0.3 mg/kg zilucoplan (RA101495) for Myasthenia Gravis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Myasthenia Graviszilucoplan (RA101495) - Drug
Eligibility
18 - 85
All Sexes
What conditions do you have?
Select

Study Summary

The purpose of the study is to evaluate the safety and tolerability of switching from intravenous (IV) complement component 5 (C5) inhibitors to subcutaneous (SC) Zilucoplan in study participants with generalized myasthenia gravis (gMG)

Eligible Conditions
  • Myasthenia Gravis

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

4 Primary · 6 Secondary · Reporting Duration: From Baseline (Day 1) to Safety Follow-Up Visit (up to 18 weeks)

Week 12
Incidence of study withdrawal
Incidence of study withdrawal over the Main Treatment Period
Week 18
Incidence of serious treatment-emergent adverse events (serious TEAEs)
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events (TEAEs) leading to withdrawal of study medication
Week 18
Incidence of serious treatment-emergent adverse events (serious TEAEs) over the Main Treatment Period
Incidence of treatment-emergent adverse events (TEAEs) leading to withdrawal of study medication over the Main Treatment Period
Incidence of treatment-emergent adverse events (TEAEs) over the Main Treatment Period
Week 12
Change from Baseline to Week 12 in MG ADL score
Change from Baseline to Week 12 in the QMG score

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Zilucoplan 0.3 mg/kg
16%Injection site bruising
15%Headache
10%Diarrhoea
9%Injection site pain
9%Myasthenia gravis
8%Urinary tract infection
8%Contusion
7%Lipase increased
6%Nasopharyngitis
6%Amylase increased
3%Vomiting
3%Rash
1%Oesophageal candidiasis
1%Oral candidiasis
1%Pneumonia
1%Sepsis
1%Anaemia
1%Aphthous ulcer
1%COVID-19
1%COVID-19 pneumonia
1%Basal cell carcinoma
1%Pulmonary embolism
1%Angioedema
This histogram enumerates side effects from a completed 2021 Phase 3 trial (NCT04115293) in the Zilucoplan 0.3 mg/kg ARM group. Side effects include: Injection site bruising with 16%, Headache with 15%, Diarrhoea with 10%, Injection site pain with 9%, Myasthenia gravis with 9%.

Trial Design

1 Treatment Group

0.3 mg/kg zilucoplan (RA101495)
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: 0.3 mg/kg zilucoplan (RA101495) · No Placebo Group · Phase 3

0.3 mg/kg zilucoplan (RA101495)
Drug
Experimental Group · 1 Intervention: zilucoplan (RA101495) · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
zilucoplan (RA101495)
2019
Completed Phase 3
~220

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from baseline (day 1) to safety follow-up visit (up to 18 weeks)

Who is running the clinical trial?

UCB Biopharma SRLLead Sponsor
85 Previous Clinical Trials
20,754 Total Patients Enrolled
5 Trials studying Myasthenia Gravis
579 Patients Enrolled for Myasthenia Gravis
UCB CaresStudy Director001 844 599 2273
189 Previous Clinical Trials
43,640 Total Patients Enrolled
8 Trials studying Myasthenia Gravis
928 Patients Enrolled for Myasthenia Gravis

Eligibility Criteria

Age 18 - 85 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have been taking an IV C5 inhibitor approved for the treatment of gMG at the recommended dose regimen for a minimum period of 3 months (eculizumab) or 4 months (ravulizumab), and your disease is clinically stable according to the Investigator.
You are willing to exchange your current intravenous C5 inhibitor for a subcutaneous administration of zilucoplan.
Your MG-ADL score has not deviated more than two points from your initial evaluation.
Your corticosteroid dose has remained steady and will not be altered over the 12-week study period.
You have not altered your immunosuppressive medication, or do not anticipate doing so, in the course of 12 weeks.
You have received a minimum of one dose each of both quadrivalent meningococcal and serotype B meningococcal vaccines within the past 14 days, or you were vaccinated with these at least 3 years ago.
You are either male or female.
You must have a certified diagnosis of gMG (Myasthenia Gravis Foundation of America; MGFA Class II-IVa) recorded prior to the start of this study, supported by pertinent reviews.
You have previously tested positive for acetylcholine receptor binding autoantibodies.
A male participant is encouraged to take contraceptive measures and abstain from donating sperm for at least 40 days after receiving the last dose of study medication.