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Safety, Tolerability and Immunogenic Response of CV-MG01 in Patients With Myasthenia Gravis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02609022
Recruitment Status : Active, not recruiting
First Posted : November 20, 2015
Last Update Posted : November 20, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Description
Brief Summary:
Study CV-0002 is the first clinical trial administering CV-MG01 in humans. This clinical trial is a safety and proof-of-concept study (proof of mechanism of action) intended to assess the safety, tolerability and immunogenic response following 3 subcutaneous injections of CV-MG01 as a potential therapeutic vaccine / active immunotherapy in myasthenia gravis (MG) patients.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis Biological: CV-MG01 Biological: Placebo Phase 1 Phase 2

Detailed Description:

Part A of the trial has been designed as a human safety pharmacology and therapeutic exploratory, parallel group, randomised, placebo-controlled, single centre, Investigator and subject-blind study using adaptive dose and sample size approaches.

At the end of part A of the present study, all patients, including those receiving placebo, will be monitored in an open label, long-term safety follow-up part B of the study to assess the treatment effects over time.


Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A First-in-human and Proof-of-concept Study to Assess the Safety, Tolerability and Immunogenic Response of CV-MG01, Acetylcholine Receptor Mimetic Peptides, as Potential Therapeutic Vaccine, in Patients With Myasthenia Gravis
Actual Study Start Date : March 2016
Primary Completion Date : September 12, 2017
Estimated Study Completion Date : November 30, 2017

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: CV-MG01
The therapeutic vaccine candidate, CV-MG01 comprises two short synthetic peptides separately conjugated to a carrier protein for the potential treatment of myasthenia gravis
Biological: CV-MG01

3 consecutive subcutaneous injections of CV-MG01. The three injections are planned for each patient on Days 1, 29 (+/- 3 days) and 85 (+/- 7 days), respectively.

Two dose levels: low and high dose.

Placebo Comparator: Placebo
Aluminium hydroxide adjuvant alone
Biological: Placebo
3 consecutive subcutaneous injections of placebo. The three injections are planned for each patient on Days 1, 29 (+/- 3 days) and 85 (+/- 7 days), respectively.


Outcome Measures

Primary Outcome Measures :
  1. Safety [ Time Frame: End of study part A (38 weeks) ]
    Safety assessed by laboratory tests, vital signs, electrocardiogram (ECG), adverse events, assessment of local tolerance, physical exams

  2. Immunogenicity [ Time Frame: End of study part A (38 weeks) ]
    To assess the immunogenic response after subcutaneous injections of CV-MG01 on the plasma levels of anti-peptide antibodies.


Secondary Outcome Measures :
  1. Biomarker [ Time Frame: End of study part A (38 weeks) ]
    To assess the effect of CV-MG01 subcutaneous injections on the plasma level of acetylcholine receptor antibodies.

  2. Clinical efficacy [ Time Frame: End of study part A (38 weeks) ]
    Clinical efficacy assessed by Quantitative MG testing Procedure extended with MG Composite Scale and MG-ADL (myasthenia gravis activities of daily living)


Other Outcome Measures:
  1. Immune response [ Time Frame: End of study part A (38 weeks) ]
    To explore changes in the humoral and cellular immune responses.


Eligibility Criteria

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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects, with ocular and generalised myasthenia gravis (grade 1-2-3).
  • Between the ages of 18 and 64 years, inclusive, at the time of the first injection.
  • A Body Mass Index (BMI) between 18 and 35 kg/m2, inclusive.
  • Patient with positive antibodies to AChR based on radioimmunoassay(RIA) (AChRAb ≥ 1 nmol/l); if available, historical data on AChR Ab levels over the last 2 years will be collected in the study records.
  • Patient may use corticosteroid treatment, equivalent to a daily dose of 30 mg prednisone or lower and stable (dose +/- 5 mg) during the 3 months before participation.
  • Patient may use one immunosuppressive drug with or without concomitant use of corticosteroid, providing that the dosage has been stable/unchanged for 3 months before participation.
  • Blood pressure and heart rate (supine & standing) within normal reference range for the population or investigator site, or results with acceptable deviations that are judged to be not clinically significant by the investigator.
  • Venous access sufficient to allow blood sampling as per the protocol.
  • Are reliable and willing to make themselves available for the duration of the study and are willing to follow study procedures.
  • Have given written informed consent approved by the relevant Ethics Committee (EC) governing the site.

Exclusion Criteria:

  • MG patients of Grade 4 or 5 based on myasthenia gravis foundation of America (MGFA) classification.
  • Patients with history or presence of a primary or recurrent malignant disease including the presence or history of a thymoma.
  • Thymectomy planned during part A of the study period or performed within 1 year prior to the first dose of study vaccine.
  • Any confirmed or suspected immunosuppressive or immunodeficient condition not related to the treatment of MG, including human immunodeficiency virus (HIV) infection, or a family history of congenital or hereditary immunodeficiency.
  • History or evidence of administration of immunoglobulins and/or any blood products within 3 months prior to the first dose of study vaccine or a planned administration of immunoglobulins during the first 3 months of the trial.
  • History or evidence of rituximab treatment within 6 months prior to first dose of study vaccine.
  • History or evidence of plasmapheresis within 3 months prior to the first dose of study vaccine or a planned plasmapheresis during the first 3 months of the trial.
  • At high risk for aspiration.
  • Pulmonary: forced vital capacity reduced to less than 70% of predicted capacity.
  • History of severe allergic disease or reactions likely to be exacerbated by any component of the vaccine.
  • History or evidence of Lambert-Eaton myasthenic syndrome, drug-induced myasthenia gravis, hereditary forms of myasthenic syndrome.
  • History of relevant chronic degenerative, psychiatric, or neurological disorder other than MG.
  • Severe hepatic, renal or cardiac insufficiency.
  • Major congenital defects or serious chronic illness other than MG.
  • Positive pregnancy test or desire to become pregnant during the study.
  • Female patients of child-bearing potential that do not use a reliable and highly effective method of contraception at least one month before first injection, during the study and until 3 months after the last injection.
  • Any significant out-of-range Clinical Laboratory results considered as clinically significant according to Investigator's judgment.
  • Previous completion or withdrawal from this study.
  • Sponsor employees or investigator site personnel directly affiliated with this study, and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biological or legally adopted.
  • Any medical condition that, in the opinion of the Investigator, might interfere with the subject's participation in the study, poses any added risk for the subject, or confounds the assessment of the subjects.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02609022


Locations
Belgium
University Hospital, Antwerp
Edegem, Antwerp, Belgium, 2650
Sponsors and Collaborators
CuraVac
Aepodia
University Hospital, Antwerp
Leiden University Medical Center
Investigators
Principal Investigator: Rudy Mercelis, MD, PhD University Hospital, Antwerp
More Information

Additional Information:
Publications:
Responsible Party: CuraVac
ClinicalTrials.gov Identifier: NCT02609022     History of Changes
Other Study ID Numbers: CV-0002
2015-002880-41 ( EudraCT Number )
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: November 20, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by CuraVac:
Myasthenia Gravis
CV-MG01 therapeutic vaccine
Acetylcholine receptor mimetic peptides
Safety
Immunogenic response
First-in-Human
Proof-of-Concept
Myasterix

Additional relevant MeSH terms:
Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Vaccines
Acetylcholine
Immunologic Factors
Physiological Effects of Drugs
Vasodilator Agents
Cholinergic Agonists
Cholinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action