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

NCT06106672 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CNP-106-5.001
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 17

Brief 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.

Conditions

Myasthenia Gravis; Generalized Myasthenia; AChR Myasthenia Gravis; MuSK MG

Outcome Measures

Primary Outcomes (1)

• Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs),

  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 Outcomes (2)

• Change from baseline in antigen specific CD4+ and CD8+ T cell levels in PBMC at Day 60, 90, and 180.

  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.

  Through study day 180

Study Arms (2)

CNP-106

EXPERIMENTAL

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

Drug: CNP-106

Placebo

PLACEBO COMPARATOR

CNP-106 Placebo

Other: Placebo

Interventions

Placebo OTHER

CNP-106 Placebo

Placebo

CNP-106 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).

CNP-106

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 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 II-IV.
• Subjects positive for anti-AChR antibodies by radioimmunoassay (RIA) (LabCorp Central Laboratory Services Inc.).
• Subjects with MG-ADL Score ≥6 at Screening and Baseline Visit with ≥50% of the score derived from non-ocular symptoms.
• Subjects with QMG Score ≥11 at Screening and Baseline Visit.
• For subjects on any medication used to treat the symptoms of MG (e.g. 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 throughout the study duration unless reviewed and approved by the medical monitor and the site investigator.
• Female subjects who agree to not breastfeed starting at initial screening and throughout the study duration.
• Female subjects who agree to not donate ova starting at initial screening and throughout the study duration.
• 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.

You may not qualify if:

• 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 may enter the study following the washout periods for medications as described below prior to screening and refrain from usage throughout the study:
• Tacrolimus within 6 months
• Azathioprine, cyclosporine, methotrexate, or mycophenolate mofetil within 90 days
• Anti-FcRn therapies:
• Cyclic Anti-FcRns (e.g., efgartigimod alfa, rozanolixizumab) within 50 days
• Chronic Anti-FcRns (e.g., nipocalimab) within 80 days;
• C5 complement inhibitor (e.g., eculizumab) within 90 days
• Anti-CD20 (e.g., rituximab) within 6 months;
• 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.

Sponsors & Collaborators

• COUR Pharmaceutical Development Company, Inc.: lead

Study Sites (17)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

Infusion for Health

Brea, California, 92835, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

University of California, Davis

Sacramento, California, 95817, United States

Location

Yale University

New Haven, Connecticut, 06516, United States

Location

Atlantis Research

Miami, Florida, 33173, United States

Location

Quantix Research, LLC

Miami, Florida, 33173, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Insight Hospital and Medical Center

Chicago, Illinois, 60608, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Insight Research Institute, Dearborn

Dearborn, Michigan, 48126, United States

Location

University of Missouri, NextGen Precision Health

Columbia, Missouri, 65211, United States

Location

Ohio State University Wexner Medical Center

Colombus, Ohio, 43221, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

Prolato Clinical Research Center

Houston, Texas, 77054, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Related Publications (1)

• 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.

  PMID: 39720510 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Paul Peloso, MD

  COUR Pharmaceutical

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2023
• First Posted: October 30, 2023
• Study Start: May 30, 2024
• Primary Completion: June 1, 2026
• Study Completion: August 1, 2026
• Last Updated: June 9, 2026

Record last verified: 2026-06

Locations

US (17)