CNP-106
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.
Eligibility
- Subjects who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations.
- Men and non-pregnant women, ages 18-75 years inclusive.
- 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.
- 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.
- Subjects positive for anti-AChR antibodies by radioimmunoassay (RIA) (Mayo Clinic).
- 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.
Main
- Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class I or V.
- Subjects with a history of cerebrovascular accident in the past 12 months.
- 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.
- Subjects with QMG Score < 11 at Screen.
- 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.
- Subjects who have used immunoglobulins given SC or IV (SCIg or IVIg) or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
- Subjects who have had thymectomy or any other thymic surgery performed within 12 months prior to Screening.
- Subjects with untreated thymic malignancy, carcinoma, or thymoma.
- Subjects with a history of tuberculosis or positive PPD skin test.