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Trial record 36 of 103 for:    Gaucher Disease

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

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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Groups were not assigned in this study. All subjects received the same intervention.

Reporting Groups
  Description
NAC in PD single intravenous administration of N-acetylcysteine in PD patients
NAC in GD single intravenous administration of N-acetylcysteine in GD patients
NAC in Controls single intravenous administration of N-acetylcysteine in control subjects

Participant Flow:   Overall Study
    NAC in PD   NAC in GD   NAC in Controls
STARTED   3   3   3 
COMPLETED   3   3   3 
NOT COMPLETED   0   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
NAC in PD single intravenous administration of N-acetylcysteine in PD patients
NAC in GD single intravenous administration of N-acetylcysteine in GD patients
NAC in Controls single intravenous administration of N-acetylcysteine in control subjects
Total Total of all reporting groups

Baseline Measures
   NAC in PD   NAC in GD   NAC in Controls   Total 
Overall Participants Analyzed 
[Units: Participants]
 3   3   3   9 
Age 
[Units: Participants]
       
<=18 years   0   1   0   1 
Between 18 and 65 years   3   2   3   8 
>=65 years   0   0   0   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 54.33  (3.21)   44  (23.58)   58  (0)   52.11  (13.46) 
Gender 
[Units: Participants]
       
Female   3   0   2   5 
Male   0   3   1   4 
Region of Enrollment 
[Units: Participants]
       
United States   3   3   3   9 


  Outcome Measures

1.  Primary:   Brain GSH   [ Time Frame: Baseline and up to 110 minutes post-NAC administration ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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
e-mail: holma101@umn.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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