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Trial record 1 of 1 for:    NCT04124965
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A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients 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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04124965
Recruitment Status : Enrolling by invitation
First Posted : October 14, 2019
Last Update Posted : January 6, 2020
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Biopharma S.P.R.L. )

Brief Summary:
The purpose of the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG).

Condition or disease Intervention/treatment Phase
Myasthenia Gravis Drug: Rozanolixizumab Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 240 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Actual Study Start Date : October 29, 2019
Estimated Primary Completion Date : April 2022
Estimated Study Completion Date : April 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Rozanolixizumab dosage regimen 1
Study participants randomized in dosage regimen 1 will receive assigned dosages of rozanolixizumab at pre-specified time points during Treatment Period.
Drug: Rozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in 2 different dosages (dose 1 and dose 2) in dosage regimen 1 and 2.
Other Name: UCB7665

Experimental: Rozanolixizumab dosage regimen 2
Study participants randomized in dosage regimen 2 will receive assigned dosages of rozanolixizumab at pre-specified time points during Treatment Period.
Drug: Rozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in 2 different dosages (dose 1 and dose 2) in dosage regimen 1 and 2.
Other Name: UCB7665




Primary Outcome Measures :
  1. Percentage of participants with treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline until Final Visit (up to Week 60) ]
    An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

  2. Percentage of participants with treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication [ Time Frame: From Baseline until Final Visit (up to Week 60) ]
    An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.


Secondary Outcome Measures :
  1. Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at each scheduled assessment during Treatment and Observation Periods [ Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60) ]

    The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability.

    A positive change indicates worsening and a negative change indicates improvement!


  2. Change from Baseline in Myasthenia Gravis (MG)-Composite score at each scheduled assessment during Treatment and Observation Periods [ Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60) ]
    The total MG-Composite score is obtained by summing the responses to each individual item (10 items; Grade:0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity.

  3. Change from Baseline in Quantitative Myasthenia Gravis (QMG) score at each scheduled assessment during Treatment and Observation Periods [ Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60) ]
    The total QMG score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.

  4. Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX)) during the study [ Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60) ]
    Rescue therapy for the study will consist of IVIg or PEX. Study participants who experience disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator.



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

Inclusion Criteria:

  • Participant must be ≥18 years of age at the time of signing the informed consent
  • Participant was eligible for MG0003 [NCT03971422] at the time of enrollment into MG0003 and the participant either completed the Observation Period of MG0003 or required rescue therapy during the Observation Period of MG0003
  • Body weight >35 kg at Visit 1
  • Study participants may be male or female
  • A male study participant must agree to use contraception
  • Female study participants of childbearing potential must agree to use a highly effective method of birth control
  • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:

    1. Not a woman of childbearing potential (WOCBP) OR
    2. A WOCBP who agrees to follow the contraceptive guidance

Exclusion Criteria:

  • Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold test
  • Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication
  • Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs
  • Study participant with severe (defined as Grade 3 on the myasthenia gravis-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
  • Participant has absolute neutrophil count <1500 cells/mm3
  • Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant's ability to participate in this study
  • Participant has 12-lead electrocardiogram (ECG) with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the Investigator to determine eligibility, and any queries regarding continuation of the study participant must be addressed with the Medical Monitor
  • Study participant has renal impairment, defined as serum creatinine level of ≥1.4 mg/dL for females and ≥1.5mg/dL for males
  • Study participant has >2x upper limit of normal (ULN) of any of the following at Visit 1: alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%). If study participant has elevations only in total bilirubin that are >ULN and <1.5xULN, fractionate bilirubin to identify possible undiagnosed Gilbert's syndrome (ie, direct bilirubin <35%). For randomized study participants with a baseline result >ULN for ALT, AST, ALP, or total bilirubin but <1.5xULN, a baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the electronic Case Report form (eCRF). Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit (>2xULN) may be repeated once for confirmation
  • Study participant has positive human immunodeficiency virus antibody test
  • Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 [NCT03971422] or discontinued study medication in MG0003, with the exception of discontinuation due to a need for rescue treatment
  • Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator
  • Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening

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): NCT04124965


Locations
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United States, Florida
Mg0004 50120
Miami, Florida, United States, 33144
Mg0004 50073
Tampa, Florida, United States, 33612
United States, Kentucky
Mg0004 50121
Lexington, Kentucky, United States, 40536
Sponsors and Collaborators
UCB Biopharma S.P.R.L.
Investigators
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Study Director: UCB Cares 001 844 599 22733 (UCB)

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Responsible Party: UCB Biopharma S.P.R.L.
ClinicalTrials.gov Identifier: NCT04124965    
Other Study ID Numbers: MG0004
2019-000969-21 ( EudraCT Number )
First Posted: October 14, 2019    Key Record Dates
Last Update Posted: January 6, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.clinicalstudydatarequest.com and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if a determination is made that the data cannot be adequately anonymized.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.clinicalstudydatarequest.com and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
URL: http://clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by UCB Pharma ( UCB Biopharma S.P.R.L. ):
UCB7665
Myasthenia Gravis
Rozanolixizumab
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Rozanolixizumab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs