NCT06106672

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
54

participants targeted

Target at P50-P75 for phase_1

Timeline
2mo left

Started May 2024

Typical duration for phase_1

Geographic Reach
1 country

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress89%
May 2024Aug 2026

First Submitted

Initial submission to the registry

October 24, 2023

Completed
6 days until next milestone

First Posted

Study publicly available on registry

October 30, 2023

Completed
7 months until next milestone

Study Start

First participant enrolled

May 30, 2024

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2026

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2026

Last Updated

February 13, 2025

Status Verified

January 1, 2025

Enrollment Period

2 years

First QC Date

October 24, 2023

Last Update Submit

February 12, 2025

Conditions

Myasthenia GravisGeneralized MyastheniaAChR Myasthenia GravisMuSK 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

CNP-106DRUG

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
PlaceboOTHER

CNP-106 Placebo

Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (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 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).
  • , 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. 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.
  • \. 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 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);
  • 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.
  • 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 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

RECRUITING

Neuromuscular Clinic and Research Center

Phoenix, Arizona, 85028, United States

NOT YET RECRUITING

Infusion for Health

Brea, California, 92835, United States

RECRUITING

University of California, Irvine

Orange, California, 92868, United States

RECRUITING

Yale University

New Haven, Connecticut, 06516, United States

RECRUITING

Atlantis Research

Miami, Florida, 33173, United States

RECRUITING

Quantix Research, LLC

Miami, Florida, 33173, United States

RECRUITING

University of South Florida

Tampa, Florida, 33612, United States

RECRUITING

Insight Hospital and Medical Center

Chicago, Illinois, 60608, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

NOT YET RECRUITING

Insight Research Institute, Dearborn

Dearborn, Michigan, 48126, United States

RECRUITING

University of Missouri, NextGen Precision Health

Columbia, Missouri, 65211, United States

RECRUITING

Ohio State University Wexner Medical Center

Colombus, Ohio, 43221, United States

NOT YET RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

NOT YET RECRUITING

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

RECRUITING

Prolato Clinical Research Center

Houston, Texas, 77054, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

NOT YET RECRUITING

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: 39720510DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous SystemNervous System NeoplasmsNeoplasms by SiteNeoplasmsParaneoplastic SyndromesAutoimmune Diseases of the Nervous SystemNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Junction DiseasesNeuromuscular DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • Roy First, MD

    COUR Pharmaceutical

    STUDY CHAIR

Central Study Contacts

Joseph Mide

CONTACT

281-254-6305jmide@courpharma.com

Harold Lee

CONTACT

859-613-9147hlee@courpharma.com

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 (Estimated)

June 1, 2026

Study Completion (Estimated)

August 1, 2026

Last Updated

February 13, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will not share

Locations

US(17)