Antigen-specific immune therapy (CNP-106) for treatment of generalised myasthenia gravis: rationale and design of first-in-human randomised controlled trial.
Samantha G Brew, Molly Frey, Derrick P McCarthy...
https://pubmed.ncbi.nlm.nih.gov/39720510Actively Recruiting
Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
ID06106672
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
Led by COUR Pharmaceutical Development Company, Inc. · Updated on 2025-02-13
54
Participants Needed
17
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a Phase 1b/2a first-in-human clinical trial to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106 in adults aged 18 to 75 with generalized myasthenia gravis. The study aims to understand how the drug behaves in the body and its potential effects on this condition, with participants carefully selected based on specific disease criteria. Participants will receive either CNP-106, delivered as a 200 mL intravenous infusion on Day 1 and Day 8, or a placebo infusion, in a randomized and double-blind design. The study includes a screening period of up to 42 days followed by 180 days of active study participation, during which researchers will monitor responses to the treatment and any adverse events. During the trial, participants will undergo various assessments including blood tests to measure immune cell changes at days 60, 90, and 180, and evaluations of safety through monitoring adverse events. The primary focus is on the frequency of adverse events throughout the 180-day treatment period. The study lasts a total of 222 days, including screening and active treatment, with participants regularly followed to assess both safety and biological effects of the drug.
CONDITIONS
Brief Title
Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis
Who Can Participate
Age: 18Years - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written informed consent.
- Men and non-pregnant women aged 18 to 75 years.
- Female participants of childbearing potential must agree to use two effective forms of birth control or practice abstinence during the study.
- Diagnosed with Myasthenia Gravis Foundation of America Clinical Classification Class III or IV (Cohort 1); Cohort 2 includes Class II-IV after safety review.
- Positive for anti-AChR antibodies by radioimmunoassay.
- MG-ADL Score of 6 or higher with at least 50% non-ocular symptoms.
- QMG Score of 11 or higher at screening and baseline.
- Stable dose of MG symptom medications for at least 90 days prior to enrollment.
- Female participants agree not to breastfeed or donate ova during the study.
- Male participants with partners of childbearing potential agree to use effective birth control throughout the study.
You will not qualify if you...
- MGFA Clinical Classification Class I or V.
- History of cerebrovascular accident within the past 12 months.
- MG-ADL Score less than 6 or with less than 50% non-ocular symptoms.
- QMG Score less than 11 at screening.
- Use of certain immunosuppressive or immunomodulatory drugs within specified timeframes before dosing.
- Use of immunoglobulins or plasma exchange within 4 weeks before screening.
- Thymectomy or thymic surgery within 12 months prior to screening.
- Untreated thymic malignancy, carcinoma, or thymoma.
- History of tuberculosis or positive PPD skin test.
- Recent live or subunit vaccinations prior to screening or planned vaccinations during the study.
- Positive tests for hepatitis B, hepatitis C, or HIV.
- Active immune disorders other than MG unless approved by medical monitor.
- Significant cardiovascular disease or recent malignancy.
- History of mast cell activation disease.
- Inability to adhere to study procedures.
- Prior investigational therapy use except CNP-106 within specified timeframes.
- Known sensitivity to components of CNP-106 (PLGA, sucrose, mannitol, sodium citrate).
AI-Screening
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Your Study Journey
Screening
Duration - Up to 6 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) prior to enrollment
Treatment
Duration - Approximately 26 weeks (180 days)
Participants receive intravenous infusions of CNP-106 or placebo on Day 1 and Day 8. The treatment phase includes assessments of safety, tolerability, pharmacodynamics, and efficacy.
Multiple visits including infusions and follow-up assessments through Day 180
Trial Site Locations
Total: 17 locations
1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Actively Recruiting
2
Neuromuscular Clinic and Research Center
Phoenix, Arizona, United States, 85028
Not Yet Recruiting
3
Infusion for Health
Brea, California, United States, 92835
Actively Recruiting
4
University of California, Irvine
Orange, California, United States, 92868
Actively Recruiting
5
Yale University
New Haven, Connecticut, United States, 06516
Actively Recruiting
6
Atlantis Research
Miami, Florida, United States, 33173
Actively Recruiting
7
Quantix Research, LLC
Miami, Florida, United States, 33173
Actively Recruiting
8
University of South Florida
Tampa, Florida, United States, 33612
Actively Recruiting
9
Insight Hospital and Medical Center
Chicago, Illinois, United States, 60608
Actively Recruiting
10
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Not Yet Recruiting
11
Insight Research Institute, Dearborn
Dearborn, Michigan, United States, 48126
Actively Recruiting
12
University of Missouri, NextGen Precision Health
Columbia, Missouri, United States, 65211
Actively Recruiting
13
Ohio State University Wexner Medical Center
Colombus, Ohio, United States, 43221
Not Yet Recruiting
14
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Not Yet Recruiting
15
Nerve and Muscle Center of Texas
Houston, Texas, United States, 77030
Actively Recruiting
16
Prolato Clinical Research Center
Houston, Texas, United States, 77054
Actively Recruiting
17
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Not Yet Recruiting
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Research Team
J
Joseph Mide
H
Harold Lee
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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