ClinicalTrials.gov
ClinicalTrials.gov Menu

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01427517
Recruitment Status : Completed
First Posted : September 1, 2011
Results First Posted : August 2, 2013
Last Update Posted : August 14, 2013
Sponsor:
Collaborators:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Science (NCATS)
National Institute of Neurological Disorders and Stroke (NINDS)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Parkinson's Disease
Gaucher's Disease
Intervention Drug: N-acetylcysteine
Enrollment 9

Recruitment Details Subjects were primarily recruited from the investigators' clinics at the University of Minnesota, and from research volunteers signed up with the University's Center for Magnetic Resonance Research.
Pre-assignment Details Groups were not assigned in this study. All subjects received the same intervention.
Arm/Group Title NAC in PD NAC in GD NAC in Controls
Hide Arm/Group Description single intravenous administration of N-acetylcysteine in PD patients single intravenous administration of N-acetylcysteine in GD patients single intravenous administration of N-acetylcysteine in control subjects
Period Title: Overall Study
Started 3 3 3
Completed 3 3 3
Not Completed 0 0 0
Arm/Group Title NAC in PD NAC in GD NAC in Controls Total
Hide Arm/Group Description single intravenous administration of N-acetylcysteine in PD patients single intravenous administration of N-acetylcysteine in GD patients single intravenous administration of N-acetylcysteine in control subjects Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
<=18 years
0
   0.0%
1
  33.3%
0
   0.0%
1
  11.1%
Between 18 and 65 years
3
 100.0%
2
  66.7%
3
 100.0%
8
  88.9%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 3 participants 9 participants
54.33  (3.21) 44  (23.58) 58  (0) 52.11  (13.46)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
Female
3
 100.0%
0
   0.0%
2
  66.7%
5
  55.6%
Male
0
   0.0%
3
 100.0%
1
  33.3%
4
  44.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants 3 participants 3 participants 9 participants
3 3 3 9
1.Primary Outcome
Title Brain GSH
Hide Description change in brain GSH levels from baseline to post-NAC administration (90 - 110 minutes) in all subjects
Time Frame Baseline and up to 110 minutes post-NAC administration
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title NAC in PD NAC in GD NAC in Controls
Hide Arm/Group Description:
single intravenous administration of N-acetylcysteine in PD patients
single intravenous administration of N-acetylcysteine in GD patients
single intravenous administration of N-acetylcysteine in control subjects
Overall Number of Participants Analyzed 3 3 3
Mean (Standard Deviation)
Unit of Measure: percent increase from baseline
55  (24.4) 41  (16.3) 34  (19.8)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title NAC in PD NAC in GD NAC in Controls
Hide Arm/Group Description single intravenous administration of N-acetylcysteine in PD patients single intravenous administration of N-acetylcysteine in GD patients single intravenous administration of N-acetylcysteine in control subjects
All-Cause Mortality
NAC in PD NAC in GD NAC in Controls
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
NAC in PD NAC in GD NAC in Controls
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/3 (0.00%)      0/3 (0.00%)      0/3 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
NAC in PD NAC in GD NAC in Controls
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/3 (33.33%)      0/3 (0.00%)      0/3 (0.00%)    
Respiratory, thoracic and mediastinal disorders       
allergic reaction  [1]  1/3 (33.33%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0
Indicates events were collected by systematic assessment
[1]
Following scan, a subject complained of itchy of hands, nasal congestion, sore throat. Study staff noted swelling around eyes and flushed cheeks & neck. Subject was taken to ER (not admitted)& received standard treatment for allergic reaction to NAC.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Mary Holmay
Organization: University of Minnesota
Phone: 612-812-5854
Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT01427517     History of Changes
Other Study ID Numbers: 6721
U54NS065768 ( U.S. NIH Grant/Contract )
First Submitted: August 30, 2011
First Posted: September 1, 2011
Results First Submitted: May 22, 2013
Results First Posted: August 2, 2013
Last Update Posted: August 14, 2013