Trial record 31 of 52 for:    phenylbutyrate

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01881984
Recruitment Status : Completed
First Posted : June 20, 2013
Results First Posted : July 11, 2017
Last Update Posted : September 25, 2017
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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups

Open Label Study

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

Participant Flow:   Overall Study

  Baseline Characteristics

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

Open Label Study

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

Baseline Measures
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
Count of Participants
<=18 years      0   0.0% 
Between 18 and 65 years      4 100.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      4 100.0% 
Male      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of 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 
[Units: Participants]
United States   4 

  Outcome Measures

1.  Primary:   Metabolic Stress   [ Time Frame: 7 weeks ]

2.  Secondary:   Pharmacokinetic (pK)Analysis   [ Time Frame: 7 weeks ]

  Serious Adverse Events

  Other Adverse Events

  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
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.

  More Information

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 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