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

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

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.

Study Overview

• Status: Completed
• Conditions: Primary Ciliary Dyskinesia
• Intervention / Treatment: Dietary supplement: QD-27

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.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • West Yorkshire
    • Leeds, West Yorkshire, United Kingdom, LS2 9NL
      • University of Leeds

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Study Population; 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.

Dietary supplement: QD-27; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage consumption of supplement gels	To measure through percentage (%) consumed by return of empty sachets over period of three months for each participant	3 months
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	3 months
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	3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)	Determine if any changes occur during 3-month period of supplementation	3 months
Body Mass Index	Weight and height will be combined to report BMI in kg/m2	3 months
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	3 months
Vitamin D levels	To determine any changes during supplementation period	3 months
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	3 months
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	3 months
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.	3 months
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%.	3 months
Hand grip strength dynamometry	Measurement of muscle functionality using hand grip dynamometer recorded as kg/f (kilograms of force)	3 months
Tricep skinfold measure	To determine any changes during supplementation period. Measured in millimetres using Harpenden skinfold calipers	3 months
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	3 months
Six minute walk tests	To determine any changes during supplementation period	3 months
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	3 months
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	3 months
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	3 months

Collaborators and Investigators

• Sponsor: University of Leeds
• Investigators: Principal Investigator: Daniel G Peckham, Univeristy of Leeds

Publications and helpful links

General Publications

• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (Actual): May 17, 2021
• Primary Completion (Actual): November 29, 2021
• Study Completion (Actual): February 28, 2022

Study Registration Dates

• First Submitted: August 24, 2023
• First Submitted That Met QC Criteria: August 31, 2023
• First Posted (Actual): September 8, 2023

Study Record Updates

• Last Update Posted (Actual): September 13, 2023
• Last Update Submitted That Met QC Criteria: September 8, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Respiratory Tract Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Otorhinolaryngologic Diseases; Movement Disorders; Abnormalities, Multiple; Ciliopathies; Dyskinesias; Ciliary Motility Disorders
• Other Study ID Numbers: Version 1.0

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No