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

February 12, 2025 updated by: COUR Pharmaceutical Development Company, Inc.

A Phase 1b/2a Double Blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of CNP-106 in Subjects Ages 18-75 with Generalized Myasthenia Gravis

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

54

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
      • Phoenix, Arizona, United States, 85028
        • Not yet recruiting
        • Neuromuscular Clinic and Research Center
        • Contact:
        • Contact:
          • Kumaraswamy Sivakumar, MD
    • California
      • Brea, California, United States, 92835
      • Orange, California, United States, 92868
        • Recruiting
        • University of California, Irvine
        • Contact:
        • Contact:
          • Ali A Habib, MD
    • Connecticut
      • New Haven, Connecticut, United States, 06516
        • Recruiting
        • Yale University
        • Contact:
        • Contact:
          • Bhaskar Roy, MD
    • Florida
      • Miami, Florida, United States, 33173
      • Miami, Florida, United States, 33173
      • Tampa, Florida, United States, 33612
        • Recruiting
        • University of South Florida
        • Contact:
          • Tuan Vu, MD
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60608
        • Recruiting
        • Insight Hospital and Medical Center
        • Contact:
        • Contact:
          • Aburashed
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Not yet recruiting
        • University of Kansas Medical Center
        • Contact:
        • Contact:
          • Mazen DiMachkie
    • Michigan
      • Dearborn, Michigan, United States, 48126
        • Recruiting
        • Insight Research Institute, Dearborn
        • Contact:
        • Contact:
          • Taofik Nasrat
    • Missouri
      • Columbia, Missouri, United States, 65211
        • Recruiting
        • University of Missouri, NextGen Precision Health
        • Contact:
        • Contact:
          • William Arnold
    • Ohio
      • Colombus, Ohio, United States, 43221
        • Not yet recruiting
        • Ohio State University Wexner Medical Center
        • Contact:
        • Contact:
          • Miriam Freimer, MD
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Not yet recruiting
        • Medical University of South Carolina
        • Contact:
          • Katherine Ruzhansky, MD
        • Contact:
    • Texas
      • Houston, Texas, United States, 77054
        • Recruiting
        • Prolato Clinical Research Center
        • Contact:
        • Contact:
          • Eddie Patton
      • Houston, Texas, United States, 77030
        • Recruiting
        • Nerve And Muscle Center Of Texas
        • Contact:
        • Contact:
          • Aziz Shaibani, MD
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Not yet recruiting
        • Virginia Commonwealth University
        • Contact:
        • Contact:
          • Gordon Smith, MD, FAAN

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 during the clinical study, have a negative pregnancy test at the Screening Visit, and agree to one of the following:

    • Use two highly effective forms of birth control starting at initial screening and continuing throughout the study duration.
    • Practice abstinence starting at initial screening and continuing throughout the study duration.
  4. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class III-IV (Cohort 1). Upon successful DMC review and approval of preliminary safety data obtained from Cohort 1 through Day 15, Cohort 2 will enroll subjects with MGFA Clinical Classification Class II-IV.
  5. 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.

7. Subjects with QMG Score ≥ 11 at Screening and Baseline Visit. 8. For subjects on any medication 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 and must agree not to increase their dose through clinical study duration unless reviewed and approved by the medical monitor and the site investigator.

9. Female subjects who agree to not breastfeed starting at initial screening and throughout the study duration.

10. Female subjects who agree to not donate ova starting at initial screening and throughout the study duration.

11. Male subjects with a spouse or partner of childbearing potential, who themselves and their spouse or partner agree to practice an effective form of birth control as discussed with the study doctor or study staff starting at Screening and throughout the study duration.

Exclusion Criteria:

  1. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class 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 Screen with ˂ 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 is longer);
    • Anti-FcRn inhibitors (ex. Efgartigimod) within 5 half-lives or 90 days after last dose (whichever is longer);
    • C5 complement inhibitor (ex. Eculizumab) within 5 half-lives or 90 days after last 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 discretion of the medical monitor and study site investigator.
  6. Subjects who have used immunoglobulins given SC or IV (SCIg or IVIg) or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
  7. Subjects who have had thymectomy or any other thymic surgery performed within 12 months prior to Screening.
  8. Subjects with untreated thymic malignancy, carcinoma, or thymoma.
  9. Subjects with a history of tuberculosis or positive PPD skin test.
  10. Subjects who have received administration of any live vaccine (other than intranasal Influenza) within 28 days or subunit vaccine within 14 days prior to Screening or are planning to receive any vaccination throughout the study duration.
  11. 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 for the study until 14 days following their last dose of the vaccine if applicable.
  12. Subjects with laboratory test results at Screening or prior to study dosing that are outside the normal limits and considered by the investigator to be clinically significant. Note: Clinically significant laboratory test results at screening that are related to the condition (MG) are acceptable as long as all inclusion and no other exclusion criteria are met.
  13. 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.
  14. Subjects with a history of or currently active immune disorders other than MG (including autoimmune disease) unless the condition, after discussion with the medical monitor and study site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
  15. Subjects with a history of or current active diseases other than myasthenia gravis requiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone, budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil) unless the condition, after discussion with the medical monitor and site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
  16. Subjects with a clinical history of significant cardiovascular disease as determined by the Investigator.
  17. Subjects with a complication or medical history of malignancy within the past 5 years which, in the investigator's opinion, makes the subject unsuitable for study participation.
  18. Subjects with a history of mast cell activation disease.
  19. Subjects who, in the investigator's opinion, will be unable to adhere to study procedures.
  20. Subjects who have received an investigational therapy other than CNP-106 within 28 days or 5 half-lives, whichever is longer, prior to Screening.
  21. Subjects with any known active condition which, in the investigator's opinion, makes the subject unsuitable for study participation.
  22. Known sensitivity to any components of CNP-106 (PLGA, sucrose, mannitol or sodium citrate).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CNP-106
200 mL intravenous infusion on Day 1 and Day 8: CNP-106
Drug: CNP-106
CNP-106 is comprised of an antigenic AChR Peptide Pool (~1 μg of each AChRα and AChRε peptide comprising AChR Peptide Pool Drug Substance per mg particles) dispersed within a negatively charged (-30 to -60 mV) polymer matrix of PLGA (Poly (DL-lactide-co-glycolide, 50:50 acid-end group)) particles (400-800 nm in size).
Placebo Comparator: Placebo
CNP-106 Placebo
Other: Placebo
CNP-106 Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs),
Time Frame: Through study day 180
Frequency tables will be presented by treatment group for all AEs and SAEs by System Organ Class (SOC) and Preferred Term (PT). Frequency tables will also be produced by treatment group for AEs leading to COUR Pharmaceuticals Development Company, Inc. Confidential CNP-106-5.001 Protocol; IND 28774 Page 53 of 65 discontinuation from IP and study, by severity, and by causality. No formal statistical testing will be done.
Through study day 180

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in antigen specific CD4+ and CD8+ T cell levels in PBMC at Day 60, 90, and 180.
Time Frame: Through study day 180
The mean change from Baseline to the endpoint within CNP-106 and Placebo treatment groups will be analyzed using ANOVA.
Through study day 180
Change from baseline in activated antigen specific CD4+ and CD8+ T cell levels in PBMC at Day 60, 90, and 180.
Time Frame: Through study day 180
The mean change from Baseline to the endpoint within CNP-106 and Placebo treatment groups will be analyzed using ANOVA.
Through study day 180

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

COUR Pharmaceutical Development Company, Inc.

Investigators

  • Study Chair: Roy First, MD, COUR Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 30, 2024

Primary Completion (Estimated)

June 1, 2026

Study Completion (Estimated)

August 1, 2026

Study Registration Dates

First Submitted

October 24, 2023

First Submitted That Met QC Criteria

October 24, 2023

First Posted (Actual)

October 30, 2023

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 12, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CNP-106-5.001

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

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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