The main objective: to evaluate the prescribed intake of micronutrients and fatty acids from natural protein and amino acid mixtures, and to correlate it to blood levels of these micronutrients and fatty acids, in patients with PKU. Secondary…
ID
Source
Brief title
Condition
- Metabolic and nutritional disorders congenital
- Protein and amino acid metabolism disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
- To asses the prescribed micronutrient and fatty acid intake of patients with
PKU treated with a protein restricted diet with supplementation of amino acids
in a mixture with vitamins and minerals, or sapropterin.
- To correlate the prescribed intake of micronutrients and fatty acids from the
diet supplemented with an amino acid mixture, or sapropterin, to blood levels
of micronutrients and fatty acids.
Secondary outcome
- To assess the BMD and bone turnover in patients with PKU treated with a
protein restricted diet with supplementation of amino acids in a mixture with
vitamins and minerals, or sapropterin.
- To correlate the prescribed intake of micronutrients and fatty acids from a
protein restricted diet supplemented with an amino acid mixture, or
sapropterin, to BMD and bone turnover.
- To correlate blood levels of micronutrients and fatty acids in patients with
phenylketonuria to BMD and bone turnover.
Background summary
In order to optimize treatment for patients with phenylketonuria (PKU), and to
prevent deficiencies as well as to prevent overdosing of micronutrients there
is a strong need for more insight into the micronutrient and fatty acid intake
of patients from their prescribed diet and into the effects of this intake on
blood levels. Because lower bone mass (BMD) in phenylketonuria patients has
been reported in previous studies, a better insight in the effects of
micronutrient and fatty acid intake on BMD and bone turnover is warranted.
Study objective
The main objective: to evaluate the prescribed intake of micronutrients and
fatty acids from natural protein and amino acid mixtures, and to correlate it
to blood levels of these micronutrients and fatty acids, in patients with PKU.
Secondary objectives: to evaluate the BMD and bone turnover of a large cohort
of patients with PKU and to correlate it to prescribed intake and blood levels
of micronutrients and fatty acids from natural protein and amino acid mixtures.
Study design
This study constitutes an observational multicenter study with a single
invasive measurement.
Study burden and risks
Risks and burden for this study are very limited. No physical discomfort is
expected, and no risks are associated with participating in providing extra
blood samples.
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
This study includes patients with PKU, age of 1 year and older, diagnosed by newborn screening and treated with a protein restricted diet with supplementation of an amino acid mixture, recruited from three academic metabolic centers in the Netherlands. Patients treated with sapropterin are also included, with or without dietary treatment.
Exclusion criteria
Significant changes in the type or amount of amino acid supplements used, within one month before the inclusion date.
Pregnancy.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL43580.018.13 |