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Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

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ClinicalTrials.gov Identifier: NCT00750867
Recruitment Status : Completed
First Posted : September 11, 2008
Results First Posted : October 26, 2015
Last Update Posted : February 23, 2017
Sponsor:
Information provided by (Responsible Party):
Peter Novak, University of Massachusetts, Worcester

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Multiple System Atrophy
Intervention Drug: intravenous immunoglobulin (IVIg)
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Open Label Interventional Arm
Hide Arm/Group Description intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
Period Title: Overall Study
Started 9
Completed 7
Not Completed 2
Reason Not Completed
Adverse Event             2
Arm/Group Title Open Label Interventional Arm
Hide Arm/Group Description intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 9 participants
59
(55 to 64)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
0
   0.0%
Between 18 and 65 years
9
 100.0%
>=65 years
0
   0.0%
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
3
  33.3%
Male
6
  66.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
1.Primary Outcome
Title Number of Adverse Events up to Six Months Post-treatment
Hide Description The primary outcome measure was to evaluate the safety and tolerability of the IVIG infusions in patients with multiple system atrophy. The primary endpoint was defined as the frequency of adverse events (AE). AEs including their severity and relationship to the IVIG were assessed throughout the study and at least 60 days after the last infusion. The AEs were considered to be related to the IVIG infusion (infusional AE) if they occurred during an infusion or within 72 hours afterwards. Non-infusional AEs were further classified as possible related to IVIG or likely not related to IVIG. Serious AEs assessed include medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization. Any AE was defined as occurrence of any symptom regardless of intensity grade.
Time Frame Monthly, up to 8 months (including the screening visit and the final visit)
Hide Outcome Measure Data
Hide Analysis Population Description
Two participants dropped out from the study.
Arm/Group Title Open Label Interventional Arm
Hide Arm/Group Description:
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Adverse events
All Adverse Events 43
Serious Adverse Events 0
2.Secondary Outcome
Title Preliminary Efficacy of IVIg for Treatment of MSA.
Hide Description The secondary outcome measure was to evaluate the preliminary efficacy of IVIG for the treatment of MSA. The primary efficacy endpoint was change of the Unified MSA Rating Scale (UMSARS-I and UMSAR-II) compared to baseline. UMSARS-I and UMSARS-II are validated semiquantitative rating scales for evaluation of severity of MSA. UMSARS-I comprises a historical review of disease-related impairments and UMSARS-II comprises motor examination. UMSARS-I has 12 questions, each with assigned score 0-4, where 0 is normal and > are abnormal responses. Total range of UMSARS-I is 0 to 48. UMSARS-II has 12 items rated by an examiner, each with assigned score 0-4, where 0 is normal and > are abnormal responses. Total range of UMSARS-II is 0 to 56. The scores of UMSARS-I and UMSARS-II at baseline (month 1) was compared with the scores obtained at the final visit (month 8) which was 8 months apart. The interventions occured at months 2-7, total six times.
Time Frame Monthly, up to 8 months (including the screening visit and the final visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open Label Interventional Arm
Hide Arm/Group Description:
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
UMSARS-I, baseline 23.8  (6)
UMSARS-II,baseline 26.1  (7.4)
UMSARS-I, final visit 19  (5.9)
UMSARS-II, final visit 23.3  (7.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Open Label Interventional Arm
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0128
Comments The P-Value was obtained by comparing the UMSARS-I scores at final visit and at baseline.
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Open Label Interventional Arm
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.025
Comments The P-Value was obtained by comparing the UMSARS-II scores at final visit and at baseline.
Method ANOVA
Comments [Not Specified]
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Open Label Interventional Arm
Hide Arm/Group Description intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
All-Cause Mortality
Open Label Interventional Arm
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Open Label Interventional Arm
Affected / at Risk (%) # Events
Total   0/9 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Open Label Interventional Arm
Affected / at Risk (%) # Events
Total   9/9 (100.00%)    
Endocrine disorders   
low potassium  [1]  1/9 (11.11%)  1
Gastrointestinal disorders   
viral infection of gastrointenstinal tract  [2]  1/9 (11.11%)  1
Infections and infestations   
urinary tract infection  [3]  1/9 (11.11%)  1
Musculoskeletal and connective tissue disorders   
restless leg syndrome  [4]  1/9 (11.11%)  1
wrist strain  [2]  1/9 (11.11%)  1
Renal and urinary disorders   
decreased GFR  [2]  2/9 (22.22%)  2
elevated BUN  [2]  1/9 (11.11%)  1
Reproductive system and breast disorders   
elevated PSA  [2]  1/9 (11.11%)  1
Respiratory, thoracic and mediastinal disorders   
Accidental injury  [5]  1/9 (11.11%)  1
nodular lung abnormality  [6]  1/9 (11.11%)  1
worsening of sleep apnea  [7]  1/9 (11.11%)  1
Skin and subcutaneous tissue disorders   
pruritic skin rash  [8]  3/9 (33.33%)  4
increased temperature or skin flushing  [9]  1/9 (11.11%)  5
worsening of allergies   2/9 (22.22%)  2
Vascular disorders   
elevated blood pressure  [10]  9/9 (100.00%)  32
ankle edema  [11]  1/9 (11.11%)  1
Indicates events were collected by systematic assessment
[1]
treated with potassium supplements
[2]
resolved
[3]
treated with antibiotics
[4]
treated with iron supplements
[5]
nose fracture
[6]
later determined to be a tangle of vein most likely inborn
[7]
treated with continuous positive airway pressure (CPAP)
[8]
2 subjects withdrew from the study.
[9]
treated with Diphenhydramine, Acetaminophen, and slowing the infusion rate
[10]
transient, responding to adjusting the infusion rate
[11]
treated with diuretics
The main study limitation is its small size and open label character.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr.Peter Novak
Organization: University of Massachusetts Medical School
Phone: 508-334-4973
Responsible Party: Peter Novak, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier: NCT00750867     History of Changes
Other Study ID Numbers: H-12784
First Submitted: September 9, 2008
First Posted: September 11, 2008
Results First Submitted: December 26, 2013
Results First Posted: October 26, 2015
Last Update Posted: February 23, 2017