Primary Ciliary Dyskinesia (PCD)PCDregistryNCT02419365
International Primary Ciliary Dyskinesia (PCD) Registry
An observational study of Primary Ciliary Dyskinesia (PCD), run by University Hospital Muenster at 2 sites in Germany.
Trial summary
The purpose of the international prospective PCD Patient Registry is to systematically measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-center trials. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Ciliary Dyskinesia (PCD)
- How many people
- 2,000 participants
- Where
- 2 sitesGermany
- Study dates
- Jan 2014to Dec 2030
- How long will I take part?
- 20 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with a diagnosis of Primary Ciliary Dyskinesia.
Who may be able to join
- Patients of any age who fulfil the diagnostic criteria below are eligible:
- Clinical presentation consistent with PCD and consistent findings specific for PCD in at least two of the following methods:
- high frequency video microscopic finding transmission electron microscopy finding immunofluorescence finding low nasal NO concentration/production demonstration of biallelic disease-causing mutations by genotyping
- Given the complexity of diagnosing PCD, it is anticipated that not all patients will meet these definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry. These cases usually are considered to have a possible PCD diagnosis with exceptions made on an individual basis.
- Exclusion Criteria Failure or unwillingness to give written informed consent. Missing qualification to perform legal acts or insufficient cognitive ability to give informed consent. A second disease is no exclusion criteria for including data in the PCD-Registry, especially since it is one of the fundamental goals, to learn about co-morbidities.
Who cannot join
None listed.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Germany2 sites, 2 recruiting
- Recruiting
University Hospital Münster
Münster, North Rhine-Westphalia, 48149
Heymut Omran, Prof. Dr. MD, 004925183 ext. 41097, PCDregistry.eu@ukmuenster.de
Simone Helms, +4925183 ext. 48358, Simone.Helms@ukmuenster.de
- Recruiting
University Hospital Muenster, Department of General Pediatrics
Münster, 48149
Petra Pennekamp, PhD, +4925183 ext. 41097, petra.pennekamp@ukmuenster.de
Simone Helms, +4925183 ext. 48358, Simone.Helms@ukmuenster.de
About this study
Official title: International Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
The purpose of the international prospective PCD Patient Registry is to systematically measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-center trials.
This International PCD Registry is also part of the European Reference Network ERN-LUNG. We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
Read the detailed description
PCD is a rare disorder of mucociliary clearance caused by defective hair like organelles (cilia). The purpose of the PCD Registry is to measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-centre trials.
The PCD Registry is part of the European Reference Network for Rare Lung Diseases ERN-LUNG (https://ern-lung.eu/). We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
How can the data be entered to the registry? The data can either be entered into the PCD Registry directly after login, or imported via a specific *CSV file that we provide in advance (download option after Log In or e-mail request to PCDregistry.eu@ukmuenster.de, Simone.Helms@ukmuenster.de or Petra.Pennekamp@ukmuenster.de).
Under special circumstances (e.g. import of existing datasets from other registries/cohorts) we provide support to enter your data. The PCD Registry is accessible from the web. Each authorised operator is able to enter the data for their country/centre and export own center specific data to different formats (CSV/Microsoft Excel, PDF, SAS, Stata, R, ODM or SPSS).
A highly protected system will ensure data protection through the encryption of data. Identifying patient data are not collected in the PCD Registry.
Where are the data stored? At the University of Muenster, Institute of Medical Informatics - Informatics for personalised medicine (IMI). A server is located in secure premises, where access is allowed to authorized personnel only. Data storage is in agreement with EU data protection legislation.
How to become a PCD Registry Centre? In order to get assigned as a referring centre please contact the administration under PCDregistry.eu@ukmuenster.de, Simone.Helms@ukmuenster.de and Petra.Pennekamp@ukmuenster.de.
Information for PCD Patients:
Why do we need a PCD Registry? Registries are the only way to pool data in the field of Rare Diseases (RD) such as PCD in order to achieve a sufficient sample size for epidemiological and/or clinical research. RD Registries such as the PCD Registry constitute key instruments to develop clinical research in the field of RD, to improve patient care and healthcare planning. RD Registries such as the PCD Registry are vital to assess the feasibility of clinical trials, to facilitate the planning of appropriate clinical trials and to support the enrolment of patients.
