Trial Outcomes & Findings for Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins (NCT NCT00750867)
NCT ID: NCT00750867
Last Updated: 2017-02-23
Results Overview
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.
COMPLETED
PHASE2
9 participants
Monthly, up to 8 months (including the screening visit and the final visit)
2017-02-23
Participant Flow
Participant milestones
| Measure |
Open Label Interventional Arm
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Open Label Interventional Arm
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
Baseline Characteristics
Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Baseline characteristics by cohort
| Measure |
Open Label Interventional Arm
n=9 Participants
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Age, Continuous
|
59 years
n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Gender
Female
|
3 Participants
n=5 Participants
|
|
Gender
Male
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Monthly, up to 8 months (including the screening visit and the final visit)Population: Two participants dropped out from the study.
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.
Outcome measures
| Measure |
Open Label Interventional Arm
n=7 Participants
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Number of Adverse Events up to Six Months Post-treatment
All Adverse Events
|
43 Adverse events
|
|
Number of Adverse Events up to Six Months Post-treatment
Serious Adverse Events
|
0 Adverse events
|
SECONDARY outcome
Timeframe: Monthly, up to 8 months (including the screening visit and the final visit)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.
Outcome measures
| Measure |
Open Label Interventional Arm
n=7 Participants
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Preliminary Efficacy of IVIg for Treatment of MSA.
UMSARS-I, baseline
|
23.8 units on a scale
Standard Deviation 6
|
|
Preliminary Efficacy of IVIg for Treatment of MSA.
UMSARS-II,baseline
|
26.1 units on a scale
Standard Deviation 7.4
|
|
Preliminary Efficacy of IVIg for Treatment of MSA.
UMSARS-I, final visit
|
19 units on a scale
Standard Deviation 5.9
|
|
Preliminary Efficacy of IVIg for Treatment of MSA.
UMSARS-II, final visit
|
23.3 units on a scale
Standard Deviation 7.2
|
Adverse Events
Open Label Interventional Arm
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Open Label Interventional Arm
n=9 participants at risk
intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.
|
|---|---|
|
Vascular disorders
elevated blood pressure
|
100.0%
9/9 • Number of events 32 • 1 year
|
|
Infections and infestations
urinary tract infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Accidental injury
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
pruritic skin rash
|
33.3%
3/9 • Number of events 4 • 1 year
|
|
Skin and subcutaneous tissue disorders
increased temperature or skin flushing
|
11.1%
1/9 • Number of events 5 • 1 year
|
|
Musculoskeletal and connective tissue disorders
restless leg syndrome
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
decreased GFR
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Vascular disorders
ankle edema
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
elevated BUN
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
worsening of allergies
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
nodular lung abnormality
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Endocrine disorders
low potassium
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
viral infection of gastrointenstinal tract
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Reproductive system and breast disorders
elevated PSA
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
wrist strain
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
worsening of sleep apnea
|
11.1%
1/9 • Number of events 1 • 1 year
|
Additional Information
Dr.Peter Novak
University of Massachusetts Medical School
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place