Health TrialsbyHealthPanel

Liquid Valine and Isoleucine in Maple Syrup Urine Disease

ConditionMaple Syrup Urine Disease
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age12-16
SponsorMeta Healthcare Ltd

About this trial

This is a prospective, observational research study in 5 children with Maple Syrup Urine Disease (MSUD). Subjects who are currently taking a valine and isoleucine supplement for MSUD will be recruited for a 56 day trial, of a new ready-to-use valine supplement and a new ready-to-use isoleucine supplement, to evaluate the tolerability and acceptability of the study products compared with their usual products.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male and female MSUD patients ≥1 year and <16 years of age.

Patients diagnosed with MSUD.

Taking both valine and isoleucine supplements for MSUD and willing to take the new supplements for 56 days.

Absence of comorbidities.

Disqualifiers

Age <1 year old and >16 years old.

Patients with comorbidities.

Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.

History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.

Trial design

Design model

Single group

Treatments tested in this trial

  • Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml)

    Dietary supplement

    Subjects will take valine oral solution (50mg/ml) in place of usual powdered valine supplement for 28 days. Subjects will then take isoleucine oral solution (25mg/ml) in place of their usual powdered isoleucine supplement for 28 days.

Treatment groups

5 Participants
are divided into 1 treatment group
Group A: Liquid valine and liquid isoleucine administrationExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Daily data on gastrointestinal tolerance

A daily record of any gastrointestinal symptoms compared with usual valine and isoleucine supplement

Time frame
From enrollment to end of treatment at 56 days
2

Acceptability and tolerance

Qualitative questionnaires at study start and end of Part 1 (28 days) and Part 2 (56 dys) of the study

Time frame
From enrolment to end of treatment at 56
3

Daily data on study product adherence

A daily record of study product intake (actual versus prescribed) to check for adherence

Time frame
From enrollment to the end of treatment at 56 days
4

3-day food diaries

A record of patient food and fluid intake for 3 days to monitor valine intake from food - at enrollment, end of part 1 and end of part 2

Time frame
From enrollment to the end of treatment at 56 days

Secondary outcomes

1

Weight and height measurement

Weight and height will be measured at enrollment, end of Part 1 (28 days) and end of Part 2 (56 days) of the study to check that growth is adequate

Time frame
From enrollment to the end of treatment at 56 days

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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

Meta Healthcare Ltd

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: