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Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

Primary Purpose

Multiple System Atrophy

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
intravenous immunoglobulin (IVIg)
Sponsored by
University of Massachusetts, Worcester
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Multiple System Atrophy focused on measuring multiple system atrophy, MSA,, intravenous immunoglobulin, IVIg

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female older than 17 years.
  2. Clinical diagnosis of probable multiple system atrophy
  3. Provide written informed consent to participate in the study
  4. Understand that they may withdraw their consent at any time

Exclusion Criteria:

  1. Women who are pregnant or lactating
  2. In the investigator's opinion, have any other significant systemic, hepatic, cardiac or renal illness.
  3. In the investigator's opinion, the subjects are significantly dehydrated, as determined by clinical evaluation including measurement of skin turgor, blood urea nitrogen and creatinine values.

Sites / Locations

  • University of Massachusetts Medical School

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

1

Arm Description

Interventions included monthly infusions of intravenous immunoglobulin.

Outcomes

Primary Outcome Measures

Number of Adverse Events up to Six Months Post-treatment
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.

Secondary Outcome Measures

Preliminary Efficacy of IVIg for Treatment of MSA.
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.

Full Information

First Posted
September 9, 2008
Last Updated
January 5, 2017
Sponsor
University of Massachusetts, Worcester
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1. Study Identification

Unique Protocol Identification Number
NCT00750867
Brief Title
Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Official Title
Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Study Type
Interventional

2. Study Status

Record Verification Date
September 2015
Overall Recruitment Status
Completed
Study Start Date
June 2008 (undefined)
Primary Completion Date
September 2009 (Actual)
Study Completion Date
December 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Massachusetts, Worcester

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Multiple System Atrophy (MSA) is a progressive sporadic neurodegenerative disorder leading to widespread loss of brain cells that results in parkinsonian, cerebellar and autonomic dysfunction. The cause of the MSA remains unclear. Available treatment is symptomatic only and does not alter the course of disease. Although the cause of MSA remains unclear, there is evidence of presence of common neuroinflammatory mechanisms in the MSA brains including activation of microglia and production of toxic cytokines. This research protocol is based on hypothesis that the MSA progression can be altered by blocking the neuroinflammatory activity. This protocol includes administration of intravenous immunoglobulin (IVIg). IVIg contains antibodies derived from human plasma which can block the inflammatory responses in the brain that can lead to loss of brain cells.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple System Atrophy
Keywords
multiple system atrophy, MSA,, intravenous immunoglobulin, IVIg

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Description
Interventions included monthly infusions of intravenous immunoglobulin.
Intervention Type
Drug
Intervention Name(s)
intravenous immunoglobulin (IVIg)
Other Intervention Name(s)
Privigen
Intervention Description
The intravenous immunoglobulin (brand Privigen) will be infused intravenously, monthly, 6 times, for 6 months the dose will be 0.4 gram/kg for each infusion.
Primary Outcome Measure Information:
Title
Number of Adverse Events up to Six Months Post-treatment
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)
Secondary Outcome Measure Information:
Title
Preliminary Efficacy of IVIg for Treatment of MSA.
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female older than 17 years. Clinical diagnosis of probable multiple system atrophy Provide written informed consent to participate in the study Understand that they may withdraw their consent at any time Exclusion Criteria: Women who are pregnant or lactating In the investigator's opinion, have any other significant systemic, hepatic, cardiac or renal illness. In the investigator's opinion, the subjects are significantly dehydrated, as determined by clinical evaluation including measurement of skin turgor, blood urea nitrogen and creatinine values.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter Novak, MD, PhD
Organizational Affiliation
University of Massachusetts, Worcester
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Massachusetts Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
23116538
Citation
Novak P, Williams A, Ravin P, Zurkiya O, Abduljalil A, Novak V. Treatment of multiple system atrophy using intravenous immunoglobulin. BMC Neurol. 2012 Nov 1;12:131. doi: 10.1186/1471-2377-12-131.
Results Reference
result

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Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

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