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

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ClinicalTrials.gov Identifier: NCT01881984
Recruitment Status : Completed
First Posted : June 20, 2013
Results First Posted : July 11, 2017
Last Update Posted : September 25, 2017
Sponsor:
Collaborator:
Horizon Pharma Ireland, Ltd., Dublin Ireland
Information provided by (Responsible Party):
Gerard Vockley, MD, PhD, University of Pittsburgh

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Intervention Drug: Ravicti
Enrollment 4

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ravicti
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Open Label Study

Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

Period Title: Overall Study
Started 4
Completed 4
Not Completed 0
Arm/Group Title Ravicti
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Open Label Study

Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
4
 100.0%
Male
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
4
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants
4
1.Primary Outcome
Title Metabolic Stress
Hide Description Changes in the assessments of metabolic stress pre- and post-dosing with Ravicti will be the main outcome variable.
Time Frame 7 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality.
Arm/Group Title Ravicti
Hide Arm/Group Description:

Open Label Study

Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Pharmacokinetic (pK)Analysis
Hide Description 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.
Time Frame 7 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality.
Arm/Group Title Ravicti
Hide Arm/Group Description:

Open Label Study

Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Approximately 7 weeks
Adverse Event Reporting Description The definition of adverse event and a serious adverse event used to collect this information did not differ from the clinicaltrials.gov definition.
 
Arm/Group Title Ravicti
Hide Arm/Group Description

Open Label Study

Ravicti: Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

All-Cause Mortality
Ravicti
Affected / at Risk (%)
Total   0/4 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Ravicti
Affected / at Risk (%) # Events
Total   0/4 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ravicti
Affected / at Risk (%) # Events
Total   3/4 (75.00%)    
Gastrointestinal disorders   
Dry mouth  1  1/4 (25.00%)  1
Nausea  1  2/4 (50.00%)  2
Vomiting  1  1/4 (25.00%)  1
Investigations   
Elevated PBA level  1  2/4 (50.00%)  2
Musculoskeletal and connective tissue disorders   
Neck pain  1  1/4 (25.00%)  1
Nervous system disorders   
Decreased reflexes  1  1/4 (25.00%)  1
Vascular disorders   
Thromboembolic event  1  1/4 (25.00%)  1
1
Term from vocabulary, CTCAE (4.03)
Indicates events were collected by systematic assessment
The purpose of this phase 1 trial was to establish a safe dose for treatment of patients with MCAD deficiency due to the common mutations, and to evaluate biomarkers of fatty acid metabolism in treated patients vs their pretreatment values.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Gerard Vockley
Organization: University of Pittsburgh
Phone: 412-692-7746
Responsible Party: Gerard Vockley, MD, PhD, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT01881984     History of Changes
Other Study ID Numbers: PRO13050530
First Submitted: June 13, 2013
First Posted: June 20, 2013
Results First Submitted: February 14, 2017
Results First Posted: July 11, 2017
Last Update Posted: September 25, 2017