Why should I as a patient participate? Patient involvement is the key factor in the establishment of any patient registry, especially RD Registries. For the purpose of the above aims, we need as many patients in the PCD Registry as possible in order to make sure that data are representative of the whole spectrum of the disease and to have enough patients to render research possible - research that may benefit you or other patients in the future.
I you - as a patient - have further questions please do not hesitate to contact us.
Which data are collected? We collect demographic data (e.g. current age, sex, age, diagnostic findings and symptoms at diagnosis) but also clinical data which are recorded (e.g. lung function, weight, height, infections, treatment and complications). The data are collected using a common set of definitions, following the recommendations of the EU Expert Committee on Rare Diseases (EUCERD). This ensured international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
Ways to register:
Registration will be performed by the treating physician via password-secured access. The access authorization, as well as the necessary password will be passed on by the PCD Registry administrator (Simone.Helms@ukmuenster.de).
Information about data security:
To ensure that patients are not accidentally registered twice and to take into account a changing of caring physician, personal data such as month and year of birth have to be included in the first step of patient registration. These will be directly transferred into a Patient identification-Code (PID) and automatically generated pseudonym. Tracking a patient's identification with the PID is impossible. Other identifying data such name, initials or day of birth will not be nor recorded. Data saving underlies the regulations for data security laws and the medical confidentiality. International regulations for good clinical practice (GCP) always apply. Unauthorized third parties will not have access to registry data.
Inclusion criteria:
The PCD Patient Registry is a longitudinal study. It recruits all prevalent subjects with PCD and all subsequent incident cases. Patients who fulfil the diagnostic criteria below should be included in the registry
- Clinical presentation consistent with PCD and
- Confirmation of the diagnose by at least two of the following methods
- abnormally low nasal NO concentration/production
- abnormal high frequency video microscopic (HVMA) finding
- abnormal immunofluorescence (IF) finding
- demonstration of biallelic disease-causing mutations by genotyping (Note: in those cases, where genetic diagnosis is not possible because of financial or structural issues, genetic diagnosis can be offered in the framework of a research project)
- abnormal transmission electron microscopy (TEM) finding (Note: if only high frequency video microscopy and nasal NO concentration/production are abnormal, high frequency video microscopy should be repeated at least three times and show the same abnormal results each time)
Given the complexity of diagnosing PCD, not all patients will meet the above mentioned definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry.
Ethics and Patient Confidentiality:
Obtaining the necessary patient consents is a prerequisite for entering data in the PCD Registry. As legislation differs in each participating country, it is important to check the requirements with your local Data Protection Officers and Ethics Committees. If you have any questions, please contact PCDregistry.eu@ukmuenster.de or Simone.Helms@ukmuenster.de. You will get support for the Ethics Committee approval procedure, e.g. sample consent forms for Patient Information and Consent. Patients can only be entered into the PCD Registry once you have received their signed, informed consent to do so. Please store all the consent forms that your patients have signed safely at your local institution. It is the responsibility of the reporting centres/countries/registries to have the acquired permissions to export/report data to the PCD Registry.
Data Security:
Due to data protection regulations, the data stored in the server of the PCD Registry must be anonymous (i.e. the patient must not be identifiable). Therefore, the authorised operator has to keep a logbook with the identifying patient data and the code generated by the registry software. It is your responsibility to make sure that access to this list of names and matching patient codes is restricted to authorized personnel only. Furthermore, please note that if your list of matching codes and full names is lost, it will be impossible to retrieve the data identification by means of the PCD Registry software. Moreover, data are encrypted (i.e. not de-codable) when transmitted. The PCD Registry is protected according to EU Data Protection legislation, both physically and technically, and backup is secured.
Funding:
The establishment of the PCD Registry was part of the EU funded BESTCILIA project (FP7 2007-2013) under grant agreement no 305404 (BESTCILIA). Funding continues within several projects including REGISTRY WAREHOUSE (HORIZON2020, GA no 777295)
About us:
The PCD Registry has been designed and established by the BESTCILIA consortium headed by Heymut Omran, University Children's Hospital Muenster, Department of General Pediatrics, Muenster, Germany and is part of the European Reference Network on Rare Lung Diseases ERN-LUNG. For more information, please contact PCDregistry.eu@ukmuenster.de Simone.Helms@ukmuenster.de, Petra.Pennekamp@ukmuenster.de or Johanna.Raidt@ukmuenster.de
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Keywords
- Primary Ciliary Dyskinesia (PCD)
- 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.
