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

• ClinicalTrials.gov Identifier: NCT03772587
• Recruitment Status: Completed
• First Posted: December 11, 2018
• Last Update Posted: September 9, 2020
• Sponsor: Momenta Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Momenta Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: December 10, 2018
• First Posted Date: December 11, 2018
• Last Update Posted Date: September 9, 2020
• Actual Study Start Date: April 10, 2019
• Actual Primary Completion Date: May 6, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 10, 2018)

• Number of Participants With Adverse Events [ Time Frame: Up to Day 113 ]
• Change From Baseline in the Total Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score at Day 57 [ Time Frame: Baseline; Day 57 ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 10, 2018)

• Change From Baseline in Total MG-ADL Score as a Function of Total Serum Immunoglobulin G (IgG) at Day 57 [ Time Frame: Baseline; 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 [ Time Frame: Baseline; Day 57 ]
• Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or ≥8-point Improvement in Total MG-ADL Score [ Time Frame: Baseline to Day 57 ]
• Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score at Day 57 [ Time Frame: Baseline; Day 57 ]
• Change From Baseline in Total Revised Myasthenia Gravis Quality of Life - 15 Scale (MG-QoL15r) Score at Day 57 [ Time Frame: Baseline; Day 57 ]
• Shift From Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification at Day 57 [ Time Frame: Baseline; Day 57 ]
• Change From Baseline in Total Serum IgG at Day 57 [ Time Frame: Baseline; Day 57 ]
• Change in Total MG-ADL Score Over Time After the Last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]
• Change in Total QMG Score Over Time After the Last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]
• Change in Total MG-QoL15r Score Over time After the last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]
• Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or ≥8-point Improvement in Total QMG Score Over Time After the Last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]
• Shift in MGFA Classification Over Time After the Last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]
• Change in Total Serum IgG Over Time After the Last Dose [ Time Frame: Last dose (Day 57) to Day 113 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults With Generalized Myasthenia Gravis
• Official Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults With Generalized Myasthenia Gravis
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Generalized Myasthenia Gravis

Intervention

• Drug: M281
  M281 administered as IV infusion
• Other: Placebo
  Placebo administered as intravenous (IV) infusion

Study Arms

• Placebo Comparator: Group 1
  Intervention: Other: Placebo
• Experimental: Group 2
  Intervention: Drug: M281
• Experimental: Group 3
  Intervention: Drug: M281
• Experimental: Group 4
  Intervention: Drug: M281
• Experimental: Group 5
  Intervention: Drug: M281

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 7, 2020): 69
• Original Estimated Enrollment (submitted: December 10, 2018): 60
• Actual Study Completion Date: June 25, 2020
• Actual Primary Completion Date: May 6, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Canada, Germany, Italy, Poland, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT03772587
• Other Study ID Numbers: MOM-M281-004; 2018-002247-28 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Momenta Pharmaceuticals, Inc.
• Study Sponsor: Momenta Pharmaceuticals, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Momenta General Queries; Momenta Pharmaceuticals, Inc.

• PRS Account: Momenta Pharmaceuticals, Inc.
• Verification Date: September 2020