Pulmonary Arterial HypertensionNAIADNCT01907295
National Cohort Study of Idiopathic and Heritable Pulmonary Arterial Hypertension
An observational study of Pulmonary Arterial Hypertension, run by University of Cambridge at 8 sites in United Kingdom.
Trial summary
Pulmonary arterial hypertension (PAH), or high blood pressure in the lungs, is a rare condition that can shorten life. Although the cause of this disease is usually unknown, in about 70% of heritable and 15-20% of idiopathic cases there is a change in a gene (a mutation) that controls how blood vessels grow and… Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Pulmonary Arterial Hypertension
- How many people
- 3,600 participants
- Where
- 8 sitesUnited Kingdom
- Study dates
- Feb 2014to Dec 2022
- How long will I take part?
- 3 Years
- Phase
- Observational
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients diagnosed with idiopathic, anorexigen-induced, heritable PAH or PVOD. Relative who has a family member diagnosed with idiopathic, anorexigen-induced, heritable PAH or PVOD/PCH
Who may be able to join
- Participant is willing and able to give informed consent for participation in the study.
- Male or Female, any age
- Diagnosed with idiopathic, anorexigen-induced,heritable PAH, PVOD/PCH. Inclusion Criteria-Relative
- Participant is willing and able to give informed consent for participation in the study.
- Male or Female, any age
- Has a family member diagnosed with idiopathic, anorexigen-induced, PVOD/PCH or heritable PAH
Show all 9Show fewer
- Participant is willing and able to give informed consent for participation in the study.
- Self-reported to be healthy
- Age range up to 75 years
Who cannot join
- The participant may not enter the study if ANY of the following apply:
- Patient is unable to give informed consent.
- Not suffering from idiopathic, anorexigen-induced, PVOD/PCH or heritable PAH
- The participant may not enter the study if ANY of the following apply:
- Patient is unable to give informed consent.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United Kingdom8 sites, 8 recruiting
- Recruiting
Royal United Hospitals Bath
Bath, BA1 3NG
Robert MacKenenzie Ross, PI
- Recruiting
Royal Papworth Hospital NHS Trust
Cambridge
Joanna Pepke-Zaba
- Recruiting
Golden Jubilee National Hospital
Glasgow
Colin Church
- Recruiting
Imperial Hospital
London
Martin Wilkins
- Recruiting
Royal Brompton Hospital
London
S. John Wort
- Recruiting
Royal Free Hospital
London
Gerry Goglan
- Recruiting
Freeman Hospital
Newcastle
James Lordan
- Recruiting
Sheffield Hospital
Sheffield
David Kiely
About this study
Pulmonary arterial hypertension (PAH), or high blood pressure in the lungs, is a rare condition that can shorten life. Although the cause of this disease is usually unknown, in about 70% of heritable and 15-20% of idiopathic cases there is a change in a gene (a mutation) that controls how blood vessels grow and function. The gene is called bone morphogenetic protein type receptor 2 (BMPR2). Although mutations in BMPR2 are a risk factor for PAH, not everyone with a mutation gets the disease. Additional genetic and environmental factors are likely to contribute. The investigators suspect that mutations in other genes are responsible for some cases of PAH. In this study the investigators aim to recruit all patients with PAH and some of their relatives and follow them up for several years. The investigators hope to discover new mutations for this disease and to determine what factors lead to poor outcome, and to understand what triggers disease in patients with mutations.
Who can participate? Adults with PAH, their relatives and controls (one off blood sample)
Read the detailed description
What does the study involve?
PAH patients will be seen at their local centre by their service team but they will have additional bloods taken. Relatives of PAH patients will be seen every year at their nearest PAH centre. Tests will include:
- Epidemiology Questionnaire to assess factors affecting health
- An echocardiogram (ECHO) to assess the size, shape, pumping action and the extent of any damage to the heart.
- Lung function tests which include blowing measurements to assess gas volumes within the lungs as well as assessment of how the lungs exchange gases.
- Optional right heart catheterisation (RHC) to determine how much blood your heart is pumping while you are resting and on exercise. Optional Cardiac Magnetic Resonance tests. To measure heart function. ( to be done only once)
- 6 minute walk test. To measure exercise capacity
- Cardiopulmonary exercise test. A bicycle exercise test, which will indicate how much blood your heart pumps while resting and with different levels of exercise.
- Electrocardiogram (ECG), a test that measures the electrical activity of the heart
- Blood tests
Controls:Blood sample and medical data collected once
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- idiopathic, heritable, anorexigen, PCH/PVOD
- Medical subject headings
- Pulmonary Arterial Hypertension
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 |
|---|---|---|
| Patients | Patients diagnosed with idiopathic, anorexigen-induced, heritable PAH and PVOD/PCH | |
| Relatives and controls | Relative has a family member diagnosed with idiopathic, anorexigen-induced, heritable PAH and PVOD/PCH Self declared healthy individuals |
What is being measured
The primary outcome is the main question the study is designed to answer.
To recruit a national cohort (1000 subjects) of heritable, idiopathic PAH and PVOD/PCH cases.
The purpose of this study is to set up a national cohort of heritable, idiopathic PAH cases, PVOD/PCH and their relatives, to study the genetic and environmental contributions to disease. Setting up of this cohort of patients and relatives will provide the best resource for understanding what causes or triggers the disease, how to predict risk of death and response to therapy in individual patients, and to provide new ways of preventing and treating pulmonary arterial hypertension. The study will enable a better understanding for the first time the natural history of PAH, whether inherited or not. National outcomes to be measured will include survival, progression of the disease, changes in 6 minute walk distance, admissions to hospital for PAH and cause of death. Incidence of new cases of PAH will be measured in relatives as well.
Measured: 8 years
Secondary outcomes (1)
To recruit PAH patients (1000) and family members to a Biorepository for serum/plasma and urine to identify biomarkers of disease onset, progression and response to treatment.
To establish a Biorepository for serum/plasma, urine, tissues and cells from heritable pulmonary arterial hypertension (HPAH) patients, PVOD/PCH and their relatives, and patients with idiopathic PAH. This will allow studies to identify novel biomarkers of disease onset, progression and response to individual or combination therapies.
Measured: 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2013 (estimated)
- Study startFeb 2014
- Primary completionDec 2022 (estimated)
- Study completionDec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies 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
- 3,600 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- University of Cambridge
- Sponsor type
- University, hospital or other
- Collaborators
- British Heart Foundation, Medical Research Council
- Responsible party
- Principal investigator, Nick Morrell, University of Cambridge
- Investigators
- Nicholas Morrell, Principal investigator, University of Cambridge
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01907295
- Sponsor's protocol ID
- A092860
- Acronym
- NAIAD
- Status last verified
- May 2022
- First submitted
- 8 Jul 2013
- Last updated
- 20 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No: Consent for sharing of non identifiable study data for regulatory authorities, third parties including commercial companies outside the UK and NHS trusts where it is relevant to taking part in this research.
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- National Cohort Study of Idiopathic and Heritable Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT01907295
- Acronym / sponsor ID
- NAIAD / A092860
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- idiopathic, heritable, anorexigen, PCH/PVOD
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 3,600 participants (estimated)
- Lead sponsor
- University of CambridgeUniversity, hospital or other
- Collaborators
- British Heart Foundation, Medical Research Council
- Study start
- Feb 2014 (actual)
- Primary completion
- Dec 2022 (estimated)
- Study completion
- Dec 2022 (estimated)
- First posted
- 24 Jul 2013 (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.
Pulmonary arterial hypertension (PAH), or high blood pressure in the lungs, is a rare condition that can shorten life. Although the cause of this disease is usually unknown, in about 70% of heritable and 15-20% of idiopathic cases there is a change in a gene (a mutation) that controls how blood vessels grow and function. The gene is called bone morphogenetic protein type receptor 2 (BMPR2). Although mutations in BMPR2 are a risk factor for PAH, not everyone with a mutation gets the disease. Additional genetic and environmental factors are likely to contribute. The investigators suspect that mutations in other genes are responsible for some cases of PAH. In this study the investigators aim to recruit all patients with PAH and some of their relatives and follow them up for several years. The investigators hope to discover new mutations for this disease and to determine what factors lead to poor outcome, and to understand what triggers disease in patients with mutations.
Who can participate? Adults with PAH, their relatives and controls (one off blood sample)
Detailed description
What does the study involve?
PAH patients will be seen at their local centre by their service team but they will have additional bloods taken. Relatives of PAH patients will be seen every year at their nearest PAH centre. Tests will include:
- Epidemiology Questionnaire to assess factors affecting health
- An echocardiogram (ECHO) to assess the size, shape, pumping action and the extent of any damage to the heart.
- Lung function tests which include blowing measurements to assess gas volumes within the lungs as well as assessment of how the lungs exchange gases.
- Optional right heart catheterisation (RHC) to determine how much blood your heart is pumping while you are resting and on exercise. Optional Cardiac Magnetic Resonance tests. To measure heart function. ( to be done only once)
- 6 minute walk test. To measure exercise capacity
- Cardiopulmonary exercise test. A bicycle exercise test, which will indicate how much blood your heart pumps while resting and with different levels of exercise.
- Electrocardiogram (ECG), a test that measures the electrical activity of the heart
- Blood tests
Controls:Blood sample and medical data collected once
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients | – | –Patients diagnosed with idiopathic, anorexigen-induced, heritable PAH and PVOD/PCH |
| Relatives and controls | – | –Relative has a family member diagnosed with idiopathic, anorexigen-induced, heritable PAH and PVOD/PCH Self declared healthy individuals |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with idiopathic, anorexigen-induced, heritable PAH or PVOD. Relative who has a family member diagnosed with idiopathic, anorexigen-induced, heritable PAH or PVOD/PCH
Key inclusion criteria (9 listed)
- Participant is willing and able to give informed consent for participation in the study.
- Male or Female, any age
- Diagnosed with idiopathic, anorexigen-induced,heritable PAH, PVOD/PCH. Inclusion Criteria-Relative
- Participant is willing and able to give informed consent for participation in the study.
- Male or Female, any age
Key exclusion criteria (5 listed)
- Patient is unable to give informed consent.
- Not suffering from idiopathic, anorexigen-induced, PVOD/PCH or heritable PAH
- Patient is unable to give informed consent.
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
To recruit a national cohort (1000 subjects) of heritable, idiopathic PAH and PVOD/PCH cases.
Time frame: 8 years
The purpose of this study is to set up a national cohort of heritable, idiopathic PAH cases, PVOD/PCH and their relatives, to study the genetic and environmental contributions to disease. Setting up of this cohort of patients and relatives will provide the best resource for understanding what causes or triggers the disease, how to predict risk of death and response to therapy in individual patients, and to provide new ways of preventing and treating pulmonary arterial hypertension. The study will enable a better understanding for the first time the natural history of PAH, whether inherited or not. National outcomes to be measured will include survival, progression of the disease, changes in 6 minute walk distance, admissions to hospital for PAH and cause of death. Incidence of new cases of PAH will be measured in relatives as well.
1 secondary outcome
To recruit PAH patients (1000) and family members to a Biorepository for serum/plasma and urine to identify biomarkers of disease onset, progression and response to treatment.
Time frame: 8 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2022.
- Study websiteLink provided by the sponsor.
- Pulmonary Hypertension patient websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No. Consent for sharing of non identifiable study data for regulatory authorities, third parties including commercial companies outside the UK and NHS trusts where it is relevant to taking part in this research.
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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.
Nicholas Morrell principal investigator
University of Cambridge
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pulmonary Arterial Hypertension, run by University of Cambridge at 8 sites 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 9 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: status not confirmed, as of the sponsor's last update on 20 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Nicholas Morrell (01223 331666, nwm23@cam.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To recruit a national cohort (1000 subjects) of heritable, idiopathic PAH and PVOD/PCH cases. (time frame: 8 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 May 2022.