Trial Title:
Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis
NCT ID:
NCT06106672
Condition:
Myasthenia Gravis
Generalized Myasthenia
AChR Myasthenia Gravis
MuSK MG
Conditions: Official terms:
Myasthenia Gravis
Muscle Weakness
Study type:
Interventional
Study phase:
Phase 1/Phase 2
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Intervention:
Intervention type:
Drug
Intervention name:
CNP-106
Description:
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).
Arm group label:
CNP-106
Intervention type:
Other
Intervention name:
Placebo
Description:
CNP-106 Placebo
Arm group label:
Placebo
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.
Detailed 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.
Criteria for eligibility:
Criteria:
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.
12. Subjects with laboratory test results at Screening or prior to study dosing that are
outside the normal limits and considered by the investigator to be clinically
significant. Note: Clinically significant laboratory test results at screening that
are related to the condition (MG) are acceptable as long as all inclusion and no
other exclusion criteria are met.
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).
Gender:
All
Minimum age:
18 Years
Maximum age:
75 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Barrow Neurological Institute
Address:
City:
Phoenix
Zip:
85013
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Nicole Turcotte
Phone:
602-406-4775
Email:
nicole.turcotte@dignityhealth.org
Investigator:
Last name:
Shafeeq Ladha, MD
Email:
Principal Investigator
Facility:
Name:
Neuromuscular Clinic and Research Center
Address:
City:
Phoenix
Zip:
85028
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Lucia Rodriguez
Phone:
480-314-1007
Phone ext:
6
Email:
lrodriguez@nrcaz.com
Investigator:
Last name:
Kumaraswamy Sivakumar, MD
Email:
Principal Investigator
Facility:
Name:
Infusion for Health
Address:
City:
Brea
Zip:
92835
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Danielle Mendoza
Phone:
626-536-8974
Email:
damendoza@infusionforhealth.com
Investigator:
Last name:
Derrick Florin, MD
Email:
Principal Investigator
Facility:
Name:
University of California, Irvine
Address:
City:
Orange
Zip:
92868
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Archita Patel
Phone:
714-456-2275
Email:
chuny@hs.uci.edu
Investigator:
Last name:
Ali A Habib, MD
Email:
Principal Investigator
Facility:
Name:
Yale University
Address:
City:
New Haven
Zip:
06516
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Erika Renkl
Phone:
860-304-5626
Email:
erika.renkl@yale.edu
Investigator:
Last name:
Bhaskar Roy, MD
Email:
Principal Investigator
Facility:
Name:
Quantix Research, LLC
Address:
City:
Miami
Zip:
33173
Country:
United States
Status:
Recruiting
Contact:
Last name:
Hector Fernandez
Phone:
305-230-7371
Email:
hector.fernandez@quantixresearch.com
Investigator:
Last name:
Sergio Jaramillo, MD
Email:
Principal Investigator
Facility:
Name:
University of South Florida
Address:
City:
Tampa
Zip:
33612
Country:
United States
Status:
Recruiting
Contact:
Last name:
Naraly Requena
Phone:
813-974-0575
Email:
naraly@usf.edu
Investigator:
Last name:
Tuan Vu, MD
Email:
Principal Investigator
Facility:
Name:
University of Kansas Medical Center
Address:
City:
Kansas City
Zip:
66160
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Abby Davis
Phone:
913-945-9934
Email:
adavis54@kumc.edu
Investigator:
Last name:
Mazen DiMachkie
Email:
Principal Investigator
Facility:
Name:
University of Minnesota
Address:
City:
Minneapolis
Zip:
55455
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Sarah Hilbert
Phone:
612-624-5978
Email:
hilbe010@umn.edu
Investigator:
Last name:
Jeffrey Allen, MD
Email:
Principal Investigator
Facility:
Name:
Ohio State University Wexner Medical Center
Address:
City:
Columbus
Zip:
43221
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Julie Agriesti
Phone:
614-293-4098
Email:
Julie.Agriesti@osumc.edu
Investigator:
Last name:
Miriam Freimer, MD
Email:
Principal Investigator
Facility:
Name:
Medical University of South Carolina
Address:
City:
Charleston
Zip:
29425
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Alison Line
Phone:
843-792-2845
Email:
line@musc.edu
Investigator:
Last name:
Katherine Ruzhansky, MD
Email:
Principal Investigator
Facility:
Name:
Nerve and Muscle Center of Texas
Address:
City:
Houston
Zip:
77030
Country:
United States
Status:
Recruiting
Contact:
Last name:
Amy Megerle
Phone:
713-654-4900
Email:
houneuamy@msn.com
Investigator:
Last name:
Aziz Shaibani, MD
Email:
Principal Investigator
Facility:
Name:
Virginia Commonwealth University
Address:
City:
Richmond
Zip:
23298
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Taylor Parkinson
Phone:
804-482-1833
Email:
taylor.parkinson@vcuhealth.org
Investigator:
Last name:
Gordon Smith, MD, FAAN
Email:
Principal Investigator
Start date:
May 30, 2024
Completion date:
August 2026
Lead sponsor:
Agency:
COUR Pharmaceutical Development Company, Inc.
Agency class:
Industry
Source:
COUR Pharmaceutical Development Company, Inc.
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06106672
