N/A
N=32
Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
Phenylketonuria
Bottom Line
View on ClinicalTrials.gov: NCT01428258 ↗Enrolled (actual)
32
Serious AEs
0.0%
Results posted
Mar 2017
Primary outcome: Primary: Change in the Plasma Phenylalanine Concentration of PKU Subjects Fed the Glycomacropeptide Diet Compared With the Change When Fed the Amino Acid Diet — 62; -85 micro moles per liter plasma — p=0.0008
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- GMP Diet/GMP Medical Foods (Other); AA Diet/AA Medical Foods (Other)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- All
- Sponsor
- University of Wisconsin, Madison
- Primary completion
- Nov 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in the Plasma Phenylalanine Concentration of PKU Subjects Fed the Glycomacropeptide Diet Compared With the Change When Fed the Amino Acid Diet |
62; -85 | 0.0008 sig |
| SECONDARY Dietary Compliance |
0.74; 0.76 | 0.576 |
| SECONDARY Executive Function Assessed by BRIEF |
49.0; 48.8 | 0.902 |
| SECONDARY Vitamin D (25-OH) Plasma Concentration at Day 22 |
33.8; 33.6 | 0.797 |
| SECONDARY Comparison of Phe Concentrations in Plasma With Concentrations in Dried Blood Spots |
731; 514 | 0.0001 sig |
| SECONDARY Bone-specific Alkaline Phosphatase (BSAP) Plasma Concentration at Day 22 |
17.0; 17.0 | — |
| SECONDARY N-terminal Telopeptide (NTX) Plasma Concentration at Day 22 |
17.5; 17.1 | — |
Summary
For individuals with Phenylketonuria (PKU), the investigators hypothesize that glycomacropeptide will provide an acceptable form of low-phenylalanine dietary protein that will improve dietary compliance, blood phenylalanine levels, cognitive function, and ultimately quality of life compared with the usual amino acid based diet. The study is funded by the Food and Drug Administration (FDA) Office of Orphan Products Development Grants Program, R01 FD003711.
Eligibility Criteria
Inclusion Criteria
- Identified PKU by newborn screening; started diet treatment before 1 mo age
- Diagnosis of classical or variant PKU with documented phenylalanine level of greater than 600 umol/L at 7-10d of age
- Follows or willing to follow PKU diet and consume amino acid medical formula providing more than 50% of protein needs
- Acceptance of glycomacropeptide foods determined prior to enrollment
Exclusion Criteria
- Females who are pregnant or planning pregnancy
- Individuals with mental deficits due to untreated or poorly controlled PKU
- Individuals with any health condition deemed to interfere with participation
Data sourced from ClinicalTrials.gov (NCT01428258). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.