Primary Ciliary DyskinesiaNCT01246258
Otolith Function in Patients With Primary Ciliary Dyskinesia
An observational study of Primary Ciliary Dyskinesia, run by Imperial College Healthcare NHS Trust at 1 site in United Kingdom.
Trial summary
The purpose of this study is to determine whether patients with primary ciliary dyskinesia (PCD) have reduced or absent otolith function.The otolith system is a specific part of the inner ear vestibular (balance) system that detects linear movement. Read more about this study
- Who is it for?
- Ages 16 to 30 years
- Conditions the study is for
- Primary Ciliary Dyskinesia
- What is being tested?
- Vestibular evoked myogenic potentials (VEMPs) and Utricular centrifugation test
- How many people
- 5 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2010to Dec 2013
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 16 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Ten patients from the PCD clinic at the Royal Brompton Hospital, London.
Who may be able to join
- aged 16-30 years
- Confirmed diagnosis of PCD under the care of the PCD team at the Royal Brompton Hospital
Who cannot join
- Aged under 16 years
- Unconfirmed or "suspected" PCD
- Previous history of balance disorders or diagnoses
- Previous history of sensorineural hearing loss
- Previous middle or inner ear surgery (other than grommet insertion)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Otolith Function in Patients With Primary Ciliary Dyskinesia: a Pilot Study
The purpose of this study is to determine whether patients with primary ciliary dyskinesia (PCD) have reduced or absent otolith function.The otolith system is a specific part of the inner ear vestibular (balance) system that detects linear movement.
Read the detailed description
Primary ciliary dyskinesia (PCD) is a genetically inherited condition. It is due to structural abnormalities of cilia, which are microscopic hairs found in organs and cells throughout the body. Patients with this condition typically develop upper respiratory tract symptoms such as sinusitis and glue ear, lower respiratory tract problems such as recurrent chest infections, and fertility problems. There is currently no evidence that patients with PCD have a higher incidence of balance problems. However, recent animal studies have shown that cilia may also be important in the development of part of the inner ear balance (vestibular) system, specifically the part that detects linear movement known as the otolith system.
The investigators hope to determine whether patients with PCD have absent or reduced otolith function compared to the normal population. Balance problems are not currently screened for in PCD patients, and could be unrecognized and therefore untreated. It is also possible that PCD patients have compensated for absent otolith function and so are unaffected under normal circumstances; any additional insult to their balance system would cause more pronounced difficulties than expected and treatment might need to reflect this.
- Conditions
- Primary Ciliary Dyskinesia
- Being tested
- Vestibular evoked myogenic potentials (VEMPs), Utricular centrifugation test
- Keywords
- primary ciliary dyskinesia, otolith, vestibular tests
- 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.
Vestibular evoked myogenic potentials (VEMPs)Other
Standard test of balance function
Utricular centrifugation testOther
Standard test of balance function
| Study group | Role | What they receive |
|---|---|---|
| Test group | Standard tests of balance function Receives: Other: Vestibular evoked myogenic potentials (VEMPs); Other: Utricular centrifugation test |
What is being measured
The primary outcome is the main question the study is designed to answer.
Vestibular Evoked Myogenic Potentials (VEMPs)
These are the balance tests that specifically assess the otolith organ.
Measured: one day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2010 (estimated)
- Study startSep 2010
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)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
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Imperial College Healthcare NHS Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Joanne Rimmer, MA FRCS, Principal investigator, Imperial College Healthcare NHS TrustJonny Harcourt, MA FRCS, Study director, Imperial College Healthcare NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01246258
- Sponsor's protocol ID
- JROHH0046
- Status last verified
- Aug 2014
- First submitted
- 22 Nov 2010
- Last updated
- 6 Apr 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Rimmer J, Patel M, Agarwal K, Hogg C, Arshad Q, Harcourt J. Peripheral vestibular dysfunction in patients with primary ciliary dyskinesia: abnormal otoconial development? Otol Neurotol. 2015 Apr;36(4):662-9. doi: 10.1097/MAO.0000000000000592.
PubMed 25226371
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Otolith Function in Patients With Primary Ciliary Dyskinesia: a Pilot Study
- ClinicalTrials.gov ID
- NCT01246258
- Acronym / sponsor ID
- JROHH0046
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- primary ciliary dyskinesia, otolith, vestibular tests
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Imperial College Healthcare NHS TrustUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 23 Nov 2010 (estimated)
- Results
- Posted 6 Apr 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether patients with primary ciliary dyskinesia (PCD) have reduced or absent otolith function.The otolith system is a specific part of the inner ear vestibular (balance) system that detects linear movement.
Detailed description
Primary ciliary dyskinesia (PCD) is a genetically inherited condition. It is due to structural abnormalities of cilia, which are microscopic hairs found in organs and cells throughout the body. Patients with this condition typically develop upper respiratory tract symptoms such as sinusitis and glue ear, lower respiratory tract problems such as recurrent chest infections, and fertility problems. There is currently no evidence that patients with PCD have a higher incidence of balance problems. However, recent animal studies have shown that cilia may also be important in the development of part of the inner ear balance (vestibular) system, specifically the part that detects linear movement known as the otolith system.
The investigators hope to determine whether patients with PCD have absent or reduced otolith function compared to the normal population. Balance problems are not currently screened for in PCD patients, and could be unrecognized and therefore untreated. It is also possible that PCD patients have compensated for absent otolith function and so are unaffected under normal circumstances; any additional insult to their balance system would cause more pronounced difficulties than expected and treatment might need to reflect this.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Test group | – | Other: Vestibular evoked myogenic potentials (VEMPs), Other: Utricular centrifugation testStandard tests of balance function |
Who may qualify
- Ages
- 16 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Ten patients from the PCD clinic at the Royal Brompton Hospital, London.
Key inclusion criteria (2 listed)
- aged 16-30 years
- Confirmed diagnosis of PCD under the care of the PCD team at the Royal Brompton Hospital
Key exclusion criteria (5 listed)
- Aged under 16 years
- Unconfirmed or "suspected" PCD
- Previous history of balance disorders or diagnoses
- Previous history of sensorineural hearing loss
- Previous middle or inner ear surgery (other than grommet insertion)
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 |
|---|---|---|
| Vestibular evoked myogenic potentials (VEMPs)Standard test of balance function | Other | Test group |
| Utricular centrifugation testStandard test of balance function | Other | Test group |
Primary outcome
Vestibular Evoked Myogenic Potentials (VEMPs)
Time frame: one day
These are the balance tests that specifically assess the otolith organ.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 25226371Rimmer J, Patel M, Agarwal K, Hogg C, Arshad Q, Harcourt J. Peripheral vestibular dysfunction in patients with primary ciliary dyskinesia: abnormal otoconial development? Otol Neurotol. 2015 Apr;36(4):662-9. doi: 10.1097/MAO.0000000000000592.
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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.
Joanne Rimmer, MA FRCS principal investigator
Imperial College Healthcare NHS Trust
Jonny Harcourt, MA FRCS study director
Imperial College Healthcare NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Ciliary Dyskinesia, run by Imperial College Healthcare NHS Trust 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 2 inclusion and 5 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 6 Apr 2016. 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?
Yes. Results are posted in the registry (first posted 6 Apr 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Vestibular Evoked Myogenic Potentials (VEMPs) (time frame: one day). 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 NCT01246258, which also lists 1 linked publication.
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 6 Apr 2016.