A Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults With Generalized Myasthenia Gravis

NCT03772587 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: MOM-M281-0042018-002247-28
• Study Type: interventional
• Target: 68
• Locations: 8 countries
• Sites: 61

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of M281 administered to participants with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing standard of care therapy.

Conditions

Generalized Myasthenia Gravis

Keywords

M281; Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) as a Measure of Safety and Tolerability

  An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAE are defined as any AE occurring during or after the initiation of the first infusion of study drug.

  Up to Day 113

• Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)

  An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. An SAE is defined as any AE occurring at any dose that results in any of the following outcomes: death, life-threatening AE, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect.

  Up to Day 113

• Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI)

  An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. For this study, any common terminology criteria for adverse events (CTCAE) Grade 3 or higher event of severe infection or hypoalbuminemia was considered as AESI.

  Up to Day 113

• Change From Baseline to Day 57 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score

  The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.

  Baseline to Day 57

Secondary Outcomes (16)

• Change From Baseline in Total MG-ADL Score as a Function of Total Serum Immunoglobulin G (IgG) at Day 57

  Baseline and Day 57

• Change From Baseline in Total MG-ADL Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57

  Baseline and Day 57

• Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score as a Function of Total Serum IgG at Day 57

  Baseline and Day 57

• Change From Baseline in Total QMG Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57

  Baseline and Day 57

• Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or Greater Than or Equal to (>=) 8-point Improvement in Total MG-ADL Score at Day 57

  Day 57

Study Arms (5)

Group 1

PLACEBO COMPARATOR

Other: Placebo

Group 2

EXPERIMENTAL

Drug: M281

Group 3

EXPERIMENTAL

Drug: M281

Group 4

EXPERIMENTAL

Drug: M281

Group 5

EXPERIMENTAL

Drug: M281

Interventions

M281 DRUG

M281 administered as IV infusion

Group 2; Group 3; Group 4; Group 5

Placebo OTHER

Placebo administered as intravenous (IV) infusion

Group 1

Eligibility Criteria

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

Participants must be ≥18 years of age with a documented history of Generalized Myasthenia Gravis (gMG) and clinical signs/symptoms of gMG, not pregnant or breastfeeding, and no history of any neurologic disorder other than MG that might interfere with the accuracy of study assessments. Additional, more specific criteria are defined in the protocol.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Momenta Pharmaceuticals, Inc.: lead

Study Sites (61)

Momenta Investigational Site

Phoenix, Arizona, 85013, United States

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Momenta Investigational Site

Tucson, Arizona, 85724, United States

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Los Angeles, California, 90048, United States

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Momenta Investigational Site

Orange, California, 92868, United States

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Stanford, California, 94305, United States

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Momenta Investigational Site

Aurora, Colorado, 80045, United States

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Momenta Investigational Site

New Haven, Connecticut, 06511, United States

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Momenta Investigational Site

Boca Raton, Florida, 33487, United States

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Momenta Investigational Site

Maitland, Florida, 32751, United States

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Momenta Investigational Site

Port Charlotte, Florida, 33952, United States

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Momenta Investigational Site

St. Petersburg, Florida, 33713, United States

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Augusta, Georgia, 30912, United States

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Meridian, Idaho, 83642, United States

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Lake Barrington, Illinois, 60010, United States

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Fairway, Kansas, 66205, United States

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Boston, Massachusetts, 02114, United States

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Boston, Massachusetts, 02115, United States

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Boston, Massachusetts, 02215, United States

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Detroit, Michigan, 48202, United States

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East Lansing, Michigan, 48824, United States

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Columbia, Missouri, 65212, United States

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New Brunswick, New Jersey, 08550, United States

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New York, New York, 10021, United States

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Charlotte, North Carolina, 28207, United States

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Durham, North Carolina, 27710, United States

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Raleigh, North Carolina, 27607, United States

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Cincinnati, Ohio, 45267, United States

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Columbus, Ohio, 43210, United States

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Cordova, Tennessee, 38106, United States

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Austin, Texas, 78756, United States

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Round Rock, Texas, 78681, United States

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Leuven, Vlaams Brabant, 3000, Belgium

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Antwerp, 2650, Belgium

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Brussels, 1070, Belgium

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Edmonton, Alberta, T6G 2G3, Canada

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London, Ontario, N6A 5A5, Canada

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Toronto, Ontario, M5G 2C4, Canada

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Québec, Quebec, G1J 1Z4, Canada

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Momenta Investigational Site

Göttingen, Lower Saxony, 37075, Germany

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Düsseldorf, 40225, Germany

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Momenta Investigational Site

Gummersbach, 51643, Germany

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Momenta Investigational Site

Münster, 48149, Germany

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Momenta Investigational Site

Cefalù, 90015, Italy

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Momenta Investigational Site

Messina, 98125, Italy

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Momenta Investigational Site

Milan, 20133, Italy

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Momenta Investigational Site

Krakow, 31-505, Poland

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Lodz, 90-324, Poland

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Momenta Investigational Site

Warsaw, 01-684, Poland

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Momenta Investigational Site

Warsaw, 01-868, Poland

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Momenta Investigational Site

Barcelona, Catalan, 08035, Spain

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Momenta Investigational Site

Barcelona, Catalan, 08041, Spain

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Momenta Investigational Site

Badalona, Catalonia, 08036, Spain

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Momenta Investigational Site

L'Hospitalet de Llobregat, Catalonia, 08907, Spain

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Momenta Investigational Site

Donostia / San Sebastian, Gipuzkoa, 20014, Spain

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Momenta Investigational Site

Madrid, 28007, Spain

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Momenta Investigational Site

Madrid, 28040, Spain

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Momenta Investigational Site

Seville, 41013, Spain

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Momenta Investigational Site

Valencia, 46026, Spain

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Momenta Investigational Site

Birmingham, B15 2TH, United Kingdom

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Momenta Investigational Site

Sheffield, S11 9NE, United Kingdom

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Momenta Investigational Site

Southampton, SO16 6YD, United Kingdom

Location

Related Publications (1)

• Antozzi C, Guptill J, Bril V, Gamez J, Meuth SG, Nowak RJ, Quan D, Sevilla T, Jouvin MH, Jin J, Karcher K, Ramchandren S, Sun H, Ling L, Zhu Y, Arroyo S; Vivacity-MG Phase 2 Study Group. Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study. Neurology. 2024 Jan 23;102(2):e207937. doi: 10.1212/WNL.0000000000207937. Epub 2023 Dec 21.

  PMID: 38165333 DERIVED

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

Limitations and Caveats

Limitations to this study include the small sample sizes of each treatment arm and the study activity disruption due to the COVID-19 pandemic, especially the missed Quantitative Myasthenia Gravis (QMG) assessments which hampered the analysis of the endpoint.

Results Point of Contact

• Title: DIRECTOR CLINICAL RESEARCH
• Organization: Momenta Pharmaceuticals, Inc.

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Momenta General Queries

  Momenta Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: December 10, 2018
• First Posted: December 11, 2018
• Study Start: April 10, 2019
• Primary Completion: June 25, 2020
• Study Completion: June 25, 2020
• Last Updated: June 27, 2023
• Results First Posted: October 27, 2021

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

ES (9); CA (4); PL (4); IT (3); US (31); DE (4); BE (3); GB (3)