Double-Blind, Placebo Controlled Pilot Study of Octanoic Acid in Essential Tremor

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Mark Hallett, National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier:
NCT00848172
First received: February 19, 2009
Last updated: November 22, 2012
Last verified: November 2012
Results First Received: August 6, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Essential Tremor
Interventions: Drug: Octanoic Acid
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects with a diagnosis of ET were recruited by referral from the Motor Control outpatient clinic, a listing on the NINDS web page, and from the general community.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
29 subjects (12f, 17m) were screened for eligibility. 10 subjects were considered screening failures due to the following reasons: failure to confirm ET according to diagnostic consensus criteria (n=7), other medical conditions precluding a safe participation (n=2), or the lack of objective alcohol-response (n=1). Nineteen subjects were randomized.

Reporting Groups
  Description
Sequence OA / Placebo During the 3-day inpatient Visit 2 (Drug Administration), after the admissions day (day 1 of Visit 2) patients randomized to Sequence OA / Placebo received a single oral dose of 4 mg/kg OA on the second day of Visit 2, and matching Placebo on the third day of Visit 2. Patients were discharged at the end of the third day of Visit 2, which ended this study visit.
Sequence Placebo / OA During the 3-day inpatient Visit 2 (Drug Administration), after the admissions day (day 1 of Visit 2) patients randomized to Sequence OA / Placebo received Placebo on the second day of Visit 2, and a single oral dose of 4 mg/kg OA on the third day of Visit 2. Patients were discharged at the end of the third day of Visit 2, which ended this study visit.

Participant Flow for 2 periods

Period 1:   Visit 2 - Drug Administration
    Sequence OA / Placebo     Sequence Placebo / OA  
STARTED     10     9  
COMPLETED     10     8  
NOT COMPLETED     0     1  

Period 2:   Visit 3 - Follow Up
    Sequence OA / Placebo     Sequence Placebo / OA  
STARTED     10     8  
COMPLETED     10     8  
NOT COMPLETED     0     0  



  Baseline Characteristics
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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
Sequence OA / Placebo First day: octanoic acid Second day: placebo
Sequence Placebo / OA First day: placebo Second day: octanoic acid
Total Total of all reporting groups

Baseline Measures
    Sequence OA / Placebo     Sequence Placebo / OA     Total  
Number of Participants  
[units: participants]
  10     9     19  
Age  
[units: participants]
     
<=18 years     0     0     0  
Between 18 and 65 years     6     4     10  
>=65 years     4     5     9  
Age  
[units: years]
Mean ± Standard Deviation
  59.90  ± 10.97     64.22  ± 8.60     61.95  ± 9.90  
Gender  
[units: participants]
     
Female     4     1     5  
Male     6     8     14  
Region of Enrollment  
[units: participants]
     
United States     10     9     19  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Normalized Accelerometric Tremor Power, Dominant Hand, 80min After Administration, Weighted Condition   [ Time Frame: 80 min after administration of the study drug on day 1 and 2 of Visit 2 ]

2.  Secondary:   Normalized Tremor Power, 300 Min After Administration, Weighted Condition, Dominant Hand, OA vs Placebo   [ Time Frame: 300 min post dose ]

3.  Secondary:   TMax Octanoic Acid   [ Time Frame: between 5 and 300 min post dose ]

4.  Secondary:   PK: AUC After OA   [ Time Frame: 5 to 300 min post dose ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


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.  


Results Point of Contact:  
Name/Title: Mark Hallett
Organization: NINDS
phone: 301-496-9526
e-mail: hallettm@ninds.nih.gov


Publications:

Responsible Party: Mark Hallett, National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier: NCT00848172     History of Changes
Other Study ID Numbers: 090084, 09-N-0084
Study First Received: February 19, 2009
Results First Received: August 6, 2012
Last Updated: November 22, 2012
Health Authority: United States: Food and Drug Administration