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

• ClinicalTrials.gov Identifier: NCT06028607
• Recruitment Status: Completed
• First Posted: September 8, 2023
• Last Update Posted: September 13, 2023
• Sponsor: University of Leeds
• Information provided by (Responsible Party): Professor Daniel G Peckham, University of Leeds

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.

Condition or disease	Intervention/treatment	Phase
Primary Ciliary Dyskinesia	Dietary Supplement: QD-27	Not Applicable

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: All service users will be approached for inclusion in feasibility
• Masking: None (Open Label)
• Primary Purpose: Other
• 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
• Actual Study Start Date: May 17, 2021
• Actual Primary Completion Date: November 29, 2021
• Actual Study Completion Date: February 28, 2022

Arms and Interventions

Arm

Intervention/treatment

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

Outcome Measures

Primary Outcome Measures :

1. Percentage consumption of supplement gels [ Time Frame: 3 months ]
   To measure through percentage (%) consumed by return of empty sachets over period of three months for each participant
2. Assessment of palatability of supplement gels [ Time Frame: 3 months ]
   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. Measurement of dietary intake [ Time Frame: 3 months ]
   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 :

1. Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %) [ Time Frame: 3 months ]
   Determine if any changes occur during 3-month period of supplementation
2. Body Mass Index [ Time Frame: 3 months ]
   Weight and height will be combined to report BMI in kg/m2
3. Medical Research Council (MRC) breathlessness score [ Time Frame: 3 months ]
   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
4. Vitamin D levels [ Time Frame: 3 months ]
   To determine any changes during supplementation period
5. Number of infections over study period [ Time Frame: 3 months ]
   Recorded as part of routine care the number of infections requiring antibiotic treatment either intravenous or oral) over the study period
6. Quality of life outcome SF-36 questionnaire [ Time Frame: 3 months ]
   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
7. Quality of Life outcome St Georges Respiratory Questionnaire [ Time Frame: 3 months ]
   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.
8. Habitual activity estimation scale (HAES) [ Time Frame: 3 months ]
   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%.
9. Hand grip strength dynamometry [ Time Frame: 3 months ]
   Measurement of muscle functionality using hand grip dynamometer recorded as kg/f (kilograms of force)
10. Tricep skinfold measure [ Time Frame: 3 months ]
    To determine any changes during supplementation period. Measured in millimetres using Harpenden skinfold calipers
11. Mid upper arm circumference [ Time Frame: 3 months ]
    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
12. Six minute walk tests [ Time Frame: 3 months ]
    To determine any changes during supplementation period
13. Bio electrical impedance analysis [ Time Frame: 3 months ]
    Measure the electrical resistance of tissue to determine skeletal muscle mass reported as percentages and kilograms of participants at baseline and end point
14. Bio electrical impedance analysis [ Time Frame: 3 months ]
    Measure the electrical resistance of tissue to determine fat mass (FM), reported as percentages and kilograms of participants at baseline and end point
15. Bio electrical impedance analysis [ Time Frame: 3 months ]
    Measure the electrical resistance of tissue to determine fat free mass (FFM) reported as percentages and kilograms of participants at baseline and end point

Eligibility Criteria

• Ages Eligible for Study: 17 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

United Kingdom

University of Leeds

Leeds, West Yorkshire, United Kingdom, LS2 9NL

Sponsors and Collaborators

University of Leeds

Investigators

• Principal Investigator: Daniel G Peckham; Univeristy Of Leeds

More Information

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.

• Responsible Party: Professor Daniel G Peckham, Professor of Respiratory Medicine, University of Leeds
• ClinicalTrials.gov Identifier: NCT06028607
• Other Study ID Numbers: Version 1.0
• First Posted: September 8, 2023
• Last Update Posted: September 13, 2023
• Last Verified: September 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Ciliary Motility Disorders; Dyskinesias; Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Genetic Diseases, Inborn