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Evaluation of PKU Sphere in Maternal PKU (Maternal PKU)

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ClinicalTrials.gov Identifier: NCT04224142
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : February 5, 2020
Sponsor:
Collaborator:
University College London Hospitals
Information provided by (Responsible Party):
Vitaflo International, Ltd

Brief Summary:
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.

Condition or disease Intervention/treatment
Maternal Phenylketonuria Dietary Supplement: PKU sphere

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.

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Study Type : Observational
Estimated Enrollment : 10 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Observational Study to Evaluate Metabolic Control and Dietary Management in Women With PKU Taking PKU Sphere During Pre-conception and/or Pregnancy
Actual Study Start Date : July 29, 2019
Estimated Primary Completion Date : July 29, 2021
Estimated Study Completion Date : July 29, 2021


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.





Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. Change in weight [ Time Frame: Baseline, every 24 weeks during preconception (max. 2 years) and pregnancy, one month postpartum ]
    Weight (kg)

  6. 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

  7. Change in plasma micronutrient profile [ Time Frame: Baseline, all routine results during preconception (max. 2 years) and pregnancy, one month postpartum ]
    Plasma micronutrient profile

  8. Pregnancy outcome [ Time Frame: End of pregnancy ]
    Standard of care data on the outcome of the pregnancy



Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Female
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.
Criteria

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 sapropterin dihydrochloride (Kuvan), 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04224142


Contacts
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Contact: Karl Harvey 0151 709 9020 ext 300 Karl.Harvey@Vitaflo.co.uk
Contact: Clinical Trials 0151 709 9020 ClinicalTrialsTeam@Vitaflo.co.uk

Locations
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United Kingdom
NHS Greater Glasgow and Clyde Recruiting
Glasgow, United Kingdom
Contact: Sarah Adam    0141 452 6451    Sarah.Adam@GGC.Scot.NHS.UK   
University College London Hospitals NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Charlotte Ellerton    0203 448 4332    C.Ellerton@NHS.Net   
Contact: Harriet Churchill    0203 448 3604    Harriet.Churchill@NHS.Net   
Sponsors and Collaborators
Vitaflo International, Ltd
University College London Hospitals
Investigators
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Principal Investigator: Charlotte Ellerton University College London Hospitals
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Responsible Party: Vitaflo International, Ltd
ClinicalTrials.gov Identifier: NCT04224142    
Other Study ID Numbers: MCT-GMP-2018-08-14
256857 ( Other Identifier: IRAS )
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: February 5, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Vitaflo International, Ltd:
Maternal
PKU
Phenylketonuria
GMP
Glycomacropeptide
Additional relevant MeSH terms:
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Phenylketonurias
Phenylketonuria, Maternal
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Pregnancy Complications