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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01529060 |
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Date of registration:
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06/02/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Phenylbutyrate Therapy for Maple Syrup Urine Disease
MSUD |
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Scientific title:
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A Double-Blind, Randomized, Placebo-Controlled, Crossover Trial of Phenylbutyrate in the Treatment of Maple Syrup Urine Disease |
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Date of first enrolment:
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February 2013 |
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Target sample size:
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20 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01529060 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Brendan Lee, M.D., Ph.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor College of Medicine |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Must be 3 years or older at enrollment.
- Must have a diagnosis of maple syrup urine disease (MSUD) confirmed by the presence of
plasma alloisoleucine (>5 micromol/L) and/or genetic testing showing mutations in both
alleles of any subunit of BCKDHA (E1alpha subunit gene, MSUD type 1A), BCKDHB (E1beta
subunit gene, MSUD type 1B), or DBT (E2 subunit gene, MSUD type 2).
- Participants must have a history of compliance to diet and treatment.
- Signed informed consent by subject and/or subject's legally acceptable representative.
- Must be capable of completing study procedures, including taking oral or G- tube
medication.
- Negative pregnancy test for all females of childbearing potential.
- All females of childbearing potential and all sexually active males must agree to use
an acceptable method of contraception throughout the study. Appropriate contraceptive
methods include hormonal contraceptives (oral, injected, implanted, or transdermal),
tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier
methods. Abstinence is an acceptable form of birth control, though appropriate
contraception must be used if the subject becomes sexually active.
Exclusion Criteria:
- May not have used sodium phenylbutyrate within 30 days of Visit 1.
- May not have an active infection (viral or bacterial) or any condition which may
exacerbate their MSUD causing metabolic decompensation.
- Cannot have any clinical or laboratory abnormality of Grade 3 or greater according to
the Common Terminology Criteria for Adverse Events v3.0 (CTCAE) (or for conditions not
covered by the CTCAE, a severe or life-threatening toxicity).
- May not have taken any medications known to significantly affect renal clearance or to
increase protein catabolism within the 24 hours prior to Visit 1.
- May not participate if they have a known hypersensitivity to phenylacetate or
phenylbutyrate or creatinine levels 1.5 times or more ULN.
- Since a total of 53 mL will be drawn over Days 14 and 15 of both treatment periods,
only subjects weighing more than 30 pounds can be enrolled.
Age minimum:
3 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Maple Syrup Urine Disease
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Intervention(s)
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Drug: Placebo powder
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Drug: Phenylbutyrate
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Primary Outcome(s)
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Leucine CMax 0-24 Hours
[Time Frame: 24 hours]
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0-24 Hour AUC Leucine (Samples Collected at 0, 2, 4, 8, 12, 16, 20, and 24 Hours)
[Time Frame: 24 Hours]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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