Multifocal Motor Neuropathy (MMN)Phase 2NCT00701662
A Study of Purified Human Antibodies Administered Subcutaneously to Patients With Multifocal Motor Neuropathy (MMN)
A Phase 2 study testing Vivaglobin in people with Multifocal Motor Neuropathy (MMN), run by CSL Behring at 3 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multifocal Motor Neuropathy (MMN)
- What is being tested?
- Vivaglobin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesin 3 countries
- Study dates
- Nov 2007to Jan 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United Kingdom1 site
Dept. Clinical Immunology, Oxford Radcliffe Hospitals
Oxford
About this study
Official title: A Multicentre Study of Subcutaneous Immunoglobulin (SCIG) in Patients With Multifocal Motor Neuropathy (MMN)
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.
- Conditions
- Multifocal Motor Neuropathy (MMN)
- Being tested
- Vivaglobin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VivaglobinBiological
Also called Human Normal Immunoglobulin
| Study group | Role | What they receive |
|---|---|---|
| Vivaglobin | Experimental | Vivaglobin® is a 16% (160 mg/mL) liquid formulation of human normal immunoglobulin for subcutaneous infusion. Subjects will receive weekly infusions of Vivaglobin® at a weekly dosage calculated based on previous intravenous immunoglobulin treatment (between 0.1 to 0.5 g/kg body weight per week). Receives: Biological: Vivaglobin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 24 in Muscle Strength
The change in Medical Research Council (MRC) score was determined at week 24 compared to baseline using descriptive statistics and nonparametric, two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available.
The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement. Positive values for change in MRC sum score indicate improvement, with a more positive value indicating greater muscle contraction/ limb movement compared with the value at baseline.
Measured: Baseline to week 24
Mean Overall MRC Score at Baseline and Week 24
The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement.
Measured: Baseline and week 24
Secondary outcomes (12)
Change From Baseline to Week 24 in Disability
The change in disability score was determined at week 24 compared to baseline using descriptive statistics and nonparametric two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available.
Disability was measured using a modified Guy's Neurological Disability Scale, which comprises subscales for upper and lower limb disability. Both subscales comprise 6 grades, numbered from 0 (no upper limb problem/walking is not affected) to 5 (unable to use either arm for any purposeful movements/usually uses a wheelchair indoors). The disability score is calculated as the sum of both subscales, resulting in a score ranging from 0 to 10. A higher disability score indicates greater disability. Negative values for change in disability score indicate improvement, with a more negative value indicating greater improvement compared with the value at baseline.
Measured: Baseline to week 24
Mean Disability Score at Baseline and Week 24
Disability was measured using a modified Guy's Neurological Disability Scale, which comprises subscales for upper and lower limb disability. Both subscales comprise 6 grades, numbered from 0 (no upper limb problem/walking is not affected) to 5 (unable to use either arm for any purposeful movements/usually uses a wheelchair indoors). The disability score is calculated as the sum of both subscales, resulting in a score ranging from 0 to 10. A higher disability score indicates greater disability.
Measured: Baseline and Week 24
Change From Baseline to the Completion Visit in Motor Function
The change in motor function was determined at the completion visit compared to baseline using descriptive statistics and nonparametric two-sided 95% confidence intervals based on the Hodges-Lehmann method.
For each patient, four specific tasks were defined according to his/her weakened muscle group. The patient had to grade each of the tasks on a 5-point scale ranging from 0 (normal function) to 4 (not possible). The overall motor function score was calculated as the sum of the 4 grades, resulting in a score ranging from 0 (optimal) to 16 (worst). The baseline motor function score was calculated as the mean of the patient's assessments at Screening and Week 1. Negative values for change in motor function score indicate improvement, with a more negative value indicating greater improvement compared with the value at baseline.
Measured: Baseline to the completion visit (up to week 25)
Mean Motor Function Score at Screening and Week 25
For each patient, four specific tasks were defined according to his/her weakened muscle group. The patient had to grade each of the tasks on a 5-point scale ranging from 0 (normal function) to 4 (not possible). The overall motor function score was calculated as the sum of the 4 grades, resulting in a score ranging from 0 (optimal) to 16 (worst).
Measured: Screening and week 25
Health-Related Quality of Life at Baseline and Week 25
Assessed using a questionnaire on patients' satisfaction with current immunoglobulin G (IgG) treatment, treatment at home, and treatment at the hospital/doctor's office. The questions were answered by choosing a number between 1 (extremely good) and 7 (extremely bad).
Note: No patients received IgG treatment at the hospital/doctor's office at Week 25.
Measured: At baseline and week 25
Treatment Satisfaction at Baseline and Week 25
Treatment satisfaction was assessed using the Life Quality Index, which comprises 15 items rated on a 7-point scale (1 = worst rating, 7 = best rating) with a possible maximum score of 105. The highest score indicates the highest satisfaction with the impact of treatment on social factors. The 15 items were summarized to 4 scales: treatment interference, therapy-related problems, therapy setting, and treatment costs. The raw scores for these scales were transformed to a score ranging from 0 to 100, with 100 being the best score achievable.
Measured: At baseline and week 25
Overall Health Status at Baseline and Week 25
Overall Health Status was assessed using a Visual Analogue Scale (VAS). Patients were asked to rate their overall health status by placing a mark on a 100 mm VAS, with 0 being the worst imaginable state and 100 being the best imaginable state.
Measured: Baseline and week 25
Number of Patients With Adverse Events (AEs) by Severity and Relatedness
Included all AEs that occurred during the entire study period.
Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Measured: For the duration of the study, up to Week 25
Rate of AEs by Severity and Relatedness
The rate was the number of AEs over the number of infusions administered. Included all AEs that occurred during the entire study period.
Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Measured: For the duration of the study, up to Week 25
Number of Patients With Local/Injection Site Reactions
All AEs arising from local/injection site reactions.
Measured: For the duration of the study, up to Week 25
Number of Patients With Clinically Relevant Changes in Laboratory Parameters
Laboratory parameters included hematology, serum chemistry, and urinalysis parameters.
Measured: Baseline to Week 25
Number of Patients With Clinically Relevant Changes in Vital Signs
Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.
Measured: Baseline to Week 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2008 (estimated)
- Study startNov 2007
- Primary completionJan 2009
- Study completionJan 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Multifocal Motor Neuropathy (MMN)Studies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthias Sturzenegger, MD, Principal investigator, Inselspital, University Hospital of BernBernd Kieseier, MD, Principal investigator, Neurologische Klinik, Heinrich-Heine-University, DüsseldorfGiancarlo Comi, MD, Principal investigator, San Raffaele HospitalSiraj Misbah, MD, Principal investigator, Dept. Clinical Immunology, Oxford Radcliffe Hospitals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00701662
- Sponsor's protocol ID
- 1464
- Other IDs
- ZLB06_006CR, 2007-000710-37
- Status last verified
- Jun 2013
- First submitted
- 18 Jun 2008
- Last updated
- 1 Aug 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
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.
PubMed 21692906
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicentre Study of Subcutaneous Immunoglobulin (SCIG) in Patients With Multifocal Motor Neuropathy (MMN)
- ClinicalTrials.gov ID
- NCT00701662
- Acronym / sponsor ID
- 1464
- Conditions
- Multifocal Motor Neuropathy (MMN)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Nov 2007
- Primary completion
- Jan 2009 (actual)
- Study completion
- Jan 2009 (actual)
- First posted
- 19 Jun 2008 (estimated)
- Results
- Posted 1 Aug 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vivaglobin | Experimental | Biological: VivaglobinVivaglobin® is a 16% (160 mg/mL) liquid formulation of human normal immunoglobulin for subcutaneous infusion. Subjects will receive weekly infusions of Vivaglobin® at a weekly dosage calculated based on previous intravenous immunoglobulin treatment (between 0.1 to 0.5 g/kg body weight per week). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VivaglobinAlso: Human Normal Immunoglobulin | Biological | Vivaglobin |
Primary outcome
Change From Baseline to Week 24 in Muscle Strength
Time frame: Baseline to week 24
The change in Medical Research Council (MRC) score was determined at week 24 compared to baseline using descriptive statistics and nonparametric, two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available. The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement. Positive values for change in MRC sum score indicate improvement, with a more positive value indicating greater muscle contraction/ limb movement compared with the value at baseline.
Mean Overall MRC Score at Baseline and Week 24
Time frame: Baseline and week 24
The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement.
12 secondary outcomes
Change From Baseline to Week 24 in Disability
Time frame: Baseline to week 24
Mean Disability Score at Baseline and Week 24
Time frame: Baseline and Week 24
Change From Baseline to the Completion Visit in Motor Function
Time frame: Baseline to the completion visit (up to week 25)
Mean Motor Function Score at Screening and Week 25
Time frame: Screening and week 25
Health-Related Quality of Life at Baseline and Week 25
Time frame: At baseline and week 25
Treatment Satisfaction at Baseline and Week 25
Time frame: At baseline and week 25
Overall Health Status at Baseline and Week 25
Time frame: Baseline and week 25
Number of Patients With Adverse Events (AEs) by Severity and Relatedness
Time frame: For the duration of the study, up to Week 25
Rate of AEs by Severity and Relatedness
Time frame: For the duration of the study, up to Week 25
Number of Patients With Local/Injection Site Reactions
Time frame: For the duration of the study, up to Week 25
Number of Patients With Clinically Relevant Changes in Laboratory Parameters
Time frame: Baseline to Week 25
Number of Patients With Clinically Relevant Changes in Vital Signs
Time frame: Baseline to Week 25
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publicationMisbah 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.
- Results publication: PubMed 21692906Misbah 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Matthias Sturzenegger, MD principal investigator
Inselspital, University Hospital of Bern
Bernd Kieseier, MD principal investigator
Neurologische Klinik, Heinrich-Heine-University, Düsseldorf
Giancarlo Comi, MD principal investigator
San Raffaele Hospital
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vivaglobin in people with Multifocal Motor Neuropathy (MMN), run by CSL Behring at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy, Switzerland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Aug 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline to Week 24 in Muscle Strength (time frame: Baseline to week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00701662, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Aug 2013.