search
Back to results

Feasibility of Consumption of Nutritional Supplementation in Primary Ciliary Dyskinesia ((PCD))

Primary Purpose

Primary Ciliary Dyskinesia

Status
Completed
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
QD-27
Sponsored by
University of Leeds
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Primary Ciliary Dyskinesia

Eligibility Criteria

17 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: People aged 17 or over with a confirmed diagnosis of Primary ciliary dyskinesia PCD (nasal brushings) People who attend the Leeds Regional PCD Clinics People who have capacity to give informed consent Exclusion Criteria: People who are pregnant Those with existing co-morbidities such as malignancy People with connective tissue disorders and immunoglobulin deficiencies People with renal insufficiency r

Sites / Locations

  • University of Leeds

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Study Population

Arm Description

Participants will be recruited and consented as part of routine care. All will trial supplement initially and have standard and additional measures taken. they will be followed up at month 1 and 2 collecting the primary outcomes (acceptability/palatability and dietary intake) and all initial measures repeated at month 3. Each participant will receive 3 months supply of gels to consume 2 per day.

Outcomes

Primary Outcome Measures

Percentage consumption of supplement gels
To measure through percentage (%) consumed by return of empty sachets over period of three months for each participant
Assessment of palatability of supplement gels
To measure using hedonic rating scale (like a lot, like a little, neither like nor dislike, dislike a little, dislike a lot) the appearance, aroma, taste, sweetness, texture mouth feel over the study period of 3 months for each participant
Measurement of dietary intake
To collect dietary intake at monthly intervals (baseline, one, two and three) using multiple pass 24 hour recalls conducted through face to face and telephone interviews at each time point recording all food and drink consumption. This is then analysed via Nutritics software, for each participant

Secondary Outcome Measures

Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)
Determine if any changes occur during 3-month period of supplementation
Body Mass Index
Weight and height will be combined to report BMI in kg/m2
Medical Research Council (MRC) breathlessness score
Will be assessed at initial and end points using the degree of breathlessness related activity. Scale is 1-5 (not troubled by breathless except on strenuous exercise to too breathless to leave the house or breathless when dressing and undressing) therefore the higher the score the worse the outcome
Vitamin D levels
To determine any changes during supplementation period
Number of infections over study period
Recorded as part of routine care the number of infections requiring antibiotic treatment either intravenous or oral) over the study period
Quality of life outcome SF-36 questionnaire
Completion of SF-36 quality of life questionnaire questionnaire which is used to determine generic health concepts of functional status and wellbeing. Completed at baseline and end of study period. The higher the score the better the health outcomes
Quality of Life outcome St Georges Respiratory Questionnaire
Completion of St Georges Respiratory questionnaire designed to measure health impairment in respiratory conditions. The results are calculated out of 100 with 100 being the worst possible outcomes and 0 the best.
Habitual activity estimation scale (HAES)
HAES scale which establishes daily activity levels at baseline and end of study for one typical weekday and one typical weekend day and using percentage of time spent on those activities out of 100%.
Hand grip strength dynamometry
Measurement of muscle functionality using hand grip dynamometer recorded as kg/f (kilograms of force)
Tricep skinfold measure
To determine any changes during supplementation period. Measured in millimetres using Harpenden skinfold calipers
Mid upper arm circumference
To determine any changes during supplementation period measured in centimetres, measured using an identified mid point between the acromion and olecranon processes using a tape measure
Six minute walk tests
To determine any changes during supplementation period
Bio electrical impedance analysis
Measure the electrical resistance of tissue to determine skeletal muscle mass reported as percentages and kilograms of participants at baseline and end point
Bio electrical impedance analysis
Measure the electrical resistance of tissue to determine fat mass (FM), reported as percentages and kilograms of participants at baseline and end point
Bio electrical impedance analysis
Measure the electrical resistance of tissue to determine fat free mass (FFM) reported as percentages and kilograms of participants at baseline and end point

Full Information

First Posted
August 24, 2023
Last Updated
September 8, 2023
Sponsor
University of Leeds
search

1. Study Identification

Unique Protocol Identification Number
NCT06028607
Brief Title
Feasibility of Consumption of Nutritional Supplementation in Primary Ciliary Dyskinesia
Acronym
(PCD)
Official Title
A Study to Explore the Feasibility of Consumption of Specific Complex Amino Acid Supplementation in the Form of an Oral Gel With Additional Leucine (40%) and Vitamin D in Patients With Primary Ciliary Dyskinesia: a Feasibility Study
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
May 17, 2021 (Actual)
Primary Completion Date
November 29, 2021 (Actual)
Study Completion Date
February 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Leeds

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Feasibility study to understand and gain preliminary information on tolerability and palatability of an oral nutritional supplement gel in a clinical Primary Ciliary Dyskinesia population. To determine if dietary intake is affected by the consumption of the nutritional supplement. Explore possible future outcome measures that could be important in determining impact of this nutritional intervention on this patient group. Participants will be provided with the gel supplements and alongside will have other standard and non standard care measures assessed lung function (FEV1%) number of exacerbation's during study period vitamin D status Bio impedance analysis (BIA) skin-fold measures (e.g. Tricep Skin-fold (TSF) Mid upper arm circumference (MUAC) Handgrip strength (HGS) quality of life measures 6-minute walking tests. Hypothesis Patients with PCD can successfully consume 2 gel supplements per day for a period of 3 months with no effect on dietary intake.
Detailed Description
This is a single site study taking place in a regional PCD clinic. Participants are screened via patient online data system by lead consultants and contacted via telephone to outline study and provide information and contact for queries. participant information sheet and pre-test guidance to be sent to participant prior to next scheduled clinic appointment. Participants attend their usual clinic appointments where routine care measures are completed by healthcare assistants and lead consultants. They then have opportunity to ask study coordinator any questions and complete consent for additional measures to be recorded. This is followed by a trial of the supplement to check for immediate tolerance followed by completion of consent to partake in study and initial palatability and acceptability records. 3 month supply of supplement is provided along with storage and consumption instructions (consume 1 at breakfast and 1 at lunch) a waste box to retain consumed sachets and record time of consumption. Additional telephone appointment made for 1 month to collect further palatability and acceptability along with dietary 24 hour recall. Participant is contacted via telephone at month 1 and 2 to recheck tolerability, acceptability, palatability and dietary intake. Participant can withdraw at any time if unable to continue to tolerate gel supplement. Participant returns to clinic at month 3 to repeat all standard measures, additional outcomes and palatability, acceptability and dietary intake. participant has completed study offered further dietary support through usual clinic route with registered dietitian. Participants can withdraw from the study at any point without giving any reason. Any data collected from them up until that point can be used in analysis. As this is a feasibility study a sample size calculation is not required. The PCD population within the regional clinic at time of recruitment is 54. All patients with PCD will be approached for inclusion in this feasibility if they meet the inclusion criteria until minimum number of 15 and maximum of 20 is reached.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Ciliary Dyskinesia

7. Study Design

Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
All service users will be approached for inclusion in feasibility
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Study Population
Arm Type
Other
Arm Description
Participants will be recruited and consented as part of routine care. All will trial supplement initially and have standard and additional measures taken. they will be followed up at month 1 and 2 collecting the primary outcomes (acceptability/palatability and dietary intake) and all initial measures repeated at month 3. Each participant will receive 3 months supply of gels to consume 2 per day.
Intervention Type
Dietary Supplement
Intervention Name(s)
QD-27
Intervention Description
Oral Protein gel with greater Leucine proportion and vitamin D Consumption of 2 each day for 3 months to be taken at breakfast and lunch
Primary Outcome Measure Information:
Title
Percentage consumption of supplement gels
Description
To measure through percentage (%) consumed by return of empty sachets over period of three months for each participant
Time Frame
3 months
Title
Assessment of palatability of supplement gels
Description
To measure using hedonic rating scale (like a lot, like a little, neither like nor dislike, dislike a little, dislike a lot) the appearance, aroma, taste, sweetness, texture mouth feel over the study period of 3 months for each participant
Time Frame
3 months
Title
Measurement of dietary intake
Description
To collect dietary intake at monthly intervals (baseline, one, two and three) using multiple pass 24 hour recalls conducted through face to face and telephone interviews at each time point recording all food and drink consumption. This is then analysed via Nutritics software, for each participant
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)
Description
Determine if any changes occur during 3-month period of supplementation
Time Frame
3 months
Title
Body Mass Index
Description
Weight and height will be combined to report BMI in kg/m2
Time Frame
3 months
Title
Medical Research Council (MRC) breathlessness score
Description
Will be assessed at initial and end points using the degree of breathlessness related activity. Scale is 1-5 (not troubled by breathless except on strenuous exercise to too breathless to leave the house or breathless when dressing and undressing) therefore the higher the score the worse the outcome
Time Frame
3 months
Title
Vitamin D levels
Description
To determine any changes during supplementation period
Time Frame
3 months
Title
Number of infections over study period
Description
Recorded as part of routine care the number of infections requiring antibiotic treatment either intravenous or oral) over the study period
Time Frame
3 months
Title
Quality of life outcome SF-36 questionnaire
Description
Completion of SF-36 quality of life questionnaire questionnaire which is used to determine generic health concepts of functional status and wellbeing. Completed at baseline and end of study period. The higher the score the better the health outcomes
Time Frame
3 months
Title
Quality of Life outcome St Georges Respiratory Questionnaire
Description
Completion of St Georges Respiratory questionnaire designed to measure health impairment in respiratory conditions. The results are calculated out of 100 with 100 being the worst possible outcomes and 0 the best.
Time Frame
3 months
Title
Habitual activity estimation scale (HAES)
Description
HAES scale which establishes daily activity levels at baseline and end of study for one typical weekday and one typical weekend day and using percentage of time spent on those activities out of 100%.
Time Frame
3 months
Title
Hand grip strength dynamometry
Description
Measurement of muscle functionality using hand grip dynamometer recorded as kg/f (kilograms of force)
Time Frame
3 months
Title
Tricep skinfold measure
Description
To determine any changes during supplementation period. Measured in millimetres using Harpenden skinfold calipers
Time Frame
3 months
Title
Mid upper arm circumference
Description
To determine any changes during supplementation period measured in centimetres, measured using an identified mid point between the acromion and olecranon processes using a tape measure
Time Frame
3 months
Title
Six minute walk tests
Description
To determine any changes during supplementation period
Time Frame
3 months
Title
Bio electrical impedance analysis
Description
Measure the electrical resistance of tissue to determine skeletal muscle mass reported as percentages and kilograms of participants at baseline and end point
Time Frame
3 months
Title
Bio electrical impedance analysis
Description
Measure the electrical resistance of tissue to determine fat mass (FM), reported as percentages and kilograms of participants at baseline and end point
Time Frame
3 months
Title
Bio electrical impedance analysis
Description
Measure the electrical resistance of tissue to determine fat free mass (FFM) reported as percentages and kilograms of participants at baseline and end point
Time Frame
3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: People aged 17 or over with a confirmed diagnosis of Primary ciliary dyskinesia PCD (nasal brushings) People who attend the Leeds Regional PCD Clinics People who have capacity to give informed consent Exclusion Criteria: People who are pregnant Those with existing co-morbidities such as malignancy People with connective tissue disorders and immunoglobulin deficiencies People with renal insufficiency r
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Daniel G Peckham
Organizational Affiliation
Univeristy Of Leeds
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Leeds
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS2 9NL
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
11124729
Citation
Ware JE Jr. SF-36 health survey update. Spine (Phila Pa 1976). 2000 Dec 15;25(24):3130-9. doi: 10.1097/00007632-200012150-00008. No abstract available.
Results Reference
background
PubMed Identifier
1759018
Citation
Jones PW, Quirk FH, Baveystock CM. The St George's Respiratory Questionnaire. Respir Med. 1991 Sep;85 Suppl B:25-31; discussion 33-7. doi: 10.1016/s0954-6111(06)80166-6.
Results Reference
background

Learn more about this trial

Feasibility of Consumption of Nutritional Supplementation in Primary Ciliary Dyskinesia

We'll reach out to this number within 24 hrs