized
    Enrollment
    168 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    PIPE-307 Dose A
    Arm Type
    Experimental
    Arm Title
    PIPE-307 Dose B
    Arm Type
    Experimental
    Arm Title
    Placebo
    Arm Type
    Placebo Comparator
    Intervention Type
    Drug
    Intervention Name(s)
    PIPE-307 Dose A
    Intervention Description
    Subjects will receive daily oral doses of PIPE-307
    Intervention Type
    Drug
    Intervention Name(s)
    PIPE-307 Dose B
    Intervention Description
    Subjects will receive daily oral doses of PIPE-307
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Subjects will receive daily oral matching dose of Placebo
    Primary Outcome Measure Information:
    Title
    Treatment-emergent adverse events (TEAE)
    Description
    Number of participants with TEAEs
    Time Frame
    From baseline to week 26 (end of treatment period)
    Title
    Change in binocular 2.5% low contrast letter acuity (LCLA)
    Time Frame
    From baseline to week 26 (end-of-study)
    Secondary Outcome Measure Information:
    Title
    Percentage of subjects with >/=5-letter gain in binocular 2.5% LCLA
    Time Frame
    From baseline to week 26
    Title
    Change in monocular 2.5% LCLA
    Time Frame
    From baseline to week 26
    Title
    Number of subjects with at least a 15% change in disability with the Timed 25-Foot Walk Test (T25WT)
    Time Frame
    From baseline to week 26
    Title
    Number of subjects with at least a 15% change in disability with the Nine-Hole Peg Test (9HPT)
    Time Frame
    From baseline to week 26
    Title
    Number of subjects with at least a 15% change in disability with the Symbol Digital Modality Test (SDMT)
    Time Frame
    From baseline to week 26
    Title
    Change in magnetic resonance imaging (MRI) measures of myelination and MS disease activity
    Time Frame
    From baseline to week 26
    Title
    Change in serum neurofilament light chain (NfL)
    Time Frame
    From baseline to week 26
    Title
    Pharmacokinetics: Change in blood concentration levels of PIPE-307
    Time Frame
    From baseline to week 30

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    50 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Jules Lee
    Phone
    1-415-819-0405
    Email
    jlee@pipeline-tx.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    Julie Iwashita
    Phone
    1-408-813-9981
    Email
    jiwashita@pipeline-tx.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Stephen Huhn, MD
    Organizational Affiliation
    Pipeline Therapeutics, Inc.
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

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

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