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Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia

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ClinicalTrials.gov Identifier: NCT01212484
Recruitment Status : Completed
First Posted : September 30, 2010
Results First Posted : March 28, 2016
Last Update Posted : March 28, 2016
Sponsor:
Information provided by (Responsible Party):
New York University School of Medicine

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Familial Dysautonomia
Interventions Drug: Carbidopa
Drug: Placebo
Enrollment 12
Recruitment Details All patients were recruited at the NYU medical center's Dysautonomia Center.
Pre-assignment Details Patients with FD that complain of severe nausea will be screened.
Arm/Group Title Placebo (First), Carbidopa (Second) Carbidopa (First), Placebo (Second)
Hide Arm/Group Description Subjects will be given placebo first (4 weeks), followed by carbidopa (4 weeks). Subjects will be given Carbidopa for a 4 week period followed by placebo for a 4 week period.
Period Title: Carbidopa Titration Period (4 Weeks)
Started 6 6
Completed 5 6
Not Completed 1 0
Period Title: First Intervention (4 Weeks)
Started 5 6
Completed 5 4
Not Completed 0 2
Period Title: Second Intervention (4 Weeks)
Started 5 4
Completed 5 3
Not Completed 0 1
Arm/Group Title All Study Subjects
Hide Arm/Group Description [Not Specified]
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
<=18 years
8
  66.7%
Between 18 and 65 years
4
  33.3%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants
18.5  (6.45)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
4
  33.3%
Male
8
  66.7%
1.Primary Outcome
Title Composite Daily Score
Hide Description Daily scores were reported on a modified version of the Rhodes Index of Nausea, Vomiting and Retching, which included all 5 items relating to nausea and retching. Items addressing vomiting/throwing up were omitted, as all participants had antireflux surgery that prevented vomiting (Nissen fundoplication). Retching distress, nausea distress, number of nausea episodes per day, number of retching episodes per day, and the amount of time spent feeling nauseous were graded on a 5-point scale. Scores range from 0 (no nausea/distress) to 20 (most nausea/distress).
Time Frame 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Carbidopa Placebo
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
6.9  (2.2) 9.7  (2.5)
2.Primary Outcome
Title 24 Hour Dopamine Levels
Hide Description Assay of 24 hour dopamine level excretion in urine
Time Frame 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Carbidopa Placebo
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: ug/gCR
127  (29) 222  (41)
3.Secondary Outcome
Title Number of Episodes of Daily Nausea
Hide Description [Not Specified]
Time Frame 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Carbidopa Placebo
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: episodes of nausea
1.2  (0.8) 2.0  (1.0)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Carbidopa Placebo
Hide Arm/Group Description

carbidopa

Carbidopa : The trial will be divided into two consecutive, but independent parts. Phase 1, will address the safety and tolerability of carbidopa in patients with FD using an open-label dose titration phase followed by 4-weeks of open-label treatment. Phase 2, will address the efficacy of carbidopa for the treatment of nausea in patients with FD using a randomized, placebo controlled, double blind, 4-week cross over design.

Placebo

Placebo : The trial will be divided into two consecutive, but independent parts. Phase 1, will address the safety and tolerability of carbidopa in patients with FD using an open-label dose titration phase followed by 4-weeks of open-label treatment. Phase 2, will address the efficacy of carbidopa for the treatment of nausea in patients with FD using a randomized, placebo controlled, double blind, 4-week cross over design.

All-Cause Mortality
Carbidopa Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Carbidopa Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/12 (8.33%)      0/12 (0.00%)    
Nervous system disorders     
lightheadedness / faint  [1]  1/12 (8.33%)  1 0/12 (0.00%)  0
Indicates events were collected by systematic assessment
[1]
Patient reported feeling lightheaded and fainted. Blood results indicated patient was anemic and volume depleted, a common feature of FD patients. Patient had a history of dehydration. Patient was hospitalized and recovered without sequelae.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Carbidopa Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/12 (0.00%)      0/12 (0.00%)    
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Horacio Kaufmann, MD; director of Dysautonomia Center
Organization: NYU Medical Center
Phone: 212 263 7225
Responsible Party: New York University School of Medicine
ClinicalTrials.gov Identifier: NCT01212484     History of Changes
Other Study ID Numbers: 09-0011
First Submitted: September 13, 2010
First Posted: September 30, 2010
Results First Submitted: December 29, 2014
Results First Posted: March 28, 2016
Last Update Posted: March 28, 2016