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Myasthenia GravisPhase 2NCT04159805

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

A Phase 2 study testing TAK-079 in people with Myasthenia Gravis, run by Takeda at 50 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myasthenia Gravis
What is being tested?
TAK-079
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
36 participants
Where
50 sitesin 6 countries
Study dates
14 Jan 2020to 12 Jul 2022
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Main Inclusion Criteria:

  1. Diagnosis of Myasthenia Gravis (MG) supported by a positive serologic test for anti-AChR or anti-MuSK antibodies at screening.
  2. Myasthenia Gravis Foundation of America (MGFA) clinical classification II to IV at screening.
  3. 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.
  4. 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.
  5. 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.
  6. If receiving cholinesterase inhibitors, therapy with a stable dose is required at least 2 weeks before screening.
  7. 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.

Main Exclusion Criteria:

  1. 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.
  2. History of thymectomy within 12 months before screening.
  3. MGFA class I or V.
  4. 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.
  5. 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.
  6. 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.
  7. Known autoimmune disease other than MG that could interfere with the course and conduct of the study.
  8. Received a live vaccine within 4 weeks before screening or has any live vaccination planned during the study.
  9. 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.

Trial locations and contacts

50 sites in 6 countries.

50 sites in 6 countries

United States27 sites

  • Augusta University

    Augusta, Georgia, 30912

  • University of Colorado Hospital

    Aurora, Colorado, 80045

  • Austin Neuromuscular Center

    Austin, Texas, 78756

  • SFM Clinical Research, LLC

    Boca Raton, Florida, 33487

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27514

  • Carolinas HealthCare System Neurosciences Institute-Neurology

    Charlotte, North Carolina, 28207

  • University of Cincinnati

    Cincinnati, Ohio, 45267

  • Neurology and Sleep Disorders Clinic

    Columbia, Missouri, 65212

  • Ohio State University Medical Center

    Columbus, Ohio, 43210

  • The University of Texas South Western Medical Center

    Dallas, Texas, 75390

  • Wayne State University

    Detroit, Michigan, 48201

  • Henry Ford Health System

    Detroit, Michigan, 48202

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Michigan State University

    East Lansing, Michigan, 48824

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • Neurology Associates PA

    Maitland, Florida, 32751

  • Center for Neurological Disorders

    Milwaukee, Wisconsin, 53215

  • The Medical College of Wisconsin, Inc.

    Milwaukee, Wisconsin, 53226

  • Hospital For Special Surgery

    New York, New York, 10021

  • Consultants In Neurology

    Northbrook, Illinois, 60062

  • Stanford Neuroscience Health Center

    Palo Alto, California, 94304

  • Medsol Clinical Research Center Inc

    Port Charlotte, Florida, 33952

  • Central Texas Neurology Consultants PA

    Round Rock, Texas, 78681

  • University of California Davis Medical Center

    Sacramento, California, 95816

  • University of California Davis Medical Center

    Sacramento, California, 95817

  • The University of Arizona Medical Center

    Tucson, Arizona, 85713

  • George Washington University

    Washington D.C., District of Columbia, 20037

Other trials in United States

Spain8 sites

  • Hospital General Universitario de Alicante

    Alicante, 03010

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, 08035

  • Hospital de La Santa Creu i Sant Pau

    Barcelona, 08041

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007

  • Hospital Clinico San Carlos

    Madrid, 28040

  • Hospital Universitario La Paz - PPDS

    Madrid, 28046

  • Hospital Universitario Virgen Macarena

    Seville, 41009

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026

Other trials in Spain

Italy5 sites

  • Azienda Ospedaliera Universitaria Careggi

    Florence, 50139

  • IRCCS AOU San Martino

    Genova, 16132

  • Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta

    Milan, 20133

  • Fondazione Policlinico Universitario A Gemelli

    Rome, Lazio, 00168

  • Azienda Ospedaliera Sant'andrea

    Rome, 00189

Other trials in Italy

Poland4 sites

  • Neurocentrum Bydgoszcz sp. z o.o.

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-796

  • Centrum Neurologii Klinicznej Sp. z o.o.

    Krakow, Lesser Poland Voivodeship, 31-505

  • Centrum Medyczne NeuroProtect

    Warsaw, 01-684

  • Centrum Medyczne Warszawa - PRATIA - PPDS

    Warsaw, 01-868

Other trials in Poland

Canada3 sites

  • The Governors of the University of Calgary

    Calgary, Alberta, T3M 1M4

  • Toronto General Hospital

    Toronto, Ontario, M5G 2C4

  • Vancouver General Hospital (VGH)

    Vancouver, British Columbia, V6E2E3

Other trials in Canada

Serbia3 sites

  • Clinical Center of Serbia - PPDS

    Belgrade, 11000

  • Military Medical Academy

    Belgrade, 11000

  • Clinical Center Nis

    Niš, 18000

Other trials in Serbia

About this study

Official title: A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis

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.

