A Study of TAK-079 in People With Generalized Myasthenia Gravis

NCT04159805 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: TAK-079-10052019-003383-47
• Study Type: interventional
• Target: 36
• Locations: 6 countries
• Sites: 50

Brief Summary

Myasthenia gravis is an autoimmune condition that causes muscle weakness. Autoimmune means the body makes antibodies that attack its own cells and tissues. These types of antibodies are also known as autoantibodies. People with generalized myasthenia gravis have a weakness in many muscles. TAK-079 is a medicine to help people with generalized myasthenia gravis. The main aim of this study is to check if people with generalized myasthenia gravis have side effects from 2 doses of TAK-079. Other aims are to learn if TAK-079 improves their clinical condition and lowers their autoantibody levels. At the first visit, the study doctor will check if each person can take part. For those who can take part, participants will continue with their standard medicines for this condition during the study. Each participant will have a check-up by the study doctor. Then, the participants will have 1 of 3 treatments:

• A low dose of TAK-079.
• A high dose of TAK-079.
• A placebo. In this study, a placebo looks like TAK-079 but does not have any medicine in it. Participants will not know which treatment they received, nor will their study doctors. This is to help make sure the results are more reliable. For each treatment, participants will receive injections just under the skin, once a week for 8 weeks. The study doctors will check for side effects from the study treatments. The study doctors can stop or delay the injections in each participant if needed. Then, the study doctors will continue to check for side effects for up to 24 weeks after treatment. They will also check the clinical condition of the participants, including their autoantibody levels.

Conditions

Myasthenia Gravis

Keywords

Drug therapy

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

  AE is defined as any untoward medical occurrence in clinical investigation participant administered drug; it does not necessarily have to have causal relationship with this treatment. TEAE is defined as AE with onset that occurs after receiving study drug. SAE is an adverse event resulting in any of following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Severity of TEAEs was graded using National cancer institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 definitions of Grade 1 through Grade 5 wherein Grade 1=mild symptoms, Grade 2=moderate symptoms, Grade 3=Severe or medically significant but not immediately life-threatening, Grade 4=life-threatening consequences and Grade 5=death related to AEs. Percentages are rounded off to whole number at single decimal.

  From signing the informed consent form up to end of long-term follow-up (up to Week 32)

Secondary Outcomes (9)

• Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

  Baseline up to Week 32

• Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

  Baseline up to Week 32

• Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

  Baseline up to Week 32

• Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

  Baseline up to Week 32

• Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

  Baseline up to Week 32

Study Arms (3)

TAK-079 Placebo-matching

PLACEBO COMPARATOR

TAK-079 placebo-matching injection, subcutaneously (SC), once weekly in combination with standard background therapy for 8 weeks.

Drug: TAK-079 Placebo

TAK-079 300 mg

EXPERIMENTAL

TAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Drug: TAK-079

TAK-079 600 mg

EXPERIMENTAL

TAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Drug: TAK-079

Interventions

TAK-079 DRUG

TAK-079 subcutaneous injection

Also known as: Mezagitamab

TAK-079 300 mg; TAK-079 600 mg

TAK-079 Placebo DRUG

TAK-079 placebo-matching subcutaneous injection

TAK-079 Placebo-matching

Eligibility Criteria

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

You may qualify if:

• Diagnosis of Myasthenia Gravis (MG) supported by a positive serologic test for anti-AChR or anti-MuSK antibodies at screening.
• Myasthenia Gravis Foundation of America (MGFA) clinical classification II to IV at screening.
• Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of 6 or greater at screening, with at least 4 points attributed to nonocular items.
• If receiving immunosuppressive drugs (ie, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, cyclophosphamide), therapy must be ongoing for at least 6 months, with stable dosing ongoing for at least 3 months before screening. Participants receiving azathioprine must be on a stable dose for at least 6 months before screening.
• If receiving oral corticosteroids, therapy must be ongoing for at least 3 months, with a stable dose at least 1 month before screening. Corticosteroids, including dexamethasone, must be given as oral, daily or every-other-day therapy, as opposed to pulse therapy.
• If receiving cholinesterase inhibitors, therapy with a stable dose is required at least 2 weeks before screening.
• The doses of concomitant standard background therapy must be expected to remain stable throughout the study unless dose reduction is required due to toxicities. Allowed background therapy is defined as no more than a cholinesterase inhibitor ± corticosteroid ± 1 steroid-sparing immunosuppressive drug (limited to azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide). Participants must be on at least one allowed background medication.

