Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

Multiple System Atrophy Clinical Trial

Official title:

Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00750867
• Other study ID #: H-12784
• Status: Completed
• Phase: Phase 2
• First received: September 9, 2008
• Last updated: September 28, 2015
• Start date: June 2008
• Est. completion date: December 2012

Study information

• Verified date: September 2015
• Source: University of Massachusetts, Worcester
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: December 2012
• Est. primary completion date: September 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Atrophy
• Multiple System Atrophy
• Shy-Drager Syndrome

Intervention

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.

Locations

Country	Name	City	State
United States	University of Massachusetts Medical School	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
University of Massachusetts, Worcester

Country where clinical trial is conducted

United States, 

References & 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	Yes
Secondary	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.	Monthly, up to 8 months (including the screening visit and the final visit)	No