Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

NCT00750867 | Completed Phase 2 Results

• 1 other identifier: H-12784
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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.

Conditions

Multiple System Atrophy

Keywords

multiple system atrophy, MSA,; intravenous immunoglobulin, IVIg

Outcome Measures

Primary Outcomes (1)

• 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.

  Monthly, up to 8 months (including the screening visit and the final visit)

Secondary Outcomes (1)

• Preliminary Efficacy of IVIg for Treatment of MSA.

  Monthly, up to 8 months (including the screening visit and the final visit)

Study Arms (1)

1

EXPERIMENTAL

Interventions included monthly infusions of intravenous immunoglobulin.

Drug: intravenous immunoglobulin (IVIg)

Interventions

intravenous immunoglobulin (IVIg) DRUG

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.

Also known as: Privigen

1

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Massachusetts, Worcester: lead

Study Sites (1)

University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

Related Publications (1)

• 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.

  PMID: 23116538 RESULT

MeSH Terms

Conditions

Multiple System Atrophy; Thyroid Dyshormonogenesis 2A

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Limitations and Caveats

The main study limitation is its small size and open label character.

Results Point of Contact

• Title: Dr.Peter Novak
• Organization: University of Massachusetts Medical School

novakp@ummhc.org

508-334-4973

Study Officials

• Peter Novak, MD, PhD

  University of Massachusetts, Worcester

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Study Principle Investigator

Study Record Dates

• First Submitted: September 9, 2008
• First Posted: September 11, 2008
• Study Start: June 1, 2008
• Primary Completion: September 1, 2009
• Study Completion: December 1, 2012
• Last Updated: February 23, 2017
• Results First Posted: October 26, 2015

Record last verified: 2015-09

Locations

US (1)