Condition category
Nutritional, Metabolic, Endocrine
Date applied
04/06/2018
Date assigned
07/06/2018
Last edited
07/06/2018
Prospective/Retrospective
Prospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

Plain English Summary

Background and study aims
Familial hypercholesterolaemia (FH) is a common genetic disorder, characterised by raised levels of low density lipoprotein cholesterol (LDL-C) from birth. The lifelong exposure to these high LDL-C levels puts individuals at an increased risk of premature cardiovascular disease (CVD) and associated mortality (death). Lifelong treatment with medication is recommended for all patients. This has reduced the incidence of CVD in people with FH, but they still remain at higher risk than the general population. Lifestyle factors can also influence LDL-C levels and other CVD risk factors and therefore lifestyle advice is considered important for people with FH. However, there is limited evidence to support the effectiveness of the current nutritional and physical activity (PA) guidelines upon LDL-C levels in people with FH. The need for trials to investigate the effectiveness of nutrition and PA interventions in the FH population has been widely recognised and recommended. This study is designed to investigate if a family-based intervention based upon the current NICE nutrition and PA recommendations for people with FH can be implemented and accepted by young people and their affected parent.

Who can participate?
Patients aged 10-18 with FH and their parents

What does the study involve?
Families are randomly allocated to receive a 12-week nutrition and PA intervention or to a control group who receive usual care, which is medication only. Nutrition intake, PA levels and blood cholesterol levels are measured at the start of the study and at 12-week follow up. The feasibility of the intervention and research methods are assessed through measurement of recruitment to the study, attendance at study visits and acceptability of the intervention determined through interviews. Blood cholesterol levels, dietary intakes and PA levels are measured before the intervention and at 12-week follow up and any changes are compared between the control and intervention groups.

What are the possible benefits and risks of participating?
The results will inform the development of a full-scale study. There are no significant benefits or risks expected as a result of participation in this study.

Where is the study run from?
1. University Hospitals Bristol NHS Foundation Trust (UK)
2. Royal United Hospitals Bath NHS Foundation Trust (UK)

When is the study starting and how long is it expected to run for?
January 2018 to April 2020

Who is funding the study?
NIHR Bristol Biomedical Research Centre (UK)

Who is the main contact?
Ms Fiona Kinnear

Trial website

Contact information

Type

Scientific

Primary contact

Ms Fiona Kinnear

ORCID ID

http://orcid.org/0000-0002-4090-1554

Contact details

NIHR Bristol Biomedical Research Centre Nutrition Theme
3rd Floor
Education & Research Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

38422

Study information

Scientific title

Feasibility of a family-based nutrition and physical activity intervention in young people with familial hypercholesterolaemia: a mixed methods study

Acronym

Study hypothesis

Familial hypercholesterolaemia (FH) is a common genetic disorder, characterised by raised levels of low density lipoprotein cholesterol (LDL-C) from birth. The lifelong exposure to these high LDL-C levels puts individuals at an increased risk of premature cardiovascular disease (CVD) and associated mortality.
Lifelong treatment with medication is recommended for all patients. This has reduced the incidence of CVD in people with FH, however they still remain at higher risk than the general population. Lifestyle factors can also influence LDL-C levels and other CVD risk factors and therefore lifestyle advice is considered importance for people with FH. However, there is limited evidence to support the effectiveness of the current nutritional and physical activity (PA) guidelines upon LDL-C levels in people with FH. The need for trials to investigate the effectiveness of nutrition and PA interventions in the FH population has been widely recognised and recommended.

This study is designed to investigate if a family-based intervention based upon the current NICE nutrition and PA recommendations for people with FH can be implemented and accepted by young people and their affected parent.

Ethics approval

South West-Central Bristol research ethics committee - approval pending

Study design

Randomised; Interventional; Design type: Treatment, Education or Self-Management, Dietary, Physical

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details to request a patient information sheet

Condition

Specialty: Children, Primary sub-specialty: Inherited Metabolic Disorders; UKCRC code/ Disease: Metabolic and Endocrine/ Metabolic disorders, Cardiovascular/ Other and unspecified disorders of the circulatory system

Intervention

Family units will be randomly assigned to one of two groups to receive either usual care (control group) or the 12-week nutrition and PA intervention (intervention group). 1:1 randomisation of family units to intervention or control will be carried out according to prepared randomised lists, stratified by study site (University Hospitals Bristol or Royal United Hospitals Bath), anticipating a 50:50 split.

Control group: families will receive usual care, which at these hospital sites is medication only
Intervention group: receive an individualised family-based nutrition and PA intervention with the research dietitian lasting approximately 1 hour. This will be followed up with regular phone call and email contact throughout the 12 weeks of the intervention.
The nutrition component of the intervention aims to help family units to achieve the following daily intake guidelines:
1. Total fat intake ≤30% of total energy intake (TEI)
2. Saturated fat intake of ≤10% TEI
3. Replacement of saturated fats with mono and poly-unsaturated fats
3. Dietary cholesterol intake ≤300mg
4. Consumption of ≥ 5 portions of fruit and vegetables
5. Age appropriate fibre intake (10 year olds= 20g/day; 11-16 year olds= 25g/day and 30g/day for ≥17 year olds)
6. 2g of stanol/sterols

