Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants With Generalized Myasthenia Gravis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04159805
Recruitment Status : Recruiting
First Posted : November 12, 2019
Last Update Posted : September 4, 2020
Sponsor:
Information provided by (Responsible Party):
Takeda

Brief Summary:
The purpose of the study is to evaluate the safety and tolerability of TAK-079 in participants with generalized myasthenia gravis (MG) who are receiving stable background therapy for MG.

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.

Layout table for study information
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 14, 2020
Estimated Primary Completion Date : February 3, 2021
Estimated Study Completion Date : May 12, 2021

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 16 ]
    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 will be graded evaluated 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 and Week 16 ]
    Patient-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question was graded on a 3-point scale from 0=normal to 3=severe with a 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 and Week 16 ]
    Physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question was graded on a 3-point scale from 0=normal to 3=severe with a 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 and Week 16 ]
    An assessment scale of MG disease activity based on a combination of patient- and physician-reported items. Each question was graded on 4 levels of impact from normal to severe with a 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 and Week 16 ]
    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 was graded on a 3-point scale from 0=normal to 2=severe with a 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 and Week 16 ]
    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: Week 16 ]
    Patient-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question was graded on a 3-point scale from 0=normal to 3=severe with a 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: Week 16 ]
    Physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question was graded on a 3-point scale from 0=normal to 3=severe with a 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: Week 16 ]
    An assessment scale of MG disease activity based on a combination of patient- and physician-reported items. Each question was graded on 4 levels of impact from normal to severe with a score of 0 to 50; the higher score indicates worse MG disease activity.

  9. Percentage of Participants Meeting Minimal Clinically Important Difference Criteria in MG-QoL15r Scale Score [ Time Frame: Week 16 ]
    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 was graded on a 3-point scale from 0=normal to 2=severe with a score of 0 to 30; 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.


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

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 ± one steroid-sparing immunosuppressive drug (limited to azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide). Participants must be on at least one allowed background medication.

Exclusion Criteria:

  1. History of thymoma or other thymic neoplasms.
  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.
  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
Layout table for location contacts
Contact: Takeda Study Registration Call Center +1 877-825-3327 medinfoUS@takeda.com

Locations
Show Show 50 study locations
Sponsors and Collaborators
Takeda
Investigators
Layout table for investigator information
Study Director: Medical Director Takeda
Layout table for additonal information
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: September 4, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

Layout table for additional information
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:
Layout table for MeSH terms
Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases