Myasthenia Gravis Inebilizumab Trial

Phase 3

Active, not recruiting

• Conditions: Myasthenia Gravis
• Interventions: Drug: IV Placebo; Drug: inebilizumab

• Registration Number: NCT04524273
• Lead Sponsor: Amgen

Brief Summary

Randomized, double-blind, placebo-controlled, Phase 3, parallel-group study with optional open-label extension.

Detailed Description

This study is a phase 3, randomized, double-blind, placebo-controlled study, to be conducted at approximately 120 study sites. Approximately 230 participants (188 acetylcholine receptor antibody positive \[AChR-Ab+\] and 42 muscle-specific tyrosine kinase antibody positive \[MuSK-Ab+\]) will be enrolled. Participants with Myasthenia Gravis (MG) who are positive for anti-AChR or anti-MuSK antibodies will be enrolled and analyzed. Patients who do not have anti-AChR or anti-MuSK antibodies will not be enrolled. Patients with Myasthenia Gravis Foundation of America (MGFA) classification II, III, or IV disease, Myasthenia Gravis Activities of Daily Living (MG-ADL) score at screening and randomization between 6 and 10 with \> 50% of this score attributed to non-ocular items, or an MG-ADL score \>=11, Quantitative Myasthenia Gravis (QMG) score \>= 11 at the time of screening and randomization, and use of a corticosteroid and/or non-steroidal immunosuppressant will be included in the study.

All subjects who complete the randomized controlled period (RCP) will have the option to enroll in a 3-year (156 weeks) open-label period.

Study acquired from Horizon in 2023.

Study Timeline

• Study First Submitted: 2020-08-10
• Study First Submitted QC: 2020-08-19
• Study First Posted: 2020-08-24
• Study Start: 2020-10-15
• Primary Completion: 2024-05-28
• Status Verified: 2025-02
• Disposition First Submitted: 2025-02-14
• Last Update Submitted: 2025-02-14
• Disposition First Posted: 2025-02-17
• Last Update Posted: 2025-02-20
• Study Completion: 2027-11-29

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 238

Inclusion Criteria

1. Diagnosis of MG with anti-AChR or anti-MuSK antibody.

2. MGFA Clinical Classification Class II, III, or IV.

3. MG-ADL score of 6 or greater at screening and at randomization with > 50% of this score attributed to non-ocular items.

4. QMG score of 11 or greater.

5. Participants must be on:

   1. Corticosteroids only, with no dose increase within 4 weeks prior to randomization, or
   2. One allowed non-steroidal immunosuppressive therapy (IST), with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization, or
   3. Combination of (1) corticosteroids with no dose increase within 4 weeks prior to randomization and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.

Allowed ISTs, alone or in combination with corticosteroids, are azathioprine, mycophenolate mofetil, and mycophenolic acid.

Exclusion Criteria

1. Receipt of the following medications within the 4 weeks prior to Day 1:

   1. Cyclosporine (except eye drops)
   2. Tacrolimus (except topical)
   3. Methotrexate

2. Current use of:

   1. Corticosteroids (Prednisone > 40 milligram (mg)/day or > 80 mg over a 2-day period (or equivalent dose of other corticosteroids).
   2. Acetylcholinesterase inhibitors (pyridostigmine) > 480 mg/day) or unstable dose in the 2 weeks prior to Day 1.
   3. Azathioprine > 3 mg/kilogram (kg)/day
   4. Mycophenolate mofetil > 3 grams/day or mycophenolic acid > 1440 mg/day
   5. Any IST, alone or in combination with corticosteroids, except for azathioprine, mycophenolate mofetil, and mycophenolic acid.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo, (MuSK-Ab+) MG	IV Placebo	Participants will receive placebo administered IV on Days 1 and 15 of the RCP. Participants who elect to enter the OLP will receive inebilizumab administered IV on OLP Days 1,15, 183, 365, 547, 729, and 911.
Placebo, (AChR-Ab+) MG	IV Placebo	Participants will receive placebo administered IV on Days 1, 15, and 183 of the RCP. Participants who elect to enter the OLP will receive inebilizumab administered IV on OLP Days 1,15, 183, 365, 547, 729, and 911.
Inebilizumab, (AChR-Ab+) MG	inebilizumab	Participants will receive inebilizumab administered intravenously (IV) on Days 1, 15, and 183 of the RCP. Participants who elect to enter the open label phase (OLP) will receive inebilizumab administered IV on OLP Days 1, IV placebo on OLP Day 15 (to avoid potential unblinding), and inebilizumab IV on OLP Days 183, 365, 547, 729, and 911.
Inebilizumab, (MuSK-Ab+) MG	inebilizumab	Participants will receive inebilizumab administered IV on Days 1 and 15 of the RCP. Participants who elect to enter the OLP will receive inebilizumab administered IV on OLP Day 1, IV placebo on OLP Day 15 (to avoid potential unblinding), and inebilizumab IV on OLP Days 183, 365, 547, 729, and 911.

Primary Outcome Measures

Name	Time	Method
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score.	Baseline to Week 26

Secondary Outcome Measures

Name	Time	Method
Change from baseline in Quantitative Myasthenia Gravis (QMG) scores.	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Proportion of participants with both ≥ 3-point improvement from baseline in MG-ADL and did not initiate rescue therapy.	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Change from baseline in MG-ADL	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Time to first gMG exacerbation.	Day 1 to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Change from baseline in Myasthenia Gravis Composite (MGC) score.	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Change from baseline in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score.	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Patient Global Impression of Change (PGIC) score.	Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Number of participants with treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and treatment-emergent serious adverse events (TESAEs) during the RCP and OLP.	Up to 208 weeks
Proportion of participants with steroid tapered to ≤ 5 mg/day.	Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Proportion of participants with a steroid dose reduction from baseline of ≥ 50%.	Baseline to Week 52 for AChR-Ab+ population and Week 26 for the overall study population
Proportion of participants achieving minimal symptom expression.	Week 26	Minimal symptom expression was defined as an MG-ADL of 0 or 1.
Proportion of participants with anti-drug antibodies (ADAs) to inebilizumab	Up to 208 weeks
Titers of ADAs	Up to 208 weeks

Trial Locations

Locations (104)

Viela Bio Investigative Site - 1015; 🇺🇸 Orange, California, United States

Viela Bio Investigative Site - 1002; 🇺🇸 New Haven, Connecticut, United States

Viela Bio Investigative Center - 1024; 🇺🇸 Washington, District of Columbia, United States

Viela Bio Investigative Site - 1005; 🇺🇸 Tampa, Florida, United States

Viela Bio Investigative Site - 1012; 🇺🇸 Kansas City, Kansas, United States

Viela Bio Investigative Site - 1018; 🇺🇸 Charlotte, North Carolina, United States

Viela Bio Investigative Site - 1025; 🇺🇸 Canton, Ohio, United States

Viela Bio Investigative Site - 1001; 🇺🇸 Cincinnati, Ohio, United States

Viela Bio Investigative Site - 1009; 🇺🇸 Columbus, Ohio, United States

Viela Bio Investigative Site - 1008; 🇺🇸 Pittsburgh, Pennsylvania, United States

Viela Bio Investigative Site - 1019; 🇺🇸 Austin, Texas, United States

Viela Bio Investigative Site - 1003; 🇺🇸 Houston, Texas, United States

Viela Bio Investigative Site - 1014; 🇺🇸 San Antonio, Texas, United States

Viela Bio Investigative Site - 1017; 🇺🇸 Salt Lake City, Utah, United States

Viela Bio Investigative Site - 1004; 🇺🇸 Burlington, Vermont, United States

Viela Bio Investigative Site - 1006; 🇺🇸 Richmond, Virginia, United States

Viela Bio Investigative Site - 2001; 🇦🇷 Buenos Aires, Argentina

VielaBio Investigative Site - 2002; 🇦🇷 Buenos Aires, Argentina

Viela Bio Investigative Site - 2003; 🇦🇷 Buenos Aires, Argentina

Viela Bio Investigative Site - 5203; 🇧🇾 Grodno, Belarus

Viela Bio Investigative Site - 5204; 🇧🇾 Minsk, Belarus

Viela Bio Investigative Site - 5201; 🇧🇾 Vitebsk, Belarus

VielaBio Investigative Site - 2201; 🇧🇷 Porto Alegre, Brazil

VielaBio Investigative Site - 2203; 🇧🇷 Porto Alegre, Brazil

VielaBio Investigative Site - 2207; 🇧🇷 Ribeirão Preto, Brazil

VielaBio Investigative Site - 2206; 🇧🇷 San Paolo, Brazil

Viela Bio Investigative Site - 2205; 🇧🇷 São Paulo, Brazil

Viela Bio Investigative Site - 1102; 🇨🇦 Toronto, Ontario, Canada

Viela Bio Investigative Site - 1101; 🇨🇦 Montréal, Quebec, Canada

Viela Bio Investigative Site - 1103; 🇨🇦 Montréal, Quebec, Canada

Viela Bio Investigative Site 4009; 🇨🇳 Beijing, China

Viela Bio Investigative Site - 4007; 🇨🇳 Beijing, China

Viela Bio Investigative Site 4003; 🇨🇳 Beijing, China

Viela Bio Investigative Site - 4005; 🇨🇳 Guiyang, China

Viela Bio Investigative site 4010; 🇨🇳 Hunan, China

Viela Bio Investigative Site - 4011; 🇨🇳 Jinan, China

Viela Bio Investigative Site - 4006; 🇨🇳 Shanghai, China

Viela Bio Investigative Site - 4008; 🇨🇳 Suzhou, China

Viela Bio Investigative Site - 4004; 🇨🇳 Tianjin, China

Viela Bio Investigative Site 4012; 🇨🇳 Xi'an, China

Viela Bio Investigative Site - 5601; 🇩🇰 Copenhagen, Denmark

Viela Bio Investigative Site - 3003; 🇫🇷 Lille Cedex, France

Viela Bio Investigative Site - 3001; 🇫🇷 Nice Cedex 01, France

Viela Bio Investigative Site - 3002; 🇫🇷 Strasbourg, France

VielaBio Investigative Site - 3101; 🇩🇪 Essen, Germany

Viela Bio Investigative Site - 4107; 🇮🇳 Nagpur, Maharashtra, India

Viela Bio Investigative Site - 4113; 🇮🇳 Nagpur, Maharashtra, India

Viela Bio Investigative Site - 4112; 🇮🇳 Hyderabad, Telangana, India

Viela Bio Investigative Site - 4104; 🇮🇳 Bangalore, India

Viela Bio investigative Site - 4105; 🇮🇳 Lucknow, India

Viela Bio Investigative Site - 4103; 🇮🇳 Manipala, India

Viela Bio Investigative Site - 4102; 🇮🇳 Nashik, India