The PA component of the intervention aims to help family units achieve the recommended population PA guidelines which are:
Adults and young people are to minimise time spent being sedentary
2. Adults (19-64 years old) are to achieve a minimum of 150 minutes a week of moderate intensity PA or 75 minutes of vigorous intensity PA, or a mixture of the two. Additionally activity focussing upon improving muscle strength should be undertaken twice a week.
3. Young people (5-18 years old) are to achieve a minimum of 60 minutes of at least moderate intensity PA each day, with 3 of these sessions each week being of vigorous activity and including activities that strengthen muscle and bone.
These guidelines are targets and will be used to guide the consultation and educate the family units. The dietitian will work with the family units to agree upon changes they will make to their nutrition and PA over the 12-week intervention to achieve intakes closer to the recommended targets than they were achieving at baseline. However, the advice delivered from the dietitian will take into consideration the individuals’ baseline dietary and PA data alongside insights gained from qualitative interviews conducted with a sub-sample of the study participants. Individual circumstances such as work or school schedules, food preferences, religious beliefs, available space for cooking and financial situations will also be taken into consideration by the dietitian. Intervention booklets, one for participants aged 10-13 years and one for participants aged ≥ 14 years, have been created for use at research contact 2 to record the agreed changes and provide a summary of the information covered in the consultation for the family units to refer to at home during the 12-week intervention period. The agreed changes to PA or nutrition may be most appropriately recorded as targets for the family unit as one, or for individual members, or a mixture of the two. This will depend on individual circumstances such as age of the child, frequency of meals eaten together and differences in baseline nutritional and PA levels. In-depth dietary planning will be conducted, in addition to the provision of behaviour change advice and practical advice to address any challenges that implementing nutrition or PA change may provoke.

Nutrition intake, PA levels and blood cholesterol levels will be measured at baseline and at 12-week follow up.

Intervention type

Behavioural

Phase

Drug names

Primary outcome measure

The feasibility of the intervention and research methods, assessed through measurement of:
1. Recruitment to the trial
2. Attendance at research visits
3. Retention rate
4. Adherence to research procedure methods including data collection
5. Acceptability of the dietary and PA intervention
These will all be measured at the end of the study. Data from adult and child participants will be analysed separately. Recruitment, attendance and retention rates will be descriptively described. Adherence to research procedures will be measured through the calculating the number of participants who completed each research procedure. Acceptability of the dietary and PA intervention will be measured through analysis of the qualitative data collected through qualitative interviews conducted with a sub-sample of the participants at their final research visit.

Secondary outcome measures

1. Blood metabolomic (primarily lipidomic) markers , measured through analysis of blood samples collected pre and post intervention
2. Energy content and nutrient composition (macro- and micro-nutrient) of dietary intake, measured using online 24hr recall tool over 4 days at home by each participant pre and post intervention
3. PA levels (minutes of moderate and vigorous PA) and sedentary activity (minutes of sedentary time), measured by Actigraph and ActivPal monitors worn by participants over 7 days 1 week pre and post intervention
4. Health related quality of life (QOL), measured using an age-appropriate QOL questionnaire pre and post intervention
These will be measured at research contacts 2 and 3. Research contact 2 will be carried out at baseline and research contact 3 approximately 12 weeks after this

Overall trial start date

08/01/2018

Overall trial end date

06/04/2020

Reason abandoned (if study stopped)

Eligibility

Participant inclusion criteria

1. Patients aged 10-18 years and their parent (≥18 years)
2. Genetically confirmed clinical diagnosis of heterozygous FH
3. Receive their care from the paediatric or adult lipid clinics at Bristol University Hospitals or Royal United Hospitals Bath NHS Trust Foundations

Participant type

Mixed

Age group

Mixed

Gender

Both

Target number of participants

Planned Sample Size: 72; UK Sample Size: 72

Participant exclusion criteria

1. Pregnant female patients, or those planning pregnancy
2. Patients who are unable to give informed consent
3. Parents or carers of young people with FH who themselves do not have FH
4. Patients with a diagnosis of homozygous FH
5. Patients not established on statin therapy for at least 3 months prior to recruitment to the study

Recruitment start date

01/09/2018

Recruitment end date

06/01/2020

Locations

Countries of recruitment

United Kingdom

Trial participating centre

University Hospitals Bristol NHS Foundation Trust
Trust Headquarters Marlborough Street
Bristol
BS1 3NU
United Kingdom

Trial participating centre

Royal United Hospitals Bath NHS Foundation Trust
Combe Park
Bath
BA1 3NG
United Kingdom

Sponsor information

Organisation

University of Bristol

Sponsor details

Research & Enterprise Development
Senate House
Tyndall Avenue
Bristol
BS8 1TH
United Kingdom

Sponsor type

University/education

Website

Funders

Funder type

Government

Funder name

NIHR Bristol Biomedical Research Centre; Grant Codes: BRC-1215-20011

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

It is anticipated that the study protocol will be available. It is not yet currently published or available online. Planned publication of the study results in a high-impact peer reviewed journal anticipated in October 2020. It will also be disseminated as part of a PhD thesis.

IPD sharing statement
The datasets generated and/or analysed during the current study during this study will be included in the subsequent results publication.

Intention to publish date

01/10/2020

Participant level data

Other

Basic results (scientific)

Publication list

Publication citations

Additional files

Editorial Notes