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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 : October 12, 2021
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, Generalized Drug: zilucoplan (RA101495) Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 174 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : September 17, 2019
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : February 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 0.3 mg/kg zilucoplan (RA101495) Drug: zilucoplan (RA101495)
Daily subcutaneous (SC) injection

Placebo Comparator: Placebo Drug: Placebo
Daily subcutaneous (SC) injection




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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).

  2. 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.

  3. 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.

  4. 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.

  5. 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).

  6. 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.

  7. 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.

  8. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 74 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT04115293


Locations
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Sponsors and Collaborators
Ra Pharmaceuticals
Investigators
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Study Director: UCB Cares UCB Pharma
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Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04115293    
Other Study ID Numbers: RA101495-02.301
First Posted: October 4, 2019    Key Record Dates
Last Update Posted: October 12, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
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
Statistical Analysis Plan (SAP)
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
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