Primary Ciliary Dyskinesia(PCD)NCT06028607
Feasibility of Consumption of Nutritional Supplementation in Primary Ciliary Dyskinesia
A study testing QD-27 in people with Primary Ciliary Dyskinesia, run by University of Leeds at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 17 years and older
- Conditions the study is for
- Primary Ciliary Dyskinesia
- What is being tested?
- QD-27
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 17 May 2021to 28 Feb 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
University of Leeds
Leeds, West Yorkshire, LS2 9NL
About this study
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
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.
Read the 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.
- Conditions
- Primary Ciliary Dyskinesia
- Being tested
- QD-27
- Medical subject headings
- Ciliary Motility Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QD-27Dietary supplement
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
| Study group | Role | What they receive |
|---|---|---|
| Study Population | Other | 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. Receives: Dietary Supplement: QD-27 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage consumption of supplement gels
To measure through percentage (%) consumed by return of empty sachets over period of three months for each participant
Measured: 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
Measured: 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
Measured: 3 months
Secondary outcomes (15)
Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)
Determine if any changes occur during 3-month period of supplementation
Measured: 3 months
Body Mass Index
Weight and height will be combined to report BMI in kg/m2
Measured: 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
Measured: 3 months
Vitamin D levels
To determine any changes during supplementation period
Measured: 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
Measured: 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
Measured: 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.
Measured: 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%.
Measured: 3 months
Hand grip strength dynamometry
Measurement of muscle functionality using hand grip dynamometer recorded as kg/f (kilograms of force)
Measured: 3 months
Tricep skinfold measure
To determine any changes during supplementation period. Measured in millimetres using Harpenden skinfold calipers
Measured: 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
Measured: 3 months
Six minute walk tests
To determine any changes during supplementation period
Measured: 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
Measured: 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
Measured: 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
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2023
- Study start17 May 2021
- Primary completion29 Nov 2021
- Study completion28 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Ciliary DyskinesiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All service users will be approached for inclusion in feasibility
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Leeds
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Professor Daniel G Peckham, University of Leeds
- Investigators
- Daniel G Peckham, Principal investigator, Univeristy Of Leeds
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06028607
- Sponsor's protocol ID
- Version 1.0
- Acronym
- (PCD)
- Status last verified
- Sep 2023
- First submitted
- 24 Aug 2023
- Last updated
- 13 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 11124729Jones 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.
PubMed 1759018
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06028607
- Acronym / sponsor ID
- (PCD) / Version 1.0
- Conditions
- Primary Ciliary Dyskinesia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University of LeedsUniversity, hospital or other
- Study start
- 17 May 2021 (actual)
- Primary completion
- 29 Nov 2021 (actual)
- Study completion
- 28 Feb 2022 (actual)
- First posted
- 8 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All service users will be approached for inclusion in feasibility
| Arm | Type | Receives |
|---|---|---|
| Study Population | Other | Dietary Supplement: QD-27Participants 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. |
Who may qualify
- Ages
- 17 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QD-27Oral Protein gel with greater Leucine proportion and vitamin D Consumption of 2 each day for 3 months to be taken at breakfast and lunch | Dietary supplement | Study Population |
Primary outcome
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
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
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
15 secondary outcomes
Lung Function (Forced Expiratory Volume1 %, Forced Vital Capacity %)
Time frame: 3 months
Body Mass Index
Time frame: 3 months
Medical Research Council (MRC) breathlessness score
Time frame: 3 months
Vitamin D levels
Time frame: 3 months
Number of infections over study period
Time frame: 3 months
Quality of life outcome SF-36 questionnaire
Time frame: 3 months
Quality of Life outcome St Georges Respiratory Questionnaire
Time frame: 3 months
Habitual activity estimation scale (HAES)
Time frame: 3 months
Hand grip strength dynamometry
Time frame: 3 months
Tricep skinfold measure
Time frame: 3 months
Mid upper arm circumference
Time frame: 3 months
Six minute walk tests
Time frame: 3 months
Bio electrical impedance analysis
Time frame: 3 months
Bio electrical impedance analysis
Time frame: 3 months
Bio electrical impedance analysis
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
- Background: PubMed 11124729Ware 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.
- Background: PubMed 1759018Jones 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel G Peckham principal investigator
Univeristy Of Leeds
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing QD-27 in people with Primary Ciliary Dyskinesia, run by University of Leeds at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage consumption of supplement gels (time frame: 3 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06028607, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2023.