Evaluation of PKU Sphere in Maternal PKU (Maternal PKU)

February 14, 2024 updated by: Vitaflo International, Ltd

An Observational Study to Evaluate Metabolic Control and Dietary Management in Women With PKU Taking PKU Sphere During Pre-conception and/or Pregnancy

This observational study aims to recruit females, aged 16 years and over, with phenylketonuria (PKU) or hyperphenylalaninemia (hyperphe) following dietary management advice pre-conception and/or during pregnancy, who are willing to take PKU sphere as part of their dietarty management.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The aim of this study is to observe dietary management and metabolic control in women taking PKU sphere pre-conceptually and/or during pregnancy. To do this the following data points will be captured:

  • Blood spot phenylalanine and tyrosine levels to measure metabolic control
  • Changes to dietary management and any adaptations arising from incorporating PKU Sphere
  • Compliance/adherence of patients to their recommended amount of protein substitute prescription.
  • Tolerance of PKU sphere with respect to gastrointestinal (GI) symptoms and pregnancy associated nausea and vomiting.
  • Acceptability of concurrent protein substitute(s) (if applicable).
  • Nutritional status and weight management.
  • Routine standard of care data on the final pregnancy outcome and postpartum.

Study Type

Observational

Enrollment (Estimated)

10

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Maternal and preconception females, aged 16 years onwards with phenylketonuria (PKU) requiring dietary management with protein substitutes.

Description

Inclusion Criteria:

  • Diagnosis of phenylketonuria (PKU) or hyperphenylalaninemia (hyperphe) which requires dietary management during pre-conception and/or pregnancy.
  • Aged 16 years and above.
  • Following dietary management advice pre-conceptually and/or during pregnancy, aiming for phenylalanine levels of 120-250µmol/L.
  • Prior positive PKU sphere taste test completed as part of routine care.
  • Chosen to take part or full requirement of protein substitute as PKU sphere pre-conceptually and/or during pregnancy.
  • Willingly given, written, informed consent from patient.
  • Participant is, in the opinion of the investigator, able to participate and can comply with the study protocol.

Exclusion Criteria:

  • Conception of pregnancy without commencement of phe-restricted diet and blood phenylalanine not maintained within target range by 10 weeks' gestation.
  • Patients with known soya, milk or fish allergies / intolerance.
  • Intake of pegvaliase or large neutral amino acids within 30 days prior to screening visit.
  • Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening visit.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
PKU sphere
PKU sphere (an FSMP) as per individual requirements determined by a dietitian.
Dietary supplement: PKU sphere

PKU sphere is a Food for Special Medical Purposes (FSMP). This product is for use in the dietary management of phenylketonuria (PKU).

It is a powdered, low phenylalanine medical food containing a balanced mix of GMP, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, and DHA.

It is available in 27g sachets providing 15g PE and contains 28mg Phe and 35g sachets providing 20g PE and contains 36mg Phe.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in blood spot phenylalanine levels
Time Frame: Baseline, 1-2 times weekly during preconception (max. 2 years), 2-3 times weekly during pregnancy, one month postpartum.
Measurement of fasting phenylalanine concentrations completed once (1) to twice (2) weekly preconception (which may last for an indeterminate amount of time), twice (2) to three (3) times weekly during pregnancy and one month postpartum. Collected as dried bloodspots on Guthrie cards in the patient's own home and posted to the hospital laboratory for analysis.
Baseline, 1-2 times weekly during preconception (max. 2 years), 2-3 times weekly during pregnancy, one month postpartum.
Change in blood spot tyrosine levels
Time Frame: Baseline, 1-2 times weekly during preconception (max. 2 years), 2-3 times weekly during pregnancy, one month postpartum.
Measurement of fasting tyrosine concentrations completed once (1) to twice (2) weekly preconception (which may last for an indeterminate amount of time), twice (2) to three (3) times weekly during pregnancy and one month postpartum. Collected as dried bloodspots on Guthrie cards in the patient's own home and posted to the hospital laboratory for analysis.
Baseline, 1-2 times weekly during preconception (max. 2 years), 2-3 times weekly during pregnancy, one month postpartum.
Change in adherence to study product intake
Time Frame: Every 12 weeks from baseline during preconception (max. 2 years) and pregnancy until the end of the pregnancy
3-day diary recording compliance/adherence of patients to their prescribed amount of protein substitute.
Every 12 weeks from baseline during preconception (max. 2 years) and pregnancy until the end of the pregnancy
Participants' gastrointestinal adverse events
Time Frame: Throughout the study until one month postpartum
Participant to report any gastrointestinal (GI) symptoms and pregnancy-associated nausea and vomiting while consuming PKU Sphere.
Throughout the study until one month postpartum
Change in weight
Time Frame: Baseline, every 24 weeks during preconception (max. 2 years) and pregnancy, one month postpartum
Weight (kg)
Baseline, every 24 weeks during preconception (max. 2 years) and pregnancy, one month postpartum
Change in plasma amino acid profile
Time Frame: Baseline, all routine results during preconception (max. 2 years) and pregnancy, one month postpartum
Plasma amino acid profile
Baseline, all routine results during preconception (max. 2 years) and pregnancy, one month postpartum
Change in plasma micronutrient profile
Time Frame: Baseline, all routine results during preconception (max. 2 years) and pregnancy, one month postpartum
Plasma micronutrient profile
Baseline, all routine results during preconception (max. 2 years) and pregnancy, one month postpartum
Pregnancy outcome
Time Frame: End of pregnancy
Standard of care data on the outcome of the pregnancy
End of pregnancy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vitaflo International, Ltd

Collaborators

University College London Hospitals

Investigators

  • Principal Investigator: Charlotte Ellerton, University College London Hospitals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 29, 2019

Primary Completion (Estimated)

June 30, 2025

Study Completion (Estimated)

June 30, 2025

Study Registration Dates

First Submitted

October 7, 2019

First Submitted That Met QC Criteria

January 7, 2020

First Posted (Actual)

January 13, 2020

Study Record Updates

Last Update Posted (Actual)

February 15, 2024

Last Update Submitted That Met QC Criteria

February 14, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MCT-GMP-2018-08-14
  • 256857 (Other Identifier: IRAS)
  • 19/NW/0167 (Other Identifier: HRA)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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