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

Generalized Myasthenia Gravis Clinical Trial

— RAISE-XT  

Official title:

A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04225871
• Other study ID #: RA101495-02.302
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 23, 2019
• Est. completion date: June 2, 2026

Study information

• Verified date: May 2024
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 200
• Est. completion date: June 2, 2026
• Est. primary completion date: June 2, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• zilucoplan (RA101495)
Daily subcutaneous (SC) injection

Locations

Country	Name	City	State
Canada	Mg0011 11	Montreal
France	Mg0011 204	Lille
France	Mg0011 118	Nice
France	Mg0011 105	Paris
France	Mg0011 137	Strasbourg
Germany	Mg0011 150	Göttingen
Germany	Mg0011 129	Tübingen
Italy	Mg0011 132	Milano
Italy	Mg0011 126	Roma
Japan	Mg0011 151	Chiba
Japan	Mg0011 136	Hanamaki-shi
Japan	Mg0011 179	Kita-gun
Japan	Mg0011 153	Meguro-ku
Japan	Mg0011 146	Nagasaki-shi
Japan	Mg0011 169	Narita
Japan	Mg0011 152	Sapporo
Japan	Mg0011 144	Sendai
Japan	Mg0011 163	Shinjuku-ku
Japan	Mg0011 141	Tokyo
Norway	Mg0011 140	Bergen
Norway	Mg0011 143	Oslo
Poland	Mg0011 195	Katowice
Poland	Mg0011 213	Katowice
Poland	Mg0011 192	Krakow
Poland	Mg0011 193	Krakow
Poland	Mg0011 211	Kraków
Poland	Mg0011 214	Lódz
Poland	Mg0011 205	Lublin
Poland	Mg0011 194	Nowa Sol
Poland	Mg0011 209	Poznan
Poland	Mg0011 201	Warszawa
Poland	Mg0011 210	Zabrze
Spain	Mg0011 133	Barcelona
Spain	Mg0011 168	Barcelona
Spain	Mg0011 138	Bilbao
United Kingdom	Mg0011 119	Oxford
United Kingdom	Mg0011 130	Sheffield
United States	Mg0011 135	Augusta	Georgia
United States	Mg0011 131	Austin	Texas
United States	Mg0011 30	Buffalo	New York
United States	Mg0011 22	Chapel Hill	North Carolina
United States	Mg0011 128	Charleston	South Carolina
United States	Mg0011 164	Charlottesville	Virginia
United States	Mg0011 122	Cleveland	Ohio
United States	Mg0011 134	Columbia	Missouri
United States	Mg0011 38	Columbus	Ohio
United States	Mg0011 28	Cordova	Tennessee
United States	Mg0011 19	Dallas	Texas
United States	Mg0011 33	Detroit	Michigan
United States	Mg0011 49	East Lansing	Michigan
United States	Mg0011 188	Glenview	Illinois
United States	Mg0011 123	Great Neck	New York
United States	Mg0011 156	Indianapolis	Indiana
United States	Mg0011 31	Irvine	California
United States	Mg0011 32	Kansas City	Kansas
United States	Mg0011 117	Las Vegas	Nevada
United States	MG0011 4	Los Angeles	California
United States	Mg0011 45	Milwaukee	Wisconsin
United States	Mg0011 41	Mobile	Alabama
United States	Mg0011 24	New Haven	Connecticut
United States	Mg0011 23	New York	New York
United States	Mg0011 47	New York	New York
United States	Mg0011 220	Pasadena	California
United States	Mg0011 116	Phoenix	Arizona
United States	Mg0011 40	Pittsburgh	Pennsylvania
United States	Mg0011 39	Salt Lake City	Utah
United States	Mg0011 160	San Francisco	California
United States	Mg0011 154	Seattle	Washington
United States	Mg0011 25	Tampa	Florida
United States	Mg0011 27	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Ra Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  France,  Germany,  Italy,  Japan,  Norway,  Poland,  Spain,  United Kingdom, 

References & Publications (1)

Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209- — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events (TEAEs)	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.	From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months)
Secondary	Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score	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).	From Baseline (Day 1) to Week 12
Secondary	Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score	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).	From Baseline (Day 1) to Week 12
Secondary	Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score	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.	From Baseline (Day 1) to Week 12
Secondary	Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score	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.	From Baseline (Day 1) to Week 12