Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects with Myasthenia Gravis

Last updated: February 12, 2025
Sponsor: COUR Pharmaceutical Development Company, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Neuropathy

Polymyositis (Inflammatory Muscle Disease)

Sarcopenia

Treatment

Placebo

CNP-106

Clinical Study ID

NCT06106672
CNP-106-5.001
  • Ages 18-75
  • All Genders

Study Summary

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subjects who are willing and able to provide Institutional Review Board (IRB)approved written informed consent and privacy language as per national regulations.

  2. Men and non-pregnant women, ages 18-75 years inclusive.

  3. Female subjects of childbearing potential must agree not to become pregnant duringthe clinical study, have a negative pregnancy test at the Screening Visit, and agreeto one of the following:

  • Use two highly effective forms of birth control starting at initial screeningand continuing throughout the study duration.

  • Practice abstinence starting at initial screening and continuing throughout thestudy duration.

  1. Subjects with a Myasthenia Gravis Foundation of America Clinical ClassificationClass III-IV (Cohort 1). Upon successful DMC review and approval of preliminarysafety data obtained from Cohort 1 through Day 15, Cohort 2 will enroll subjectswith MGFA Clinical Classification Class II-IV.

  2. Subjects positive for anti-AChR antibodies by radioimmunoassay (RIA) (Mayo Clinic).

6, Subjects with MG-ADL Score ≥ 6 at Screening and Baseline Visit with ≥ 50% of the score derived from non-ocular symptoms.

  1. Subjects with QMG Score ≥ 11 at Screening and Baseline Visit. 8. For subjects on anymedication used to treat the symptoms of MG (ex. Corticosteroids, pyridostigmine),subjects must be on a stable dose for a minimum of 90 days prior to enrollment andmust agree not to increase their dose through clinical study duration unlessreviewed and approved by the medical monitor and the site investigator.

  2. Female subjects who agree to not breastfeed starting at initial screening andthroughout the study duration.

  3. Female subjects who agree to not donate ova starting at initial screening andthroughout the study duration.

  4. Male subjects with a spouse or partner of childbearing potential, who themselves andtheir spouse or partner agree to practice an effective form of birth control asdiscussed with the study doctor or study staff starting at Screening and throughoutthe study duration.

Exclusion

Exclusion Criteria:

  1. Subjects with a Myasthenia Gravis Foundation of America Clinical ClassificationClass I or V.

  2. Subjects with a history of cerebrovascular accident in the past 12 months.

  3. Subjects with MG-ADL Score < 6 at Screen or Subjects with MG-ADL Score ≥ 6 at Screenwith ˂ 50% of the score derived from non-ocular symptoms.

  4. Subjects with QMG Score < 11 at Screen.

  5. Subjects who have used the following medications:

  • Tacrolimus within 6 months prior to the first dosing;

  • Methotrexate within 5 half-lives or 90 days after last dose (whichever islonger);

  • Anti-FcRn inhibitors (ex. Efgartigimod) within 5 half-lives or 90 days afterlast dose (whichever is longer);

  • C5 complement inhibitor (ex. Eculizumab) within 5 half-lives or 90 days afterlast dose (whichever is longer);

  • Anti-CD20 (ex. Rituximab) within 5 half-lives for 90 days after last dose (whichever is longer);

  • Inclusion of subjects on other immunomodulatory drugs will be at the discretionof the medical monitor and study site investigator.

  1. Subjects who have used immunoglobulins given SC or IV (SCIg or IVIg) orplasmapheresis/plasma exchange (PE) within 4 weeks before Screening.

  2. Subjects who have had thymectomy or any other thymic surgery performed within 12months prior to Screening.

  3. Subjects with untreated thymic malignancy, carcinoma, or thymoma.

  4. Subjects with a history of tuberculosis or positive PPD skin test.

  5. Subjects who have received administration of any live vaccine (other than intranasalInfluenza) within 28 days or subunit vaccine within 14 days prior to Screening orare planning to receive any vaccination throughout the study duration.

  6. Subjects who have received any COVID-19 vaccine within 14 days prior to Screening.Subjects who have received the first dose of any COVID-19 vaccine may not screen forthe study until 14 days following their last dose of the vaccine if applicable.

  7. Subjects with laboratory test results at Screening or prior to study dosing that areoutside the normal limits and considered by the investigator to be clinicallysignificant. Note: Clinically significant laboratory test results at screening thatare related to the condition (MG) are acceptable as long as all inclusion and noother exclusion criteria are met.

  8. Subjects with positive test results for hepatitis B surface antigen (HbsAg),hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV)antigen/antibody as determined at Screening.

  9. Subjects with a history of or currently active immune disorders other than MG (including autoimmune disease) unless the condition, after discussion with themedical monitor and study site investigator, has been deemed to be acceptable forthe subject's participation in this clinical study.

  10. Subjects with a history of or current active diseases other than myasthenia gravisrequiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone,budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil) unlessthe condition, after discussion with the medical monitor and site investigator, hasbeen deemed to be acceptable for the subject's participation in this clinical study.

  11. Subjects with a clinical history of significant cardiovascular disease as determinedby the Investigator.

  12. Subjects with a complication or medical history of malignancy within the past 5years which, in the investigator's opinion, makes the subject unsuitable for studyparticipation.

  13. Subjects with a history of mast cell activation disease.

  14. Subjects who, in the investigator's opinion, will be unable to adhere to studyprocedures.

  15. Subjects who have received an investigational therapy other than CNP-106 within 28days or 5 half-lives, whichever is longer, prior to Screening.

  16. Subjects with any known active condition which, in the investigator's opinion, makesthe subject unsuitable for study participation.

  17. Known sensitivity to any components of CNP-106 (PLGA, sucrose, mannitol or sodiumcitrate).

Study Design

Total Participants: 54
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
May 30, 2024
Estimated Completion Date:
August 31, 2026

Study Description

This is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. The clinical study lasts 222-days (up to 42 days for Screening, 180 Study Days). Subjects ages 18-75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study.

Connect with a study center

  • Barrow Neurological Institute

    Phoenix, Arizona 85013
    United States

    Active - Recruiting

  • Neuromuscular Clinic and Research Center

    Phoenix, Arizona 85028
    United States

    Site Not Available

  • Infusion for Health

    Brea, California 92835
    United States

    Active - Recruiting

  • University of California, Irvine

    Orange, California 92868
    United States

    Active - Recruiting

  • Yale University

    New Haven, Connecticut 06516
    United States

    Active - Recruiting

  • Atlantis Research

    Miami, Florida 33173
    United States

    Active - Recruiting

  • Quantix Research, LLC

    Miami, Florida 33173
    United States

    Active - Recruiting

  • University of South Florida

    Tampa, Florida 33612
    United States

    Active - Recruiting

  • Insight Hospital and Medical Center

    Chicago, Illinois 60608
    United States

    Active - Recruiting

  • Research Site

    Chicago, Illinois 60601
    United States

    Site Not Available

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • Insight Research Institute, Dearborn

    Dearborn, Michigan 48126
    United States

    Active - Recruiting

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Missouri, NextGen Precision Health

    Columbia, Missouri 65211
    United States

    Active - Recruiting

  • Ohio State University Wexner Medical Center

    Colombus, Ohio 43221
    United States

    Site Not Available

  • Ohio State University Wexner Medical Center

    Columbus, Ohio 43221
    United States

    Site Not Available

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • Nerve and Muscle Center of Texas

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Prolato Clinical Research Center

    Houston, Texas 77054
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Site Not Available

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