PKU-PAED-LIQ-2022-10-07

About this trial

Last updated 8 months ago

Study ID

Status

Recruiting

Type

Interventional

Phase

N/A

Placebo

Accepting

1 to 10 Years

All

Not accepting

Healthy Volunteers

Trial Timing

Started a year ago

PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.

Inclusion Criteria

- Diagnosis of PKU requiring a low-protein diet and protein substitute.

- Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator.

- Participants aged 1-10 years.

- Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process).

- Willingly given, written, informed consent from the parent(s)/guardian(s).

- Willingly given, written assent by the participant (if appropriate).

Exclusion Criteria

- Inability to comply with the study protocol, in the opinion of the investigator.

- Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.

- Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute.

- Allergy or intolerance to milk.

- Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit.

- Use of additional micronutrient supplements during the evaluation period, unless clinically indicated and prescribed by the Investigator (must be recorded in both the medical records and case report form).