A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)

Status: Active_not_recruiting
Location: See all (6) locations...
Intervention Type: Dietary supplement
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Healthy Volunteers: f
View:

• Diagnosis of PKU or associated disorder for example, Tetrahydrobiopterin (BH4) deficiency, requiring a low protein diet and Phe-free L-amino acid supplements (protein substitute).

• Established on PKU express or an equivalent protein substitute

• In the opinion of the study investigator, the participant is able to take the study product and meets at least one of the following criteria:

‣ taking an age inappropriate protein substitute

⁃ struggling with adherence to current protein substitute

⁃ delayed in transitioning

• Aged 3 years and above

• Willingly given, written, informed consent from patient or parent/guardian.

• Willingly given, written assent (if appropriate).

Locations
Other Locations
United Kingdom
Royal Belfast Hospital for Sick Children
Belfast
Birmingham Women's and Children's Hospital
Birmingham
Bristol Royal Hospital for Children
Bristol
Greater Glasgow and Clyde NHS Foundation Trust
Glasgow
Great Ormond Street Hospital for Children
London
Nottingham Children's Hospital
Nottingham
Time Frame
Start Date: 2021-06-14
Completion Date: 2025-12-31
Participants
Target number of participants: 28
Treatments
Experimental: express pus range (PKU, MSUD, HCU, TYR and GA)
express plus to be transitioned onto over a maximum of 6 weeks and then incorporated into each participants usual diet for 28 days. Amount taken and frequency to be determined by dietitian.
Authors
Anne Daly, Sarah Adam
Sponsors
Leads: Vitaflo International, Ltd

This content was sourced from clinicaltrials.gov