Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Phase 3

Active, not recruiting

• Conditions: Generalized Myasthenia Gravis
• Interventions: Drug: zilucoplan (RA101495)

• Registration Number: NCT04225871
• Lead Sponsor: UCB Biopharma SRL

Brief Summary

The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-10-02
• Study Start: 2019-12-23
• Study First Submitted QC: 2020-01-08
• Study First Posted: 2020-01-13
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-04
• Last Update Posted: 2025-07-09
• Primary Completion: 2026-06-02
• Study Completion: 2026-06-02

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

• Completion of a qualifying zilucoplan study

Exclusion Criteria

• With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
0.3 mg/kg zilucoplan (RA101495)	zilucoplan (RA101495)	-

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (TEAEs)	From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months)	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.

Secondary Outcome Measures

Name	Time	Method
Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score	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).
Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score	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	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	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.

Trial Locations

Locations (70)

Mg0011 41; 🇺🇸 Mobile, Alabama, United States

Mg0011 116; 🇺🇸 Phoenix, Arizona, United States

Mg0011 31; 🇺🇸 Irvine, California, United States

MG0011 4; 🇺🇸 Los Angeles, California, United States

Mg0011 220; 🇺🇸 Pasadena, California, United States

Mg0011 160; 🇺🇸 San Francisco, California, United States

Mg0011 24; 🇺🇸 New Haven, Connecticut, United States

Mg0011 27; 🇺🇸 Washington, District of Columbia, United States

Mg0011 25; 🇺🇸 Tampa, Florida, United States

Mg0011 135; 🇺🇸 Augusta, Georgia, United States

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