Read more

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.

Read the detailed description

Myasthenia gravis (MG) is an autoimmune disorder in which autoantibodies, such as those targeting the nicotinic acetylcholine receptor (AChR) or muscle specific kinase (MuSK), interfere with neuromuscular transmission, resulting in fatigue and weakness.

The drug being tested in this study is called TAK-079. TAK-079 is being tested to treat people who have generalized myasthenia gravis.

Being tested
TAK-079, TAK-079 Placebo
Keywords
Drug therapy
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TAK-079Drug

    Also called Mezagitamab

    TAK-079 subcutaneous injection

  • TAK-079 PlaceboDrug

    TAK-079 placebo-matching subcutaneous injection

Study groups in NCT04159805
Study groupRoleWhat they receive
TAK-079 Placebo-matchingPlacebo comparatorTAK-079 placebo-matching injection, subcutaneously (SC), once weekly in combination with standard background therapy for 8 weeks.
Receives: Drug: TAK-079 Placebo
TAK-079 300 mgExperimentalTAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.
Receives: Drug: TAK-079
TAK-079 600 mgExperimentalTAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.
Receives: Drug: TAK-079

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (9)

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

    Participant-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 24; the higher score indicates greater functional impairment and disability. Negative change indicates improvement. Mixed-effects model for repeated measures (MMRM) was used for the analysis.

    Measured: Baseline up to Week 32

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

    Physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 39; the higher score indicates greater disease burden. Negative change indicates improvement. MMRM was used for the analysis.

    Measured: Baseline up to Week 32

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

    An assessment scale of MG disease activity based on a combination of participant- and physician-reported items. Items are scored based on 4 potential levels of impact: normal, mild, moderate, or severe with a total score of 0 to 50; the higher score indicates worse MG disease activity. Negative change indicates improvement. MMRM was used for the analysis.

    Measured: Baseline up to Week 32

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

    A participant-reported score that assesses the participant's perception of impairment and disability and the degree to which the participant tolerates disease manifestations. Each question is graded on a 3-point scale from 0=normal to 2=severe with a total score of 0 to 30; the higher score indicates worse MG disease activity. Negative change indicates improvement. MMRM was used for the analysis.

    Measured: Baseline up to Week 32

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

    Clinical laboratory evaluations of anti-AChR antibodies were tested to monitor disease activity. MMRM was used for the analysis.

    Measured: Baseline up to Week 32

  6. Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

    Clinical laboratory evaluations of anti-MuSK antibodies were tested to monitor disease activity. Data is reported for participants who were positive for anti-MuSK antibodies at baseline.

    Measured: Baseline up to Week 32

  7. Percentage of Participants With 2-point Reduction in MG-ADL Total Score

    The percentage of responders with at least a 2-point reduction in MG-ADL total score from baseline is reported. MG-ADL is a participant-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 24; the higher score indicates greater functional impairment and disability. Percentages are rounded off to whole number at the nearest decimal.

    Measured: At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

  8. Percentage of Participants With 3-point Reduction in QMG Total Score

    The percentage of responders with at least a 3-point reduction in QMG total score from baseline is reported. QMG is a physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 39; the higher score indicates greater disease burden. Percentages are rounded off to whole number at the nearest decimal.

    Measured: At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

  9. Percentage of Participants With 3-point Reduction in MGC Total Score

    The percentage of responders with at least a 3-point reduction in MGC total score from baseline is reported. MGC is an assessment scale of MG disease activity based on a combination of participant- and physician-reported items. Items are scored based on 4 potential levels of impact: normal, mild, moderate, or severe with a total score of 0 to 50; the higher score indicates worse MG disease activity. Percentages are rounded off to whole number at the nearest decimal.

    Measured: At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Nov 2019
  2. Study start14 Jan 2020
  3. Primary completion12 Jul 2022
  4. Study completion12 Jul 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04159805
Sponsor's protocol ID
TAK-079-1005
Other IDs
2019-003383-47, 2019
Status last verified
May 2023
First submitted
8 Nov 2019
Last updated
2 Jun 2023
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing TAK-079 in people with Myasthenia Gravis, run by Takeda at 50 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 50 sites in United States, Spain, Italy, Poland, Canada and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: From signing the informed consent form up to end of long-term follow-up (up to Week 32)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04159805.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2023.

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