Clinical trial NCT00701662: A Study of Purified Human Antibodies Administered Subcutaneously to Patients With Multifocal Motor Neuropathy (MMN)
Official title: A Multicentre Study of Subcutaneous Immunoglobulin (SCIG) in Patients With Multifocal Motor Neuropathy (MMN)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multifocal Motor Neuropathy (MMN)
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2008-06-19
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study is to assess efficacy, safety, and convenience of purified human antibodies administered under the skin in the treatment of MMN patients.


Eligibility criteria: Inclusion Criteria:

* Patients with documented clinical diagnosis and electrophysiological evidence of MMN
* Patients who have previously responded to intravenous immunoglobulin (IVIG) and have been on stable treatment with IVIG for at least 12 weeks prior to screening
* Patients treated with the equivalent of ≥0.4g/kg body weight (bw) IVIG per month
* Provision of informed consent by patient

Exclusion Criteria:

* Aspartate aminotransferase (ASAT) or alanine aminotransferase (ALAT) concentration \>2.5 times the upper normal limit (UNL)
* Creatinine concentration \>1.5 times the UNL
* Known allergic reactions to blood products
* Any skin disease interfering with the assessment of injection site reactions
* Any other medical condition, which in the opinion of the investigator, might interfere with successful completion of the protocol
* Any condition likely to interfere with the evaluation of the study drug or satisfactory conduct of the trial
* Participation in a study with an investigational drug within three months prior to enrolment
* Patients treated with the equivalent of \>2.0g/kg bw IVIG per month

Primary outcomes:
measure: Change From Baseline to Week 24 in Muscle Strength
timeFrame: Baseline to week 24
measure: Mean Overall MRC Score at Baseline and Week 24
timeFrame: Baseline and week 24
Secondary outcomes:
measure: Change From Baseline to Week 24 in Disability
timeFrame: Baseline to week 24
measure: Mean Disability Score at Baseline and Week 24
timeFrame: Baseline and Week 24
measure: Change From Baseline to the Completion Visit in Motor Function
timeFrame: Baseline to the completion visit (up to week 25)
measure: Mean Motor Function Score at Screening and Week 25
timeFrame: Screening and week 25
measure: Health-Related Quality of Life at Baseline and Week 25
timeFrame: At baseline and week 25
measure: Treatment Satisfaction at Baseline and Week 25
timeFrame: At baseline and week 25
measure: Overall Health Status at Baseline and Week 25
timeFrame: Baseline and week 25
measure: Number of Patients With Adverse Events (AEs) by Severity and Relatedness
timeFrame: For the duration of the study, up to Week 25
measure: Rate of AEs by Severity and Relatedness
timeFrame: For the duration of the study, up to Week 25
measure: Number of Patients With Local/Injection Site Reactions
timeFrame: For the duration of the study, up to Week 25
measure: Number of Patients With Clinically Relevant Changes in Laboratory Parameters
timeFrame: Baseline to Week 25
measure: Number of Patients With Clinically Relevant Changes in Vital Signs
timeFrame: Baseline to Week 25
Other outcomes:
Locations:
city: Milan
country: Italy
facility: San Raffaele Hospital
city: Bern
country: Switzerland
facility: Inselspital
city: Oxford
country: United Kingdom
facility: Dept. Clinical Immunology, Oxford Radcliffe Hospitals
Study officials:
affiliation: Inselspital, University Hospital of Bern
name: Matthias Sturzenegger, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Neurologische Klinik, Heinrich-Heine-University, Düsseldorf
name: Bernd Kieseier, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: San Raffaele Hospital
name: Giancarlo Comi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Dept. Clinical Immunology, Oxford Radcliffe Hospitals
name: Siraj Misbah, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Misbah S, et al. Efficacy and safety of subcutaneous immunoglobulin, Vivaglobin, in patients with multifocal motor neuropathy. Journal of Neurology 257(Suppl 1):S105-S106, 2010.
citation: Misbah SA, Baumann A, Fazio R, Dacci P, Schmidt DS, Burton J, Sturzenegger M. A smooth transition protocol for patients with multifocal motor neuropathy going from intravenous to subcutaneous immunoglobulin therapy: an open-label proof-of-concept study. J Peripher Nerv Syst. 2011 Jun;16(2):92-7. doi: 10.1111/j.1529-8027.2011.00330.x.