Trial Outcomes & Findings for Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease (NCT NCT01427517)

NCT ID: NCT01427517

Last Updated: 2019-11-01

Results Overview

change in brain GSH levels from baseline to post-NAC administration (90 - 110 minutes) in all subjects

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 9 participants
• Primary outcome timeframe: Baseline and up to 110 minutes post-NAC administration
• Results posted on: 2019-11-01

Participant Flow

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.

Groups were not assigned in this study. All subjects received the same intervention.

Participant milestones

Measure	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
Overall Study STARTED	3	3	3
Overall Study COMPLETED	3	3	3
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	NAC in PD n=3 Participants single intravenous administration of N-acetylcysteine in PD patients	NAC in GD n=3 Participants single intravenous administration of N-acetylcysteine in GD patients	NAC in Controls n=3 Participants single intravenous administration of N-acetylcysteine in control subjects	Total n=9 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	54.33 years STANDARD_DEVIATION 3.21 • n=5 Participants	44 years STANDARD_DEVIATION 23.58 • n=7 Participants	58 years STANDARD_DEVIATION 0 • n=5 Participants	52.11 years STANDARD_DEVIATION 13.46 • n=4 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	5 Participants n=4 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	3 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants
Region of Enrollment United States	3 participants n=5 Participants	3 participants n=7 Participants	3 participants n=5 Participants	9 participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline and up to 110 minutes post-NAC administration

change in brain GSH levels from baseline to post-NAC administration (90 - 110 minutes) in all subjects

Outcome measures

Measure	NAC in PD n=3 Participants single intravenous administration of N-acetylcysteine in PD patients	NAC in GD n=3 Participants single intravenous administration of N-acetylcysteine in GD patients	NAC in Controls n=3 Participants single intravenous administration of N-acetylcysteine in control subjects
Brain GSH	55 percent increase from baseline Standard Deviation 24.4	41 percent increase from baseline Standard Deviation 16.3	34 percent increase from baseline Standard Deviation 19.8

Adverse Events

NAC in PD

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

NAC in GD

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

NAC in Controls

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	NAC in PD n=3 participants at risk single intravenous administration of N-acetylcysteine in PD patients	NAC in GD n=3 participants at risk single intravenous administration of N-acetylcysteine in GD patients	NAC in Controls n=3 participants at risk single intravenous administration of N-acetylcysteine in control subjects
Respiratory, thoracic and mediastinal disorders allergic reaction	33.3% 1/3 • Number of events 1	0.00% 0/3	0.00% 0/3

Additional Information

Mary Holmay

University of Minnesota

Phone: 612-812-5854

Email: holma101@umn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place