Trial Outcomes & Findings for Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia (NCT NCT01212484)

Last Updated: 2016-03-28

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

12 participants

Primary outcome timeframe

4 weeks

Results posted on

2016-03-28

Participant Flow

All patients were recruited at the NYU medical center's Dysautonomia Center.

Patients with FD that complain of severe nausea will be screened.

Participant milestones

Participant milestones
Measure	Placebo (First), Carbidopa (Second) Subjects will be given placebo first (4 weeks), followed by carbidopa (4 weeks).	Carbidopa (First), Placebo (Second) Subjects will be given Carbidopa for a 4 week period followed by placebo for a 4 week period.
Carbidopa Titration Period (4 Weeks) STARTED	6	6
Carbidopa Titration Period (4 Weeks) COMPLETED	5	6
Carbidopa Titration Period (4 Weeks) NOT COMPLETED	1	0
First Intervention (4 Weeks) STARTED	5	6
First Intervention (4 Weeks) COMPLETED	5	4
First Intervention (4 Weeks) NOT COMPLETED	0	2
Second Intervention (4 Weeks) STARTED	5	4
Second Intervention (4 Weeks) COMPLETED	5	3
Second Intervention (4 Weeks) NOT COMPLETED	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Study Subjects n=12 Participants
Age, Categorical <=18 years	8 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	18.5 years STANDARD_DEVIATION 6.45 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

Outcome measures

Outcome measures
Measure	Carbidopa n=8 Participants	Placebo n=8 Participants
Composite Daily Score	6.9 units on a scale Standard Deviation 2.2	9.7 units on a scale Standard Deviation 2.5

PRIMARY outcome

Timeframe: 4 weeks

Assay of 24 hour dopamine level excretion in urine

Outcome measures

Outcome measures
Measure	Carbidopa n=8 Participants	Placebo n=8 Participants
24 Hour Dopamine Levels	127 ug/gCR Standard Deviation 29	222 ug/gCR Standard Deviation 41

SECONDARY outcome

Timeframe: 4 weeks

Outcome measures

Outcome measures
Measure	Carbidopa n=8 Participants	Placebo n=8 Participants
Number of Episodes of Daily Nausea	1.2 episodes of nausea Standard Deviation 0.8	2.0 episodes of nausea Standard Deviation 1.0

Adverse Events

Carbidopa

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Carbidopa n=12 participants at risk 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 n=12 participants at risk 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.
Nervous system disorders lightheadedness / faint	8.3% 1/12 • Number of events 1	0.00% 0/12

Other adverse events

Adverse event data not reported

Additional Information

Horacio Kaufmann, MD; director of Dysautonomia Center

NYU Medical Center

Phone: 212 263 7225

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place