Clinical trial NCT06028607: Feasibility of Consumption of Nutritional Supplementation in Primary Ciliary Dyskinesia
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
Overall status: COMPLETED
Phase: NA
Conditions: Primary Ciliary Dyskinesia
Lead sponsor: University of Leeds
Sponsor class: OTHER
First posted: 2023-09-08
Enrollment: 15
Sex: ALL
Minimum age (years): 17
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: All service users will be approached for inclusion in feasibility
Primary purpose: OTHER

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.

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

Primary outcomes:
measure: Percentage consumption of supplement gels
timeFrame: 3 months
measure: Assessment of palatability of supplement gels
timeFrame: 3 months
measure: Measurement of dietary intake
timeFrame: 3 months
Secondary outcomes:
measure: Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)
timeFrame: 3 months
measure: Body Mass Index
timeFrame: 3 months
measure: Medical Research Council (MRC) breathlessness score
timeFrame: 3 months
measure: Vitamin D levels
timeFrame: 3 months
measure: Number of infections over study period
timeFrame: 3 months
measure: Quality of life outcome SF-36 questionnaire
timeFrame: 3 months
measure: Quality of Life outcome St Georges Respiratory Questionnaire
timeFrame: 3 months
measure: Habitual activity estimation scale (HAES)
timeFrame: 3 months
measure: Hand grip strength dynamometry
timeFrame: 3 months
measure: Tricep skinfold measure
timeFrame: 3 months
measure: Mid upper arm circumference
timeFrame: 3 months
measure: Six minute walk tests
timeFrame: 3 months
measure: Bio electrical impedance analysis
timeFrame: 3 months
measure: Bio electrical impedance analysis
timeFrame: 3 months
measure: Bio electrical impedance analysis
timeFrame: 3 months
Other outcomes:
Locations:
city: Leeds
country: United Kingdom
facility: University of Leeds
state: West Yorkshire
Study officials:
affiliation: Univeristy Of Leeds
name: Daniel G Peckham
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.