Myasthenia Gravis, GeneralizedPhase 2/3NCT03165435
A Study to Evaluate the Efficacy of CV-MG01 (Myasterix) in Myasthenia Gravis
A Phase 2/3 study testing CV-MG01 in people with Myasthenia Gravis, Generalized, run by CuraVac at 1 site in Belgium.
Trial summary
Study CV-0003 will be the second clinical trial administering CV-MG01 in humans. This will be a phase 2/3 proof-of-efficacy therapeutic confirmatory study following the proof-of-concept exploratory phase 1 study (CV-0002). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- CV-MG01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteBelgium
- Study dates
- Jul 2018to 31 Dec 2019
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female Patient, with generalised myasthenia gravis (Grades 2, 3 and 4a) as per myasthenia gravis foundation of America (MGFA) classification system.
- Quantitative Myasthenia Gravis (QMG) score of 10 or greater at screening and baseline.
- Age of minimum 18, at the time of the consent form signature.
- Patient with documented positive antibodies to AChR in one of the available validated laboratory test.
- Patient may use corticosteroid treatment initiated for at least 3 months before screening, equivalent to a daily dose of 30mg prednisone as maximum, and stable (+/- 5mg change) at least 1 month before the screening and up to the first injection.
- Patient may use one or two immunosuppressive drugs (initiated for a least 6 months) with or without concomitant use of corticosteroid, providing that the dosage has been stable/unchanged for 3 months before the screening and up to the first injection.
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- 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.
Who cannot join
- MG patients of Grade 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.
- 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 drug, or a planned administration of immunoglobulins during the first 3 months of the study.
- History or evidence of rituximab treatment within 6 months prior to first dose of study.
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- History or evidence of plasmapheresis within 3 months prior to the first dose of study, or a planned plasmapheresis during the first 3 months of the study.
- 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 or uncontrolled hypertension
- 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 (see Section 7.2 Contraception) at least one month before first injection, during the study and until the end of Part A.
- Any significant out-of-range Clinical Laboratory results considered as clinically significant and that prevent Subject's participation into the study according to Investigator's judgment.
- Previous completion or withdrawal from this study or study CV-0002.
- 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 or concomitant medication 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multi-centre, Randomised, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy of Subcutaneous Injections of the Active Targeted Immunotherapy CV-MG01 in Patients With Moderate to Severe Myasthenia Gravis.
Study CV-0003 will be the second clinical trial administering CV-MG01 in humans. This will be a phase 2/3 proof-of-efficacy therapeutic confirmatory study following the proof-of-concept exploratory phase 1 study (CV-0002).
Read the detailed description
The review of the clinical data accumulated so far in Study CV-0002 indicated that the safety and tolerability profile of CV-MG01 is considered very good, with positive immunogenicity results after the high dose regimen of CV-MG01. The preliminary results also show an indication of efficacy of CV-MG01, as based on the different scales and questionnaires results, all specific to MG disease. Therefore, it is proposed to investigate, in an appropriately designed, randomised, double-blind and powered study, the clinical efficacy of CV-MG01 in a larger cohort of MG patients.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- CV-MG01, Placebo
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CV-MG01Biological
Also called Myasterix
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.
PlaceboBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| CV-MG01 | Experimental | The active targeted immunotherapy candidate, CV-MG01 comprises two short synthetic peptides separately conjugated to a carrier protein for the potential treatment of myasthenia gravis Receives: Biological: CV-MG01 |
| Placebo | Placebo comparator | Aluminium hydroxide adjuvant alone Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical efficacy
To assess the efficacy of 3 subcutaneous injections of CV-MG01 compared to placebo, as measured by a decrease from baseline of the QMG total score at 24 weeks after the first injection (equivalent to 12 weeks after last injection).
Measured: 24 weeks
Secondary outcomes (2)
Safety (ohysical examens and laboratory tests) and local tolerance (FDA grading scale)
Evaluation of treatment emergent adverse events (TEAEs) including local injection site reactions (severity assessed with an overall grading scale following the FDA recommendation (FDA, CBER, September 2007)).
General safety monitoring via physical examinations, vital signs (VS), ECG and standard laboratory tests (clinical chemistry, haematology and urinalysis).
Measured: 24 weeks
Efficacy - Responder rate
Proportion of patients with improvement or worsening by ≥ 3 points in the QMG score at week 24 after the first injection in the active group compared to the placebo group
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2017
- Study startJul 2018 (estimated)
- Primary completion1 Oct 2019 (estimated)
- Study completion31 Dec 2019 (estimated)
Why it stopped: Not enough participating centres to complete recruitment in a timely manner.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia Gravis, GeneralizedStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- CuraVac
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aepodia, University Hospital, Antwerp
- Responsible party
- Sponsor
- Investigators
- Rudy Mercelis, MD, PhD, Principal investigator, University Hospital, Antwerp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03165435
- Sponsor's protocol ID
- CV-0003
- Other IDs
- 2017-000323-27, 2017
- Status last verified
- May 2018
- First submitted
- 17 May 2017
- Last updated
- 30 May 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-centre, Randomised, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy of Subcutaneous Injections of the Active Targeted Immunotherapy CV-MG01 in Patients With Moderate to Severe Myasthenia Gravis.
- ClinicalTrials.gov ID
- NCT03165435
- Acronym / sponsor ID
- CV-0003
- Conditions
- Myasthenia Gravis, Generalized
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Not enough participating centres to complete recruitment in a timely manner.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- CuraVacIndustry (pharma, biotech, devices)
- Collaborators
- Aepodia, University Hospital, Antwerp
- Study start
- Jul 2018 (estimated)
- Primary completion
- 1 Oct 2019 (estimated)
- Study completion
- 31 Dec 2019 (estimated)
- First posted
- 24 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study CV-0003 will be the second clinical trial administering CV-MG01 in humans. This will be a phase 2/3 proof-of-efficacy therapeutic confirmatory study following the proof-of-concept exploratory phase 1 study (CV-0002).
Detailed description
The review of the clinical data accumulated so far in Study CV-0002 indicated that the safety and tolerability profile of CV-MG01 is considered very good, with positive immunogenicity results after the high dose regimen of CV-MG01. The preliminary results also show an indication of efficacy of CV-MG01, as based on the different scales and questionnaires results, all specific to MG disease. Therefore, it is proposed to investigate, in an appropriately designed, randomised, double-blind and powered study, the clinical efficacy of CV-MG01 in a larger cohort of MG patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CV-MG01 | Experimental | Biological: CV-MG01The active targeted immunotherapy candidate, CV-MG01 comprises two short synthetic peptides separately conjugated to a carrier protein for the potential treatment of myasthenia gravis |
| Placebo | Placebo comparator | Biological: PlaceboAluminium hydroxide adjuvant alone |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female Patient, with generalised myasthenia gravis (Grades 2, 3 and 4a) as per myasthenia gravis foundation of America (MGFA) classification system.
- Quantitative Myasthenia Gravis (QMG) score of 10 or greater at screening and baseline.
- Age of minimum 18, at the time of the consent form signature.
- Patient with documented positive antibodies to AChR in one of the available validated laboratory test.
- Patient may use corticosteroid treatment initiated for at least 3 months before screening, equivalent to a daily dose of 30mg prednisone as maximum, and stable (+/- 5mg change) at least 1 month before the screening and up to the first injection.
Key exclusion criteria (20 listed)
- MG patients of Grade 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.
- 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 drug, or a planned administration of immunoglobulins during the first 3 months of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CV-MG01Also: Myasterix3 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. | Biological | CV-MG01 |
| Placebo3 consecutive subcutaneous injections of placebo. The three injections are planned for each patient on Days 1, 29 (+/- 3 days) and 85 (+/- 7 days), respectively. | Biological | Placebo |
Primary outcome
Clinical efficacy
Time frame: 24 weeks
To assess the efficacy of 3 subcutaneous injections of CV-MG01 compared to placebo, as measured by a decrease from baseline of the QMG total score at 24 weeks after the first injection (equivalent to 12 weeks after last injection).
2 secondary outcomes
Safety (ohysical examens and laboratory tests) and local tolerance (FDA grading scale)
Time frame: 24 weeks
Efficacy - Responder rate
Time frame: 24 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2018.
- Therapeutic Vaccines / Active Targeted Immunotherapies for Autoimmune DiseasesLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rudy Mercelis, MD, PhD principal investigator
University Hospital, Antwerp
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing CV-MG01 in people with Myasthenia Gravis, Generalized, run by CuraVac at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 30 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical efficacy (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2018.