Trial Outcomes & Findings for Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation (NCT NCT01881984)

NCT ID: NCT01881984

Last Updated: 2017-09-25

Results Overview

Changes in the assessments of metabolic stress pre- and post-dosing with Ravicti will be the main outcome variable.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 4 participants
• Primary outcome timeframe: 7 weeks
• Results posted on: 2017-09-25

Participant Flow

Participant milestones

Measure	Ravicti Open Label Study Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

Baseline characteristics by cohort

Measure	Ravicti n=4 Participants Open Label Study Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day
Age, Categorical <=18 years	0 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
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 7 weeks

Population: Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality.

Changes in the assessments of metabolic stress pre- and post-dosing with Ravicti will be the main outcome variable.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 7 weeks

Population: Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality.

Results from the pharmacokinetic (pK)analysis (the rate of conversion of the phenylbutyrate to phenylacetate) will also be reviewed to assess for changes pre- and post-dosing with Ravicti as well as changes in these levels at the different doses of Ravicti.

Outcome measures

Outcome data not reported

Adverse Events

Ravicti

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Ravicti n=4 participants at risk Open Label Study Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day
Gastrointestinal disorders Dry mouth	25.0% 1/4 • Number of events 1 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Investigations Elevated PBA level	50.0% 2/4 • Number of events 2 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Gastrointestinal disorders Nausea	50.0% 2/4 • Number of events 2 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Gastrointestinal disorders Vomiting	25.0% 1/4 • Number of events 1 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Musculoskeletal and connective tissue disorders Neck pain	25.0% 1/4 • Number of events 1 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Vascular disorders Thromboembolic event	25.0% 1/4 • Number of events 1 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
Nervous system disorders Decreased reflexes	25.0% 1/4 • Number of events 1 • Approximately 7 weeks The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.

Additional Information

Dr. Gerard Vockley

University of Pittsburgh

Phone: 412-692-7746

Email: gerard.vockley@chp.edu

Results disclosure agreements

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