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

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ClinicalTrials.gov Identifier: NCT04159805
Recruitment Status : Recruiting
First Posted : November 12, 2019
Last Update Posted : March 26, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda

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.


Condition or disease Intervention/treatment Phase
Myasthenia Gravis Drug: TAK-079 Drug: TAK-079 Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis
Actual Study Start Date : January 20, 2020
Estimated Primary Completion Date : April 13, 2022
Estimated Study Completion Date : June 25, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: TAK-079 Dose 1
TAK-079 dose 1 injection, subcutaneously, once weekly for 8 weeks.
Drug: TAK-079
TAK-079 subcutaneous injection

Experimental: TAK-079 Dose 2
TAK-079 dose 2 injection, subcutaneously, once weekly for 8 weeks.
Drug: TAK-079
TAK-079 subcutaneous injection

Placebo Comparator: TAK-079 Placebo-matching
TAK-079 placebo-matching injection, subcutaneously, once weekly for 8 weeks.
Drug: TAK-079 Placebo
TAK-079 placebo-matching subcutaneous injection




Primary Outcome Measures :
  1. Percentage of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, AEs Leading to TAK-079 Discontinuation [ Time Frame: From the first dose of study drug up to Week 32 ]
    An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug and will be graded as per national cancer institute common terminology criteria for adverse events (NCI CTCAE), version 4.03.


Secondary Outcome Measures :
  1. Change from Baseline in Myasthenia Gravis (MG) Activities of Daily Living (MG-ADL) Scale Score [ Time Frame: Baseline up to Week 32 ]
    Patient-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.

  2. Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score [ Time Frame: Baseline up to Week 32 ]
    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.

  3. Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score [ Time Frame: Baseline up to Week 32 ]
    An assessment scale of MG disease activity based on a combination of patient- and physician-reported items. Each question is graded on 4 levels of impact from normal to severe with a total score of 0 to 50; the higher score indicates worse MG disease activity.

  4. Change from Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score [ Time Frame: Baseline up to Week 32 ]
    A patient-reported score that assesses the patient's perception of impairment and disability and the degree to which the patient 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.

  5. Change from Baseline in Anti-acetylcholine receptor (AChR) or Anti- Muscle-specific Tyrosine Kinase (MuSK) Antibody Levels [ Time Frame: Baseline up to Week 32 ]
    Clinical laboratory evaluations (anti-AChR and anti-MuSK antibodies) will be tested to monitor disease activity.

  6. Percentage of Participants Meeting Minimal Clinically Important Difference Criteria in MG-ADL Scale Score [ Time Frame: Baseline up to Week 32 ]
    Patient-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.

  7. Percentage of Patients Meeting Minimal Clinically Important Difference Criteria in Quantitative Myasthenia Gravis (QMG) Scale Score [ Time Frame: Baseline up to Week 32 ]
    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.

  8. Percentage of Participants Meeting Minimal Clinically Important Difference Criteria in Myasthenia Gravis Composite (MGC) Scale Score [ Time Frame: Baseline up to Week 32 ]
    An assessment scale of MG disease activity based on a combination of patient- and physician-reported items. Each question is graded on 4 levels of impact from normal to severe with a total score of 0 to 50; the higher score indicates worse MG disease activity.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04159805


Contacts
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Contact: Takeda Study Registration Call Center +1 877-825-3327 medinfoUS@takeda.com

Locations
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Sponsors and Collaborators
Takeda
Investigators
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Study Director: Medical Director Takeda
Additional Information:
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Responsible Party: Takeda
ClinicalTrials.gov Identifier: NCT04159805    
Other Study ID Numbers: TAK-079-1005
2019-003383-47 ( EudraCT Number )
First Posted: November 12, 2019    Key Record Dates
Last Update Posted: March 26, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: 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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (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.
URL: https://vivli.org/ourmember/takeda/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Takeda:
Drug therapy
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases