Clinical trial NCT05051657: Evaluation of the Express Plus Range
Official title: 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Phenylketonurias
PKU
Homocystinuria
Maple Syrup Urine Disease
Hereditary Tyrosinemia
Glutaric Acidemia I
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
First posted: 2021-09-21
Enrollment: 28
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: single group - all patients to receive relevant express plus product
Primary purpose: SUPPORTIVE_CARE

Brief 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.

Detailed description: This is a prospective acceptability study of the express plus range in 40 participants aged at least three years of age for the dietary management of IEM (PKU, MSUD, HCU, TYR and GA ).

The study will involve a Transition Period where patients will build-up to at least one sachet of express plus per day. During the Transition Period, daily intake of express plus and the patient's other PS will be recorded, including the time taken to consume.

The Transition Period will last a maximum of 6 weeks, after which the patient will enter a 28-day Evaluation Period. During the Evaluation Period, patients will continue to record intakes, as well as GI tolerance and a final evaluation of the product's palatability and usability. Some patients, likely adults, will not require a transition from their original protein substitute to at least one sachet of express plus. These patients can enter the Evaluation Period directly.

The end of the 4-week Evaluation Period is defined as the participant's End of Study. Following this, participants will be followed up as per standard of care. Some of this standard of care data may be relayed to the sponsor for a maximum of 26 weeks. This will last for a maximum of 26 weeks and will involve the collection of routine phe and tyrosine levels, as well as amount of PS prescribed and taken. Information will also be collected from patients regarding the reasons for the success or indeed failure of transition to PKU express plus. This data will come from routine clinical contact and will be recorded by the HCP, rather than by the patient.

Eligibility criteria: Inclusion Criteria:

* 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:

  1. taking an age inappropriate protein substitute
  2. struggling with adherence to current protein substitute
  3. delayed in transitioning
* Aged 3 years and above
* Willingly given, written, informed consent from patient or parent/guardian.
* Willingly given, written assent (if appropriate).

Exclusion Criteria:

* Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein substitute.
* Patients who are tube-fed.
* Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol.
* Any co-morbidity/condition which in the opinion of the Investigator, would preclude participation in the study.
* 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.
* Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).
* Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period. (N.B.: Women who become pregnant unexpectedly during this study may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.)
* Those with allergies to fish, milk or soya.

Primary outcomes:
measure: Questionnaire of self-reported adherence to the prescribed amount of study product
timeFrame: Days 1-28
measure: Transition time
timeFrame: Transition period Weeks 1-6 (maximum)
measure: Change in gastrointestinal tolerance from week 1 to week 4
timeFrame: Days 1-7 and days 21-28
measure: Product acceptability rated on a Likert scale by the patient after 28-day intake
timeFrame: Visit 2 (end of 28 day evaluation period)
measure: Change in general neophobia
timeFrame: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
measure: Change in food neophobia
timeFrame: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
measure: Nutritional suitability: change Phenylalanine levels
timeFrame: Visit 1 until visit 2 (day 28)
measure: Nutritional suitability: change Tyrosine levels
timeFrame: Visit 1 until visit 2 (day 28)
Secondary outcomes:
Other outcomes:
Locations:
city: Belfast
country: United Kingdom
facility: Royal Belfast Hospital for Sick Children
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's Hospital
city: Bristol
country: United Kingdom
facility: Bristol Royal Hospital for Children
city: Glasgow
country: United Kingdom
facility: Greater Glasgow and Clyde NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Nottingham
country: United Kingdom
facility: Nottingham Children's Hospital
Study officials:
affiliation: Birmingham Women's and Children's Hospital
name: Anita MacDonald
role: STUDY_DIRECTOR
affiliation: Royal Belfast Hospital for Sick Children
name: Anne Grimsley
role: PRINCIPAL_INVESTIGATOR
affiliation: Greater Glasgow and Clyde NHS Foundation Trust
name: Barbara Cochrane
role: PRINCIPAL_INVESTIGATOR
References: