Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

This study has been completed.
Information provided by (Responsible Party):
Peter Novak, University of Massachusetts, Worcester Identifier:
First received: September 9, 2008
Last updated: September 28, 2015
Last verified: September 2015

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.

Condition Intervention Phase
Multiple System Atrophy
Drug: intravenous immunoglobulin (IVIg)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

Resource links provided by NLM:

Further study details as provided by University of Massachusetts, Worcester:

Primary Outcome Measures:
  • Number of Adverse Events up to Six Months Post-treatment [ Time Frame: Monthly, up to 8 months (including the screening visit and the final visit) ] [ Designated as safety issue: Yes ]
    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. [ Time Frame: Monthly, up to 8 months (including the screening visit and the final visit) ] [ Designated as safety issue: No ]
    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.

Enrollment: 9
Study Start Date: June 2008
Study Completion Date: December 2012
Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Interventions included monthly infusions of intravenous immunoglobulin.
Drug: intravenous immunoglobulin (IVIg)
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.
Other Name: Privigen


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00750867

United States, Massachusetts
University of Massachusetts Medical School
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
University of Massachusetts, Worcester
Principal Investigator: Peter Novak, MD, PhD University of Massachusetts, Worcester
  More Information

Responsible Party: Peter Novak, Study Principle Investigator, University of Massachusetts, Worcester Identifier: NCT00750867     History of Changes
Other Study ID Numbers: H-12784
Study First Received: September 9, 2008
Results First Received: December 26, 2013
Last Updated: September 28, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Massachusetts, Worcester:
multiple system atrophy, MSA,
intravenous immunoglobulin, IVIg

Additional relevant MeSH terms:
Multiple System Atrophy
Shy-Drager Syndrome
Autonomic Nervous System Diseases
Central Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
Brain Diseases
Cardiovascular Diseases
Movement Disorders
Neurodegenerative Diseases
Pathological Conditions, Anatomical
Primary Dysautonomias
Vascular Diseases
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs processed this record on December 01, 2015