| Study group | Role | What they receive |
|---|---|---|
| PCD | Individuals with Primary Ciliary Dyskinesia will be assessed in a pure observational design without intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
descriptive analysis
Descriptive statistical methods, i.e. frequency tables, location and scale statistics and graphical presentations will be applied to analyse: age at diagnosis, family history (consanguinity), associated malformations/diseases, laterality defects, clinical manifestations (otitis media, rhinosinusitis, pneumonia, bronchiectasis, neonatal respiratory distress syndrome), microbiological results, diagnostic findings (video microscopy, electron microscopy, nasal NO, immunofluorescence analysis), lung function, radiological findings and therapeutic measures (inhalation therapy, antibiotics, oxygen, ventilation, upper airways surgery, lung surgery). Furthermore, quality of life will be assessed using QOL-PCD, a disease specific questionnaire.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2015 (estimated)
- Study startJan 2014
- Primary completionDec 2030 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Ciliary Dyskinesia (PCD)Studies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
- Target duration
- 20 Years
Sponsor and investigators
- Lead sponsor
- University Hospital Muenster
- Sponsor type
- University, hospital or other
- Collaborators
- European Commission, University of Nicosia, Copenhagen University Hospital, Rigshospitalet, KU Leuven, Hannover Medical School, Attikon Hospital, Amsterdam UMC, location VUmc, University of Bern, University of Southampton, Royal Brompton & Harefield NHS Foundation Trust, University of North Carolina, Ruhr University of Bochum, Federico II University, Hospital Vall d'Hebron, Medical University of Vienna, Marmara University, University Hospital, Martin, University of Pisa, Assistance Publique - Hôpitaux de Paris, University of Alberta, University of Giessen
- Responsible party
- Principal investigator, Petra Pennekamp, University Hospital Muenster
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02419365
- Sponsor's protocol ID
- PCD Registry
- Acronym
- PCDregistry
- Status last verified
- Nov 2022
- First submitted
- 3 Feb 2014
- Last updated
- 28 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
Werner C, Lablans M, Ataian M, Raidt J, Wallmeier J, Grosse-Onnebrink J, Kuehni CE, Haarman EG, Leigh MW, Quittner AL, Lucas JS, Hogg C, Witt M, Priftis KN, Yiallouros P, Nielsen KG, Santamaria F, Uckert F, Omran H. An international registry for primary ciliary dyskinesia. Eur Respir J. 2016 Mar;47(3):849-59. doi: 10.1183/13993003.00776-2015. Epub 2015 Dec 8.
PubMed 26659107
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
- ClinicalTrials.gov ID
- NCT02419365
- Acronym / sponsor ID
- PCDregistry / PCD Registry
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Keywords
- Primary Ciliary Dyskinesia (PCD)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- University Hospital MuensterUniversity, hospital or other
- Collaborators
- European Commission, University of Nicosia, Copenhagen University Hospital, Rigshospitalet, KU Leuven, Hannover Medical School, Attikon Hospital, Amsterdam UMC, location VUmc, University of Bern, University of Southampton, Royal Brompton & Harefield NHS Foundation Trust, University of North Carolina, Ruhr University of Bochum, Federico II University, Hospital Vall d'Hebron, Medical University of Vienna, Marmara University, University Hospital, Martin, University of Pisa, Assistance Publique - Hôpitaux de Paris, University of Alberta, University of Giessen
- Study start
- Jan 2014
- Primary completion
- Dec 2030 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 17 Apr 2015 (estimated)
- 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.
The purpose of the international prospective PCD Patient Registry is to systematically measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-center trials.
This International PCD Registry is also part of the European Reference Network ERN-LUNG. We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
Detailed description
PCD is a rare disorder of mucociliary clearance caused by defective hair like organelles (cilia). The purpose of the PCD Registry is to measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-centre trials.
The PCD Registry is part of the European Reference Network for Rare Lung Diseases ERN-LUNG (https://ern-lung.eu/). We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
How can the data be entered to the registry? The data can either be entered into the PCD Registry directly after login, or imported via a specific *CSV file that we provide in advance (download option after Log In or e-mail request to PCDregistry.eu@ukmuenster.de, Simone.Helms@ukmuenster.de or Petra.Pennekamp@ukmuenster.de).
Under special circumstances (e.g. import of existing datasets from other registries/cohorts) we provide support to enter your data. The PCD Registry is accessible from the web. Each authorised operator is able to enter the data for their country/centre and export own center specific data to different formats (CSV/Microsoft Excel, PDF, SAS, Stata, R, ODM or SPSS).
A highly protected system will ensure data protection through the encryption of data. Identifying patient data are not collected in the PCD Registry.
Where are the data stored? At the University of Muenster, Institute of Medical Informatics - Informatics for personalised medicine (IMI). A server is located in secure premises, where access is allowed to authorized personnel only. Data storage is in agreement with EU data protection legislation.
How to become a PCD Registry Centre? In order to get assigned as a referring centre please contact the administration under PCDregistry.eu@ukmuenster.de, Simone.Helms@ukmuenster.de and Petra.Pennekamp@ukmuenster.de.
Information for PCD Patients:
Why do we need a PCD Registry? Registries are the only way to pool data in the field of Rare Diseases (RD) such as PCD in order to achieve a sufficient sample size for epidemiological and/or clinical research. RD Registries such as the PCD Registry constitute key instruments to develop clinical research in the field of RD, to improve patient care and healthcare planning. RD Registries such as the PCD Registry are vital to assess the feasibility of clinical trials, to facilitate the planning of appropriate clinical trials and to support the enrolment of patients.
Why should I as a patient participate? Patient involvement is the key factor in the establishment of any patient registry, especially RD Registries. For the purpose of the above aims, we need as many patients in the PCD Registry as possible in order to make sure that data are representative of the whole spectrum of the disease and to have enough patients to render research possible - research that may benefit you or other patients in the future.
I you - as a patient - have further questions please do not hesitate to contact us.
Which data are collected? We collect demographic data (e.g. current age, sex, age, diagnostic findings and symptoms at diagnosis) but also clinical data which are recorded (e.g. lung function, weight, height, infections, treatment and complications). The data are collected using a common set of definitions, following the recommendations of the EU Expert Committee on Rare Diseases (EUCERD). This ensured international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
Ways to register:
Registration will be performed by the treating physician via password-secured access. The access authorization, as well as the necessary password will be passed on by the PCD Registry administrator (Simone.Helms@ukmuenster.de).
Information about data security:
To ensure that patients are not accidentally registered twice and to take into account a changing of caring physician, personal data such as month and year of birth have to be included in the first step of patient registration. These will be directly transferred into a Patient identification-Code (PID) and automatically generated pseudonym. Tracking a patient's identification with the PID is impossible. Other identifying data such name, initials or day of birth will not be nor recorded. Data saving underlies the regulations for data security laws and the medical confidentiality. International regulations for good clinical practice (GCP) always apply. Unauthorized third parties will not have access to registry data.
Inclusion criteria:
The PCD Patient Registry is a longitudinal study. It recruits all prevalent subjects with PCD and all subsequent incident cases. Patients who fulfil the diagnostic criteria below should be included in the registry
- Clinical presentation consistent with PCD and
- Confirmation of the diagnose by at least two of the following methods
- abnormally low nasal NO concentration/production
- abnormal high frequency video microscopic (HVMA) finding
- abnormal immunofluorescence (IF) finding
- demonstration of biallelic disease-causing mutations by genotyping (Note: in those cases, where genetic diagnosis is not possible because of financial or structural issues, genetic diagnosis can be offered in the framework of a research project)
- abnormal transmission electron microscopy (TEM) finding (Note: if only high frequency video microscopy and nasal NO concentration/production are abnormal, high frequency video microscopy should be repeated at least three times and show the same abnormal results each time)
Given the complexity of diagnosing PCD, not all patients will meet the above mentioned definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry.
Ethics and Patient Confidentiality:
Obtaining the necessary patient consents is a prerequisite for entering data in the PCD Registry. As legislation differs in each participating country, it is important to check the requirements with your local Data Protection Officers and Ethics Committees. If you have any questions, please contact PCDregistry.eu@ukmuenster.de or Simone.Helms@ukmuenster.de. You will get support for the Ethics Committee approval procedure, e.g. sample consent forms for Patient Information and Consent. Patients can only be entered into the PCD Registry once you have received their signed, informed consent to do so. Please store all the consent forms that your patients have signed safely at your local institution. It is the responsibility of the reporting centres/countries/registries to have the acquired permissions to export/report data to the PCD Registry.
Data Security:
Due to data protection regulations, the data stored in the server of the PCD Registry must be anonymous (i.e. the patient must not be identifiable). Therefore, the authorised operator has to keep a logbook with the identifying patient data and the code generated by the registry software. It is your responsibility to make sure that access to this list of names and matching patient codes is restricted to authorized personnel only. Furthermore, please note that if your list of matching codes and full names is lost, it will be impossible to retrieve the data identification by means of the PCD Registry software. Moreover, data are encrypted (i.e. not de-codable) when transmitted. The PCD Registry is protected according to EU Data Protection legislation, both physically and technically, and backup is secured.
Funding:
The establishment of the PCD Registry was part of the EU funded BESTCILIA project (FP7 2007-2013) under grant agreement no 305404 (BESTCILIA). Funding continues within several projects including REGISTRY WAREHOUSE (HORIZON2020, GA no 777295)
About us:
The PCD Registry has been designed and established by the BESTCILIA consortium headed by Heymut Omran, University Children's Hospital Muenster, Department of General Pediatrics, Muenster, Germany and is part of the European Reference Network on Rare Lung Diseases ERN-LUNG. For more information, please contact PCDregistry.eu@ukmuenster.de Simone.Helms@ukmuenster.de, Petra.Pennekamp@ukmuenster.de or Johanna.Raidt@ukmuenster.de
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 20 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PCD | – | –Individuals with Primary Ciliary Dyskinesia will be assessed in a pure observational design without intervention |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with a diagnosis of Primary Ciliary Dyskinesia.
Key inclusion criteria (5 listed)
- Clinical presentation consistent with PCD and consistent findings specific for PCD in at least two of the following methods:
- high frequency video microscopic finding transmission electron microscopy finding immunofluorescence finding low nasal NO concentration/production demonstration of biallelic disease-causing mutations by genotyping
- Given the complexity of diagnosing PCD, it is anticipated that not all patients will meet these definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry. These cases usually are considered to have a possible PCD diagnosis with exceptions made on an individual basis.
- Exclusion Criteria Failure or unwillingness to give written informed consent. Missing qualification to perform legal acts or insufficient cognitive ability to give informed consent. A second disease is no exclusion criteria for including data in the PCD-Registry, especially since it is one of the fundamental goals, to learn about co-morbidities.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
descriptive analysis
Time frame: 10 years
Descriptive statistical methods, i.e. frequency tables, location and scale statistics and graphical presentations will be applied to analyse: age at diagnosis, family history (consanguinity), associated malformations/diseases, laterality defects, clinical manifestations (otitis media, rhinosinusitis, pneumonia, bronchiectasis, neonatal respiratory distress syndrome), microbiological results, diagnostic findings (video microscopy, electron microscopy, nasal NO, immunofluorescence analysis), lung function, radiological findings and therapeutic measures (inhalation therapy, antibiotics, oxygen, ventilation, upper airways surgery, lung surgery). Furthermore, quality of life will be assessed using QOL-PCD, a disease specific questionnaire.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2022.
- Derived from this study: PubMed 26659107Werner C, Lablans M, Ataian M, Raidt J, Wallmeier J, Grosse-Onnebrink J, Kuehni CE, Haarman EG, Leigh MW, Quittner AL, Lucas JS, Hogg C, Witt M, Priftis KN, Yiallouros P, Nielsen KG, Santamaria F, Uckert F, Omran H. An international registry for primary ciliary dyskinesia. Eur Respir J. 2016 Mar;47(3):849-59. doi: 10.1183/13993003.00776-2015. Epub 2015 Dec 8.
- ERN-LUNG is a patient-centric network of European healthcare providers and patient organisations, committed Europe-wide and globally to reducing morbidity and mortality from rare lung diseases in people of all agesLink provided by the sponsor.
- European Platform on Rare Disease RegistrationLink provided by the sponsor.
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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.
2 sites 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 (PCD), run by University Hospital Muenster at 2 sites in Germany.
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 5 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 28 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Petra Pennekamp, PhD (+4925183 ext. 41097, petra.pennekamp@ukmuenster.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
descriptive analysis (time frame: 10 years). 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 NCT02419365, 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 28 Nov 2022.