A Study to Evaluate the Efficacy of CV-MG01 (Myasterix) in Myasthenia Gravis

NCT03165435 | Withdrawn Phase 2 DMC

• 2 other identifiers: CV-00032017-000323-27
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Myasthenia Gravis, Generalized

Outcome Measures

Primary Outcomes (1)

• 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).

  24 weeks

Secondary Outcomes (2)

• Safety (ohysical examens and laboratory tests) and local tolerance (FDA grading scale)

  24 weeks

• Efficacy - Responder rate

  24 weeks

Study Arms (2)

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

Biological: CV-MG01

Placebo

PLACEBO COMPARATOR

Aluminium hydroxide adjuvant alone

Biological: Placebo

Interventions

CV-MG01 BIOLOGICAL

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.

Also known as: Myasterix

CV-MG01

Placebo BIOLOGICAL

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.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• CuraVac: lead
• Aepodia: collaborator
• University Hospital, Antwerp: collaborator

Study Sites (1)

University Hospital, Antwerp

Edegem, Antwerp, 2650, Belgium

Location

Related Links

• Therapeutic Vaccines / Active Targeted Immunotherapies for Autoimmune Diseases

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Rudy Mercelis, MD, PhD

  University Hospital, Antwerp

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 17, 2017
• First Posted: May 24, 2017
• Study Start: July 1, 2018
• Primary Completion: October 1, 2019
• Study Completion: December 31, 2019
• Last Updated: May 30, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)