RECRUITING

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

Conditions

Myasthenia Gravis Generalized Myasthenia AChR Myasthenia Gravis MuSK MG

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-05-30

Study Completion:2026-08

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06106672

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.

Ages Eligible for Study:18 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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). 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.	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).

Inclusion Criteria

Exclusion 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).
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.

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).

Contacts and Locations

Study Contact

Jason Penix

CONTACT

773-339-5745

jpenix@courpharma.com

Lynlee Muehring

CONTACT

773-322-0646

lmuehring@courpharma.com

Principal Investigator

Roy First, MD

STUDY_CHAIR

COUR Pharmaceutical

Study Locations (Sites)

Barrow Neurological Institute

Phoenix, Arizona, 85013

United States

Neuromuscular Clinic and Research Center

Phoenix, Arizona, 85028

United States

Infusion for Health

Brea, California, 92835

United States

University of California, Irvine

Orange, California, 92868

United States

Yale University

New Haven, Connecticut, 06516

United States

Quantix Research, LLC

Miami, Florida, 33173

United States

University of South Florida

Tampa, Florida, 33612

United States

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

University of Minnesota

Minneapolis, Minnesota, 55455

United States

Ohio State University Wexner Medical Center

Columbus, Ohio, 43221

United States

Medical University of South Carolina

Charleston, South Carolina, 29425

United States

Nerve and Muscle Center of Texas

Houston, Texas, 77030

United States

Virginia Commonwealth University

Richmond, Virginia, 23298

United States

Collaborators and Investigators

Sponsor: COUR Pharmaceutical Development Company, Inc.

Roy First, MD, STUDY_CHAIR, 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 Registration Dates

Study Start Date2024-05-30

Study Completion Date2026-08

Study Record Updates

Study Start Date2024-05-30

Study Completion Date2026-08

Terms related to this study

Additional Relevant MeSH Terms

Myasthenia Gravis
Generalized Myasthenia
AChR Myasthenia Gravis
MuSK MG