You may not qualify if:

• Presence of a thymoma (previous history of a fully encapsulated thymoma removed ≥ 12 months before screening is allowed) or history of invasive thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening.
• History of thymectomy within 12 months before screening.
• MGFA class I or V.
• Received intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg), or plasmapheresis/plasma exchange within 4 weeks before screening, or an expectation that any therapy besides the participants standard background therapies may be used for treatment of MG (eg, a rescue therapy) between screening and dosing.
• Chronic obstructive pulmonary disease (COPD) or asthma with a pre-bronchodilatory forced expiratory volume in 1 second (FEV1) \<50% of predicted normal.
• Note: FEV1 testing is required for participants suspected of having COPD or asthma.
• Received rituximab, belimumab, eculizumab, or any monoclonal antibody for immunomodulation within 6 months before first dosing. Participants with prior exposure to rituximab must have CD19 counts within the normal range at screening.
• Known autoimmune disease other than MG that could interfere with the course and conduct of the study.
• Received a live vaccine within 4 weeks before screening or has any live vaccination planned during the study.
• Opportunistic infection ≤12 weeks before initial study dosing or currently receiving treatment for a chronic opportunistic infection, such as tuberculosis (TB), pneumocystis pneumonia, cytomegalovirus, herpes simplex virus, herpes zoster, or atypical mycobacteria. A mild, localized herpes simplex infection within 12 weeks of study dosing is allowed, as long as the lesion has resolved without systemic therapy prior to Day 1.

Sponsors & Collaborators

• Takeda: lead

Study Sites (50)

The University of Arizona Medical Center

Tucson, Arizona, 85713, United States

Location

Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States

Location

University of California Davis Medical Center

Sacramento, California, 95816, United States

Location

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

George Washington University

Washington D.C., District of Columbia, 20037, United States

Location

SFM Clinical Research, LLC

Boca Raton, Florida, 33487, United States

Location

Neurology Associates PA

Maitland, Florida, 32751, United States

Location

Medsol Clinical Research Center Inc

Port Charlotte, Florida, 33952, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Consultants In Neurology

Northbrook, Illinois, 60062, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Wayne State University

Detroit, Michigan, 48201, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Neurology and Sleep Disorders Clinic

Columbia, Missouri, 65212, United States

Location

Hospital For Special Surgery

New York, New York, 10021, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Carolinas HealthCare System Neurosciences Institute-Neurology

Charlotte, North Carolina, 28207, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Ohio State University Medical Center

Columbus, Ohio, 43210, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78756, United States

Location

The University of Texas South Western Medical Center

Dallas, Texas, 75390, United States

Location

Central Texas Neurology Consultants PA

Round Rock, Texas, 78681, United States

Location

Center for Neurological Disorders

Milwaukee, Wisconsin, 53215, United States

Location

The Medical College of Wisconsin, Inc.

Milwaukee, Wisconsin, 53226, United States

Location

The Governors of the University of Calgary

Calgary, Alberta, T3M 1M4, Canada

Location

Vancouver General Hospital (VGH)

Vancouver, British Columbia, V6E2E3, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Fondazione Policlinico Universitario A Gemelli

Rome, Lazio, 00168, Italy

Location

Azienda Ospedaliera Universitaria Careggi

Florence, 50139, Italy

Location

IRCCS AOU San Martino

Genova, 16132, Italy

Location

Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta

Milan, 20133, Italy

Location

Azienda Ospedaliera Sant'andrea

Rome, 00189, Italy

Location

Neurocentrum Bydgoszcz sp. z o.o.

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-796, Poland

Location

Centrum Neurologii Klinicznej Sp. z o.o.

Krakow, Lesser Poland Voivodeship, 31-505, Poland

Location

Centrum Medyczne NeuroProtect

Warsaw, 01-684, Poland

Location

Centrum Medyczne Warszawa - PRATIA - PPDS

Warsaw, 01-868, Poland

Location

Clinical Center of Serbia - PPDS

Belgrade, 11000, Serbia

Location

Military Medical Academy

Belgrade, 11000, Serbia

Location

Clinical Center Nis

Niš, 18000, Serbia

Location

Hospital General Universitario de Alicante

Alicante, 03010, Spain

Location

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain

Location

Hospital de La Santa Creu i Sant Pau

Barcelona, 08041, Spain

Location

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Universitario La Paz - PPDS

Madrid, 28046, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

Related Links

• To obtain more information on the study, click here/on this link

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

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  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: November 8, 2019
• First Posted: November 12, 2019
• Study Start: January 14, 2020
• Primary Completion: July 12, 2022
• Study Completion: July 12, 2022
• Last Updated: June 2, 2023
• Results First Posted: June 2, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

PL (4); CA (3); SE (3); ES (8); IT (5); US (27)