To evaluate the efficacy, safety and tolerability of M281 injection for the treatment of patients with Myasthenia gravis, a neuromuscular disease
- Conditions
- Treatment of MG, with an initial focus on patients with gMG treated with M281 injection and evaluation of the expected reduction of circulating levels of antibodies by blocking IgG recycling, including the pathogenic autoantibodies that cause MG, and to ameliorate manifestations of the disease.MedDRA version: 21.1Level: PTClassification code 10028417Term: Myasthenia gravisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2018-002247-28-IT
- Lead Sponsor
- MOMENTA PHARMACEUTICALS, Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 68
1. Is a male or female outpatient =18 years of age.
2. Has a documented history of gMG and clinical signs/symptoms of gMG.
3. Has a documented diagnosis of gMG by a positive serologic test for a gMG-related pathogenic autoantibody (anti-AChR or anti-MuSK autoantibodies), confirmed at Screening, and is on stable therapy for MG. If the patient receives the first dose of M281 before the Screening results are available and the results are subsequently negative, the patient may be replaced.
4. Has a Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II, III, or IVa at Screening.
5. Has a QMG score of =12 at Screening and Baseline.
6. If taking a glucocorticosteroid, the patient must provide/obtain documentation showing the dose and regimen has been stable for at least 4 weeks prior to Screening.
7. If taking an acetylcholinesterase inhibitor, the patient must provide/obtain documentation showing the dose and regimen has been stable for at least 2 weeks prior to randomization on Day 1.
8. If taking statins at Screening, the patient must provide/obtain documentation showing the dose and regimen has been stable for at least 2 months prior to Screening.
9. If currently receiving immunosuppressants, the patient must provide/obtain documentation showing that the patient has been on the given immunosuppressant for =6 months and has been on a stable dose for =3 months prior to Screening. Allowed concomitant immunosuppressants are azathioprine, mycophenolate mofetil/mycophenolic acid, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide.
10. Has total serum IgG, serum albumin, and serum calcium concentrations within the normal range of the reference laboratory at Screening.
11. Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol.
12. Is up to date on all age-appropriate vaccinations as per routine local medical guidelines.
13. Women of childbearing potential, defined as women physiologically capable of becoming pregnant, must have a negative serum pregnancy test at the Screening visit and a negative urine pregnancy test at Baseline. Menopausal women must have an elevated serum folliclestimulating hormone level (FSH) at Screening; if the FSH is not elevated, they are considered to be of childbearing potential and must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline to be eligible.
14. Women of childbearing potential (including menopausal women who do not have elevated FSH) must agree to remain totally abstinent (ie. refrain from sexual intercourse during the study) or to consistently use a reliable and highly effective method of contraception (eg, condom plus diaphragm, condom plus spermicide, diaphragm plus spermicide, or intrauterine device or oral/injectable/implanted hormonal contraceptive used in combination with an additional barrier method) during the study and for 30 days after the last study treatment.
15. Male patients must agree to remain totally abstinent or to consistently use a reliable and highly effective method of contraception (eg, condom plus diaphragm, condom plus spermicide, diaphragm plus spermicide, or intrauterine device or oral/injectable/implanted hormonal contraceptive used in combination with an additional barrier method) to avoid pregnancy of the patient's partner(s) during the study and for 100 days following the last study treatment, unless the patient provides documentation of a v
1.Has MGFA Class I, IVb, or V disease, or presence of MG crisis (MGFA Class V) at Screening or Baseline, history of MG crisis within 1 month of the Screening visit, or fixed weakness (and/or 'burnt out' MG).
2.Has received rituximab or eculizumab within 12 months prior to Screening.
3.Has received plasmapheresis, immunoadsorption therapy, or IVIG within 6 weeks prior to randomization on Day 1.
4.Has a severe infection
5.Has a chronic infection
6.Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her gMG, including human immunodeficiency virus (HIV) infection, or has a family history of congenital or hereditary immunodeficiency.
7.Has a positive or equivocal QuantiFERON®-TB Gold test, and/or a known close exposure to family or individuals known to have tuberculosis.
8.Has had a splenectomy.
9.Received treatment for malignancy within the 5 years prior to Screening, with the exceptions of properly treated basal or squamous cell carcinoma of the skin or properly treated carcinoma in situ of the cervix.
10.Has a hypersensitivity to M281 or any constituent of the study drug solution.
11.Has had a prior severe drug reaction that included shock or severe hypersensitivity.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method