Multifocal motor neuropathy MedDRA version: 9.1 Level: PT Classification code 10065579 Term: Multifocal motor neuropathy
Conditions
Interventions
Trade Name: SOLIRIS
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration
Sponsors
Greater Glasgow and Clyde NHS Board and The University Of Glasgow
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Fulfilling the diagnostic clinical and electrophysiological entry requirements in Appendix A, as adapted from the European Federation of Neurological Societies (EFNS) and the Peripheral Nerve Society (PNS)*. 2. Able to complete self-evaluation functional rating scale 3. Documented historical responsiveness to IVIg in the opinion of the Investigator 4. Agreement to vaccination against meningococcal infection. (Tetravalent, conjugated vaccination recommended) 5. Is willing and able to give written informed consent. *Conduction Block (CB) to have been documented at some point in the patient’s history. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Below the age of 18 years old. 2. Pregnancy, planned pregnancy or lactation* 3. Inability to comply with study related procedures or appointments. 4. Unresolved Neisseria meningitidis infection. 5. Any condition that in the opinion of the Investigator could increase the patient’s risk by participating in the study or confound the outcome of the study. 6. Hypersensitivity to eculizumab, murine proteins or to any of the excipients 7. Known or suspected hereditary complement deficiencies. *A serum pregnancy test will be preformed at the first study visit. Trial participants of child-bearing age will be asked to use adequate contraception during the trial and for 5 months after study cessation. Participants will be instructed to inform the PI immediately if they suspect they may be pregnant The contraindications and special warning regarding meningococcal infection from the Summary of Product Characteristics of eculizumab in its licensed indication, Paroxysmal Nocturnal Haemoglobinuria, are reflected in the inclusion and exclusion criteria of this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety and tolerability of eculizumab in patients with Multifocal Motor Neuropathy (MMN) receiving cycles of intravenous human immunoglobulin (IVIg).;Secondary Objective: To explore the effectiveness of eculizumab in terms of reduction in IVIg requirements and improvements in clinical examination, symptom rating and functionality in IVIg- responsive MMN patients. ;Primary end point(s): -Frequency of adverse events and serious adverse events -Amount of IVIg treatment required, during Run-in compared to Treatment period. -Change in scores/rating scales/measurements between baseline and during treatment. | — |
Countries
United Kingdom
Outcome results
None listed