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Phenylbutyrate Therapy for Maple Syrup Urine Disease (MSUD)

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ClinicalTrials.gov Identifier: NCT01529060
Recruitment Status : Completed
First Posted : February 8, 2012
Results First Posted : March 6, 2019
Last Update Posted : March 19, 2019
Sponsor:
Information provided by (Responsible Party):
Brendan Lee, Baylor College of Medicine

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Maple Syrup Urine Disease
Interventions Drug: Phenylbutyrate
Drug: Placebo powder
Enrollment 20
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Phenylbutyrate First and Placebo Second Group Placebo First and Phenylbutyrate Second Group
Hide Arm/Group Description Individuals randomized to this group received phenylbutyrate powder (500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in 3 divided doses per day) for 14 days (week 1 and week 2) and then crossed over to receive placebo powder in identical doses for the next 14 days (Week 3 and week 4) Individuals randomized to this group received first received placebo powder for 14 days (week 1 and week 2) and then were crossed over to receive phenylbutyrate (500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in 3 divided doses per day) for 14 days (week 3 and week 4) week 4).
Period Title: Overall Study
Started 10 10
Completed 10 10
Not Completed 0 0
Arm/Group Title Phenylbutyrate First and Placebo Second Placebo First and Phenylbutyrate Second Total
Hide Arm/Group Description Individuals randomized to this group received phenylbutyrate (500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in divided doses per day) for 14 days (week 1 and week 2) and then crossed over to receive placebo powder in identical doses for the next 14 days (Week 3 and week 4) Individuals randomized to this group received first received placebo powder for 14 days (week 1 and week 2) and then were crossed over to receive phenylbutyrate (500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in 3 divided doses per day) for 14 days (week 3 and week 4) week 4). Total of all reporting groups
Overall Number of Baseline Participants 10 10 20
Hide Baseline Analysis Population Description
Anaysis population is same as the the one depicted in Participant Flow
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants 10 participants 20 participants
23
(15 to 43)
23
(10 to 45)
23
(10 to 45)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
Female
7
  70.0%
4
  40.0%
11
  55.0%
Male
3
  30.0%
6
  60.0%
9
  45.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Ethnic Categories Number Analyzed 10 participants 10 participants 20 participants
Not Hispanic or Latino
8
  80.0%
9
  90.0%
17
  85.0%
Hispanic or Latino
2
  20.0%
1
  10.0%
3
  15.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
United States 9 10 19
Canada 1 0 1
1.Primary Outcome
Title 0-24 Hour AUC Leucine (Samples Collected at 0, 2, 4, 8, 12, 16, 20, and 24 Hours)
Hide Description Total Leucine exposure over 24 hours was calculated by serial blood draws at times 0, 2, 4, 8, 12, 16, 20, and 24 hours
Time Frame 24 Hours
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Phenylbutyrate Placebo First and Phenylbutyrate Second Group
Hide Arm/Group Description:
Cumulative results when individuals were treated with phenylbutyrate
Cumulative results from when individuals were treated with placebo
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: micromoles*hour/L
6217  (5168) 4616  (4106)
2.Primary Outcome
Title Leucine CMax 0-24 Hours
Hide Description Maximal leucine concentration in 0-24 hours
Time Frame 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Phenylbutyrate Placebo
Hide Arm/Group Description:
Cumulative results when individuals were treated with phenylbutyrate
Cumulative results when individuals were treated with placebo
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: Micromoles/L
361  (244) 295  (211)
Time Frame Adverse events were collected over the 14 days of Treatment period 1 and 14 days of treatment period 2
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Phenylbutyrate Inactive Powder
Hide Arm/Group Description

Study Drug

Phenylbutyrate: Dosage of phenylbutyrate powder will be 500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in four divided doses per day, the standard UCD dose studied in our preliminary studies, for 14 days.

Placebo powder

Placebo powder: Dosage of inactive placebo powder will be 500 mg/kg/day in patients weighing less than 20kg and 10 g/m2/day in larger patients in four divided doses per day for 14 days. Subjects will receive the same amount of powder for each arm of the study.

All-Cause Mortality
Phenylbutyrate Inactive Powder
Affected / at Risk (%) Affected / at Risk (%)
Total   0/20 (0.00%)      0/20 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Phenylbutyrate Inactive Powder
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/20 (0.00%)      0/20 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Phenylbutyrate Inactive Powder
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   19/20 (95.00%)      16/20 (80.00%)    
Blood and lymphatic system disorders     
Labs   19/20 (95.00%)  19 16/20 (80.00%)  16
Gastrointestinal disorders     
Gastrointestinal Symptoms   16/20 (80.00%)  16 7/20 (35.00%)  7
Musculoskeletal and connective tissue disorders     
Muscular Pain   4/20 (20.00%)  4 2/20 (10.00%)  2
Nervous system disorders     
Neurological Complaints   7/20 (35.00%)  7 7/20 (35.00%)  7
Renal and urinary disorders     
Urine Odor   1/20 (5.00%)  1 1/20 (5.00%)  1
Reproductive system and breast disorders     
Menstral Cramps   2/20 (10.00%)  2 0/20 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Oral or Nasal Complaints   15/20 (75.00%)  15 4/20 (20.00%)  4
Skin and subcutaneous tissue disorders     
Minor Skin Complaints   6/20 (30.00%)  6 2/20 (10.00%)  2
Indicates events were collected by systematic assessment
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
Layout table for Results Point of Contact information
Name/Title: Alyssa Tran, BS
Organization: Baylor
Phone: 832-822-4264
EMail: alyssat@bcm.edu
Layout table for additonal information
Responsible Party: Brendan Lee, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT01529060     History of Changes
Other Study ID Numbers: H-28463
First Submitted: February 6, 2012
First Posted: February 8, 2012
Results First Submitted: September 18, 2017
Results First Posted: March 6, 2019
Last Update Posted: March 19, 2019