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Trial record 1 of 1 for:    NCT04115293
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Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)

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ClinicalTrials.gov Identifier: NCT04115293
Recruitment Status : Active, not recruiting
First Posted : October 4, 2019
Last Update Posted : November 9, 2021
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE October 2, 2019
First Posted Date  ICMJE October 4, 2019
Last Update Posted Date November 9, 2021
Actual Study Start Date  ICMJE September 17, 2019
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 1, 2021)
Change from Baseline (CFB) in the MG-ADL Score [ Time Frame: From Baseline (Day 1) to Week 12 ]
The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).
Original Primary Outcome Measures  ICMJE
 (submitted: October 2, 2019)
Change from Baseline (CFB) in the MG-ADL Score [ Time Frame: 12 Weeks ]
The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the ADL score.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 1, 2021)
  • Change from Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score [ Time Frame: From Baseline (Day 1) to Week 12 ]
    The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe).
  • Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score [ Time Frame: From Baseline (Day 1) to Week 12 ]
    The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease.
  • Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score [ Time Frame: From Baseline (Day 1) to Week 12 ]
    The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life.
  • Time to first receipt of rescue therapy over the 12-week Treatment Period [ Time Frame: Treatment Period (from Day 1 to Week 12) ]
    For patients who require rescue treatment, the time from Baseline to the first dose of rescue treatment will be calculated.
  • Percentage of participants achieving Minimal Symptom Expression (MSE) at Week 12 [ Time Frame: Week 12 ]
    MSE is defined as an MG-ADL of 0 or 1 at Week 12 without rescue therapy. The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).
  • Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy [ Time Frame: Week 12 ]
    The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe). A 3-point change in this assessment is considered clinically meaningful.
  • Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 [ Time Frame: Week 12 ]
    The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe). A change in the QMG Score of 3 points or more may be considered clinically meaningful, in a typical clinical trial population of MG patients.
  • Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline (Day 1) to Safety Follow-Up Visit (up to Week 19) ]
    A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 2, 2019)
Change from Baseline (CFB) in the QMG Score [ Time Frame: 12 Weeks ]
The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Official Title  ICMJE A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Brief Summary The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Myasthenia Gravis, Generalized
Intervention  ICMJE
  • Drug: zilucoplan (RA101495)
    Daily subcutaneous (SC) injection
  • Drug: Placebo
    Daily subcutaneous (SC) injection
Study Arms  ICMJE
  • Experimental: 0.3 mg/kg zilucoplan (RA101495)
    Intervention: Drug: zilucoplan (RA101495)
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: October 11, 2021)
174
Original Estimated Enrollment  ICMJE
 (submitted: October 2, 2019)
130
Estimated Study Completion Date  ICMJE February 2022
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
  • Positive serology for acetylcholine receptor (AChR) autoantibodies
  • MG-ADL Score of ≥ 6 at Screening and Baseline
  • QMG score ≥ 12 at Screening and Baseline
  • No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
  • No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period

Exclusion Criteria:

  • Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
  • History of meningococcal disease
  • Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 74 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   France,   Germany,   Italy,   Japan,   Norway,   Poland,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04115293
Other Study ID Numbers  ICMJE RA101495-02.301
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Data from this trial 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 individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, 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.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed;in this case and to protect participants, individual patient-level data would not be made available.
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Data from this trial 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.Vivli.org 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://www.Vivli.org
Responsible Party Ra Pharmaceuticals
Study Sponsor  ICMJE Ra Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: UCB Cares UCB Pharma
PRS Account Ra Pharmaceuticals
Verification Date November 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP