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

NCT ID: NCT06106672

Last Updated: 2025-02-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-30

Study Completion Date

2026-08-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Myasthenia Gravis Generalized Myasthenia AChR Myasthenia Gravis MuSK MG

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CNP-106

200 mL intravenous infusion on Day 1 and Day 8: CNP-106

Group Type EXPERIMENTAL

CNP-106

Intervention Type DRUG

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

CNP-106 Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

CNP-106 Placebo

Interventions

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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).

Intervention Type DRUG

Placebo

CNP-106 Placebo

Intervention Type OTHER

Eligibility Criteria

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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.
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).
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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COUR Pharmaceutical Development Company, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Roy First, MD

Role: STUDY_CHAIR

COUR Pharmaceutical

Locations

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Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status RECRUITING

Neuromuscular Clinic and Research Center

Phoenix, Arizona, United States

Site Status NOT_YET_RECRUITING

Infusion for Health

Brea, California, United States

Site Status RECRUITING

University of California, Irvine

Orange, California, United States

Site Status RECRUITING

Yale University

New Haven, Connecticut, United States

Site Status RECRUITING

Atlantis Research

Miami, Florida, United States

Site Status RECRUITING

Quantix Research, LLC

Miami, Florida, United States

Site Status RECRUITING

University of South Florida

Tampa, Florida, United States

Site Status RECRUITING

Insight Hospital and Medical Center

Chicago, Illinois, United States

Site Status RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status NOT_YET_RECRUITING

Insight Research Institute, Dearborn

Dearborn, Michigan, United States

Site Status RECRUITING

University of Missouri, NextGen Precision Health

Columbia, Missouri, United States

Site Status RECRUITING

Ohio State University Wexner Medical Center

Colombus, Ohio, United States

Site Status NOT_YET_RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status NOT_YET_RECRUITING

Nerve and Muscle Center of Texas

Houston, Texas, United States

Site Status RECRUITING

Prolato Clinical Research Center

Houston, Texas, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status NOT_YET_RECRUITING

Countries

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United States

Central Contacts

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Joseph Mide

Role: CONTACT

281-254-6305

Harold Lee

Role: CONTACT

859-613-9147

Facility Contacts

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Nicole Turcotte

Role: primary

602-406-4775

Lucia Rodriguez

Role: primary

480-314-1007 ext. 6

Danielle Mendoza

Role: primary

626-536-8974

Archita Patel

Role: primary

714-456-2275

Erika Renkl

Role: primary

860-304-5626

Laritza L Enriquez

Role: primary

786-536-7779

Hector Fernandez

Role: primary

305-230-7371

Naraly Requena

Role: primary

813-974-0575

Abeer Eghzawi

Role: primary

312-567-2224

Abby Davis

Role: primary

913-945-9934

Albaraa Alkilani

Role: primary

8102751964

Neetha Gali

Role: primary

(573) 882-3065

Julie Agriesti

Role: primary

614-293-4098

Alison Line

Role: primary

843-792-2845

Amy Megerle

Role: primary

713-654-4900

Rida Amer

Role: primary

8323389118

Taylor Parkinson

Role: primary

804-482-1833

References

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G Brew S, Frey M, P McCarthy D, Elhofy A, Nowak RJ. Antigen-specific immune therapy (CNP-106) for treatment of generalised myasthenia gravis: rationale and design of first-in-human randomised controlled trial. BMJ Neurol Open. 2024 Dec 18;6(2):e000836. doi: 10.1136/bmjno-2024-000836. eCollection 2024.

Reference Type DERIVED
PMID: 39720510 (View on PubMed)

Other Identifiers

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CNP-106-5.001

Identifier Type: -

Identifier Source: org_study_id

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