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Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU

ConditionsPhenylketonuria (PKU) and HyperphenylalaninemiaBrain Diseases, Metabolic, InbornBrain Disorder, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Disease, InbornGenetic Diseases, InbornMetabolic DiseasesNutritional and Metabolic Diseases
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-18
SponsormetaX Institut fuer Diatetik GmbH

About this trial

XPhePiccosMR is a prospective, single-arm, open label, observational study that aims to evaluate the acceptability and tolerability of XPhe Piccos, a slow-release, phenylalanine-free protein substitute in the form of tiny tablets, for use in the dietary management of phenylketonuria (PKU) in children aged three years and older.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of PKU or HPA requiring a phenylalanine-free protein substitute.

Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.

Subjects aged 3 years and above.

Written informed assent/consent obtained from subject and/or parental caregiver, depending on subject age.

Disqualifiers

Presence of serious concurrent illness.

Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements

Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.

Any subject having taken antibiotics over the previous 2 weeks leading up to the study.

Trial design

Design model

Single group

Treatments tested in this trial

  • Slow release, phenylalanine-free protein substitute tablets

    Dietary supplement

    The slow release, phenylalanine-free protein substitute in tablet form (XPhe Piccos) is a food for special medical purposes (FSMP) for use in the dietary management of Phenylketonuria or Hyperphenylalaninemia. The product is suitable from 3 years of age and over. XPhe Piccos are a highly concentrated phenylalanine-free protein supplement in the form of very small tablets/pellets enriched with vitamins, minerals and trace elements. XPhe Piccos are characterized by a prolonged release of the amino acids.

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: Participant groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Compliance

Compliance with the recommended intake of the usually prescribed protein substitute is assessed by the dietitian at baseline. Usage and compliance with the study product are recorded daily from day 1 to day 7 using a standardised form. Using the form, patients have to record the amount of the study product consumed in comparison to the prescribed doses. The reason for not consuming the full dose will be recorded by the patient/parent/caregiver as free text. Patients are asked to state whether the study product is easy to prepare with yes or no.

Time frame
daily; from day 1 to day 7

Secondary outcomes

1

Gastro-intestinal tolerance

Gastro-intestinal side effects (including diarrhoea, constipation, bloating or abdominal distension, nausea, vomiting, burping, flatulence or regurgitation and abdominal discomfort or pain) will be assessed at baseline and daily (from day 1 to day 7) via a standardised questionnaire using the following scale: none today, mild, moderate, or severe / really troublsome.

Time frame
daily; from day 1 to day 7
2

Patient Acceptability

Accepatbility (ease of use, taste, texture, palatability) will be assessed daily during the study period and at the end of the study via a standardised questionnaire with a rating scale of 5 (great) to 1 (terrible).

Time frame
daily; from day 1 to day 7
3

Metabolic Control

Weekly routine finger prick blood spots will be collected and analysed for phenylalanine and tyrosine as is usual clinical practice.

Time frame
baseline (day 1) and day 7
4

Incidence of study product emergent events

All adverse events will be recorded throughout the study (as and when they occur). There is no specific time frame as the frequency of adverse events can not be predicted. But as the study product is already commercially available occurance of any adverse event is unlikely.

Time frame
throughout the study; from day 1 to day 7

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

metaX Institut fuer Diatetik GmbH

Lead sponsor

Birmingham Women's and Children's NHS Foundation Trust

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: