Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis

• Register: EUCTR
• Last refreshed on: 16 December 2019
• Main ID: EUCTR2019-001565-33-GB
• Date of registration: 01/11/2019
• Prospective Registration: No
• Primary sponsor: Ra Pharmaceuticals, Inc.
• Public title: Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
• Scientific title: A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis - RAISE-XT
• Date of first enrolment: 30/10/2019
• Target sample size: 200
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001565-33
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Clinical Trial Information
• Affiliation: Ra Pharmaceuticals, Inc

• Name: Clinical Trial Information
• Affiliation: Ra Pharmaceuticals, Inc

Key inclusion & exclusion criteria

Inclusion criteria:
To be eligible for this study, subjects must meet ALL of the following inclusion criteria:
1.Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan study
2.Able to provide informed consent, including signing and dating the informed consent form (ICF)
3.Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine. A booster vaccination should also be administered as clinically indicated, according to the local SOC, for subjects who have been previously vaccinated against Neisseria meningitidis
4.Female subjects of childbearing potential must have a negative urine pregnancy test within 24 hours prior to the first dose of study drug
5.Sexually active female subjects of childbearing potential (i.e., women who are not postmenopausal or who have not had a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) and all male subjects (who have not been surgically sterilized by vasectomy) must agree to use effective contraception during the study. Postmenopausal women are, for the purposes of this protocol, defined as women who have gone 12 consecutive months without menstruation

Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 150
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion criteria:
Subjects who meet ANY of the following exclusion criteria must be excluded from the study:
1.Pregnant, planning to become pregnant, or nursing female subjects
2.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)
3.Unable or unwilling to comply with the requirements of the study
4.Did not start any disallowed medication per the exclusion criteria from the qualifying zilucoplan study or alter the dose of any other concomitant medication, unless medically indicated
5.Any new or worsening medical condition (since entry into the qualifying zilucoplan study) or any other reason that, in the opinion of the investigator or Sponsor, would disqualify the subject from participation in this study



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Generalized Myasthenia Gravis
MedDRA version: 20.0 Level: LLT Classification code 10028415 Term: Myasthenia System Organ Class: 100000004859

Intervention(s)

Product Name: Zilucoplan
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Zilucoplan
CAS Number: 1841136-73-9
Current Sponsor code: RA101495
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-

Primary Outcome(s)

Main Objective: •To provide access to zilucoplan for subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan study and who wish to continue receiving zilucoplan
•To evaluate the long-term efficacy of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study
•To evaluate the long-term safety and tolerability of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study

Primary end point(s): Efficacy endpoints:
•MG-ADL Score
•QMG Score
•MG-QOL15r
•MGC Score
Safety endpoints

Timepoint(s) of evaluation of this end point: during each visit

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): Not applicable

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

RA101495-02.302

Source(s) of Monetary Support

Ra Pharmaceuticals, Inc.

Ethics review

Status: Approved
Approval date:
Contact: