Phase 3
N=44
Study of the Effectiveness of Intravenous Immune Globulin (10%) for the Treatment of Multifocal Motor Neuropathy
Multifocal Motor Neuropathy
Bottom Line
View on ClinicalTrials.gov: NCT00666263 ↗Enrolled (actual)
44
Serious AEs
2.3%
Results posted
Mar 2013
Primary outcome: Primary: Grip Strength in the More Affected Hand — 18.14; 21.68; 19.54; 19.39 kilograms
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Immune Globulin Intravenous (human), 10% (Biological); 0.25% human albumin solution (Placebo) (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Aug 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Grip Strength in the More Affected Hand |
18.14; 21.68; 19.54; 19.39; 11.28; 17.77 | — |
| PRIMARY Mean Relative Change in Grip Strength in the More Affected Hand |
-16.36; -30.52; -30.11; 23.86 | 0.005 sig |
| PRIMARY Co-Primary Endpoint: Guy's Neurologic Disability Scale (GNDS) for Upper Limbs |
2.0; 2.0; 2.0; 2.0; 2.5; 2.0 | — |
| PRIMARY Co-Primary Endpoint: Proportion of Participants With Deterioration in Guy's Neurological Disability Score (GNDS) |
4.8; 35.7; 11.9; 47.6 | 0.021 sig |
| PRIMARY Rate of Temporally Associated Adverse Events (AEs) Per Infusion |
11.5; 13.2; 24.6; 11.6 | — |
| PRIMARY The Percentage of Participants for Whom the Infusion Rate of Any Infusion Was Reduced and/or the Infusion Was Interrupted or Stopped for Any Reason |
9.1; 0.0; 4.8; 4.8 | — |
| PRIMARY The Percentage of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped for Any Reason |
2.9; 0.0; 1.6; 0.7 | — |
| PRIMARY The Percentage of Participants Reporting One or More Moderate or Severe AEs That Began During Infusion or Within 72 Hours of Completion of an Infusion |
4.5; 27.3; 19.0; 4.8 | — |
| SECONDARY Percentage of Participants With at Least a 30% Decline in Relative Grip Strength in the More Affected Hand (Measured Using a DynEx Digital Dynamometer) |
4.8; 42.9; 4.8; 47.6 | <0.001 sig |
| SECONDARY Grip Strength in the Less Affected Hand |
27.98; 29.52; 29.79; 29.17; 26.58; 28.97 | — |
| SECONDARY Mean Relative Change in Grip Strength in the Less Affected Hand |
-2.52; -17.96; -29.22; 19.67 | <0.001 sig |
| SECONDARY Proportion of Participants That Were Accelerated Forward Into the Next Stabilization Phase (ie Switched to Open-Label IGIV, 10%) |
0.0; 69.0; 2.4; 28.6 | <0.001 sig |
| SECONDARY Patient Global Impression of Change |
4.0; 4.0; 4.0; 5.0; 2.0; 4.0 | — |
| SECONDARY Overall Disability Sum Score |
3; 2; 3; 2; 3; 2 | — |
| SECONDARY Overall Disability Sum Score - Standardized |
75.0; 83.3; 79.2; 83.3; 75.0; 83.3 | — |
| SECONDARY Mean Relative Change in Overall Disability Sum Score |
-3.14; -5.77; -8.46; 0.92 | 0.002 sig |
| SECONDARY Time Required by Participants to Complete the 9 Hole Peg Board Test (9-HPT) With the Dominant Hand |
20.75; 22.00; 20.25; 21.00; 20.50; 20.50 | — |
| SECONDARY Mean Relative Change in Time Required by Participants to Complete the 9 Hole Peg Board Test (9-HPT) With the Dominant Hand |
-2.57; 3.90; 29.89; 4.89 | <0.001 sig |
| SECONDARY Time Required by Participants to Complete the 9 Hole Peg Board Test (9-HPT) With the Non-Dominant Hand |
25.75; 22.50; 24.00; 23.50; 25.25; 21.00 | — |
| SECONDARY Mean Relative Change in Time Required by Participants to Complete the 9 Hole Peg Board Test (9-HPT) With the Non-Dominant Hand |
4.78; 13.06; 52.93; 8.56 | <0.001 sig |
| SECONDARY Participants' Assessment of Physical Functioning on a Visual Analog Scale (VAS) |
4.80; 2.95; 4.10; 3.50; 6.85; 4.50 | — |
| SECONDARY Mean Relative Change in Participants' Assessment of Physical Functioning on a Visual Analog Scale (VAS) |
140.92; 321.75; 258.09; 5.75 | 0.059 |
| SECONDARY Rate of Related AEs Per Infusion |
4.8; 20.6; 44.3; 15.9 | — |
| SECONDARY Rate of Related SAEs Per Infusion |
0.0; 0.0; 0.0; 0.7 | — |
| SECONDARY The Proportion of Participants for Whom the Infusion Rate of Any Infusion Was Reduced and/or the Infusion Was Interrupted or Stopped for Tolerability Concerns/AEs |
0.0; 0.0; 0.0; 4.8 | — |
| SECONDARY The Proportion of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped for Tolerability Concerns/AEs |
0.0; 0.0; 0.0; 0.7 | — |
| SECONDARY The Proportion of Infusions Associated With One or More AEs Related to the Study Product |
3.8; 19.1; 34.4; 13.0 | — |
Summary
The purpose of the study is to evaluate the efficacy (effect on grip strength and disability) and safety/tolerability of IGIV, 10% in subjects with Multifocal Motor Neuropathy.
Eligibility Criteria
Inclusion Criteria
- Written informed consent obtained from the participant prior to any study-related procedures and study product administration
- Diagnosis of definite or probable MMN based on the criteria of the American Association of Electrodiagnostic Medicine (AAEM) (Olney et al., 2003, see Section 15.1 for the full length publication). Conduction block can be identified by a drop in amplitude. Diagnosis can be based on chart records a) Hand grip (finger flexor) weakness of Medical Research Council (MRC) grade 4 or less at disease onset or appearing prior to screening; b) No upper motor signs c) No bulbar or cranial signs or symptoms; d) No clinically identifiable sensory abnormalities
- Must be on a stable regimen of IGIV for at least 3 months prior to first study product administration
- Treatment interval with IGIV of 2 to 5 weeks (+/- 3 days)
- Dose of IGIV to be 0.4 to 2.0 g per kg BW and infusion cycle
- Participants are adults, male or female, at least 18 years of age
- If female and capable of bearing children - have a negative urine pregnancy test result at enrollment and agree to employ adequate birth control measures for the duration of the study
- Ability and willingness to travel to the study site for infusions and assessments if required by the protocol
Exclusion Criteria
- Any clinical or electrophysiological evidence of coexisting neuropathy which may interfere with outcome assessments, such as diabetic neuropathy, toxic neuropathy, or neuropathy due to systemic lupus erythematosus
- Treatment with other immunosuppressive agents besides IGIV, which has demonstrated efficacy in MMN such as cyclophosphamide during the 3 months prior to enrollment (or treatment with Rituximab during the 12 months prior to enrollment). Pre-study treatment with mycophenolate mofetil or azathioprine is permitted if the dose has been stable for 3 months prior to enrollment.
- Cerebrospinal fluid protein > 100 mg/dL (if done as part of a previous evaluation)
- Participants positive at enrollment for one or more of the following: Hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for Hepatitis C (HCV), PCR for human immunodeficiency virus (HIV) Type 1
- Participants with levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 times the upper limit of normal for the testing laboratory
- Participants with neutropenia (defined as an absolute neutrophil count [ANC]≤1000/mm^3)
- Participants with serum creatinine levels greater than 1.5 times the upper limit of normal for age and gender
- Participants with malignancy other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix
- Participants with a history of thrombotic episodes (deep vein thrombosis, myocardial infarction, cerebrovascular accident)
- Participants who received any blood or blood product exposure other than an IGIV, subcutaneous immunoglobulin, immune serum globulin (ISG) preparation, or albumin within the 6 months prior to enrollment
- Participants with an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IGIV or human albumin
- Participants with immunoglobulin A (IgA) deficiency and known anti IgA antibodies
- Participants using another investigational product or device within 30 days prior to enrollment
- Participants who are unable or unwilling to meet all the requirements of this study
- If female, is pregnant or lactating at time of enrollment
Data sourced from ClinicalTrials.gov (NCT00666263). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.