Viela Bio Investigative Site - 4109; 🇮🇳 Pune, India

Viela Bio Investigative Site - 4101; 🇮🇳 Surat, India

Viela Bio Investigative Site - 3204; 🇮🇹 Milano, Italy

Viela Bio Investigative Site - 3203; 🇮🇹 Milano, Italy

Viela Bio Investigative Site - 3201; 🇮🇹 Pavia, Italy

Viela Bio Investigative Site - 3202; 🇮🇹 Rome, Italy

Viela Bio Investigative Site - 4402; 🇯🇵 Chiba, Japan

Viela Bio Investigative Site - 4410; 🇯🇵 Chiba, Japan

Viela Bio Investigative Site 4409; 🇯🇵 Fukuoka, Japan

Viela Bio Investigative Site - 4401; 🇯🇵 Hanamaki, Japan

VielaBio Investigative Site - 4408; 🇯🇵 Hokkaido, Japan

Viela Bio Investigative Site - 4406; 🇯🇵 Hyōgo, Japan

Viela Bio Investigative Site 4405; 🇯🇵 Miyagi, Japan

Viela Bio Investigative Site - 4413; 🇯🇵 Moriguchi, Japan

VielaBio Investigative Site - 4407; 🇯🇵 Morioka-shi, Japan

Viela Bio Investigative Site - 4404; 🇯🇵 Saitama, Japan

Viela Bio Investigative Site - 4202; 🇰🇷 Seoul, Korea, Republic of

Viela Bio Investigative Site - 4203; 🇰🇷 Seoul, Korea, Republic of

Viela Bio Investigative Site - 4201; 🇰🇷 Seoul, Korea, Republic of

Viela Bio Investigative Site - 3302; 🇵🇱 Katowice, Poland

Viela Bio Investigative Site - 3303; 🇵🇱 Katowice, Poland

Viela Bio Investigative Site - 3301; 🇵🇱 Kraków, Poland

Med Polonia Sp. z o.o - 3311; 🇵🇱 Poznań, Poland

Viela Bio Investigative Site - 3310; 🇵🇱 Warszawa, Poland

Viela Bio Investigative Site - 5313; 🇷🇺 Barnaul, Altai Krai, Russian Federation

Viela Bio Investigative Site - 5303; 🇷🇺 Barnaul, Russian Federation

Viela Bio Investigative Site - 5302; 🇷🇺 Nizhny Novgorod, Russian Federation

Viela Bio Investigative Site - 5304; 🇷🇺 Novosibirsk, Russian Federation

Viela Bio Investigative Site - 5311; 🇷🇺 Rostov-on-Don, Russian Federation

Viela Bio Investigative Site - 5309; 🇷🇺 Rostov-on-Don, Russian Federation

Viela Bio Investigative Site - 5308; 🇷🇺 Saint Petersburg, Russian Federation

Viela Bio Investigative Site - 3403; 🇪🇸 Badalona, Spain

Viela Bio Investigative Site - 5305; 🇷🇺 Samara, Russian Federation

Viela Bio Investigative Site - 5312; 🇷🇺 Ufa, Russian Federation

Viela Bio Investigative Site - 3402; 🇪🇸 Barcelona, Spain

Viela Bio Investigative Site - 3404; 🇪🇸 Córdoba, Spain

Viela Bio investigative Site - 4605; 🇨🇳 Kaohsiung City, Taiwan

Viela Bio Investigative Site 4608; 🇨🇳 New Taipei City, Taiwan

Viela Bio Investigative Site - 4606; 🇨🇳 Tainan, Taiwan

Viela Bio Investigative Site - 4604; 🇨🇳 Taipei, Taiwan

Viela Bio Investigative Site -4607; 🇨🇳 Taipei, Taiwan

Viela Bio Investigative Site - 4602; 🇨🇳 Taoyuan City, Taiwan

Viela Bio Investigative Site - 4603; 🇨🇳 Taipei, Taiwan

Viela Bio Investigative Site - 3903; 🇹🇷 Ankara, Turkey

Viela Bio Investigative Site - 3901; 🇹🇷 İzmir, Turkey

Viela Bio Investigative Site - 3902; 🇹🇷 İzmir, Turkey

Viela Bio Investigative Site - 3905; 🇹🇷 Kocaeli, Turkey

Viela Bio Investigative Site - 5103; 🇺🇦 Dnipro, Ukraine

Viela Bio Investigative Site - 5108; 🇺🇦 Dnipro, Ukraine

Viela Bio Investigative Site - 5105; 🇺🇦 Ivano-Frankivs'k, Ukraine

Viela Bio Investigative Site - 5104; 🇺🇦 Lutsk, Ukraine

Viela Bio Investigative Site - 5106; 🇺🇦 Vinnytsia, Ukraine