LymphangioleiomyomatosisNCT04577937
Sleep Patterns in Patients Affected by Lymphangioleiomiomatosis
A study testing LAM patients underwent PSG in people with Lymphangioleiomyomatosis and Sleep Disorder, run by dr. Stefano Nava at 1 site in Italy.
Trial summary
Lymphangioleiomyomatosis (LAM) is a rare and progressive pulmonary disease of unknown etiology that almost exclusively affects women. It is characterised by cystic radiological lung pattern and by the possible presence of angiomyolipomas in other sites or organs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lymphangioleiomyomatosis, Sleep Disorder, Sleep Apnea Syndromes
- What is being tested?
- LAM patients underwent PSG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteItaly
- Study dates
- 30 Jun 2020to 1 Nov 2021
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- confirmed diagnosis of LAM according to current guidelines
- sign of informed consent to participate
Who cannot join
- the presence of either acute or chronic respiratory failure
- the use of long-term oxygen therapy
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Azienda Ospedaliero-Universitaria di Bologna
Bologna, 40138
Stefano Nava, MD, 00390512143253, stefano.nava@aosp.bo.it
00390512143253
About this study
Lymphangioleiomyomatosis (LAM) is a rare and progressive pulmonary disease of unknown etiology that almost exclusively affects women. It is characterised by cystic radiological lung pattern and by the possible presence of angiomyolipomas in other sites or organs. Functionally LAM is associated with airway obstruction or restriction and progressive hypoxemia up to chronic respiratory failure.
There are no studies, so far, which have investigated whether during sleep these patients show changes in the sleep profile and gas exchange and if these changes are related to disease severity. Aim of the study, prospective and pilot, is to evaluate whether the physiological modification of respiratory mechanics during sleep is associated with polysomnographic alterations in LAM.
Read the detailed description
Lymphangioleiomiomatosis (LAM) is a rare progressive disease that affects primarily lungs with a cystic radiologic pattern and may be associated with angiomiolipomas in the kidneys or in other sites and an increased frequency of meningioma. Patients with sporadic LAM are usually female (incidence around 1/400.000 adult females). LAM associated with tuberous sclerosis may affect male, female and children.
LAM patients are functionally characterized by obstructive or restrictive syndrome that leads patients to hypoxemia and chronic respiratory failure, so that quality of Life (QoL) of these patients is affected by dyspnea. Hypoxemia contributes to the development of secondary pulmonary hypertension (PH) during progression of LAM history, worsening the damage.
The modifications in the neural control of ventilation during sleep in combination with anatomical disposition drive to sleep-related changes in upper airway resistance and physiologic desaturations. In healthy subjects these features are almost completely compensated but, in LAM patients, may lead to airway obstruction and/or pathologic oxygen desaturation that could be managed. So far, none have investigated the changes in sleep architecture and related blood gas exchange in patients with LAM relating these changes with disease severity.
This pilot study investigate if the physiologic modifications of breathing during sleep lead to sleep disorders in LAM patients, measured through abnormalities in polysomnography (PSG) and ih sleep disorders in LAM are associated with respiratory functional abnormalities and disease severity.
- Being tested
- LAM patients underwent PSG
- Medical subject headings
- Lymphangioleiomyomatosis, Sleep Wake Disorders, Sleep Apnea Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LAM patients underwent PSGDiagnostic test
Patients with confirmed diagnosis of LAM underwent a whole-night polysomnography. The presence of either acute or chronic respiratory failure or the use of long-term oxygen therapy are considered exclusion criteria
| Study group | Role | What they receive |
|---|---|---|
| PSG in LAM patients | Experimental | Patients affected by LAM underwent whole-night PSG Receives: Diagnostic Test: LAM patients underwent PSG |
What is being measured
The primary outcome is the main question the study is designed to answer.
prevalence of sleep disorders in LAM
Aim of the project is to assess the presence of sleep disorders (oxygen destaruration during sleep and/or sleep apnea or hypopnea) in patients affected by LAM.
Measured: 1 year
correlation between sleep-related disorders and LAM severity measured by spirometry
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured through functional impairment. By spirometry performed at the time of PSG the study population will be divided in: subjects with obstructive impairment; subjects with restrictive abnormality and subjects with no alterations.
Measured: 1 year
correlation between sleep-related disorders and LAM severity measured by annual decline of forced-expiratory volume at the first second (FEV1)
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured by annual decline of forced-expiratory volume at the first second (FEV1), expressed in ml/year. Annual decline of FEV1 will be measured subtracting the FEV1 value (ml) at the time of PSG test to the FEV1 value (ml) at the time of diagnosis and then, dividing it by disease duration in years.
Measured: 1 year
correlation between sleep-related disorders and LAM severity measured by arterial blood gas exchange
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured through arterial blood gas exchanges values: partial pressure of oxygen (PaO2 - mmHg), partial pressure of carbon dioxide (PaCO2 - mmHg), oxygen saturation (SatO2, %).
Measured: 1 year
correlation between sleep-related disorders and clincal parameters
Moreover, another aim is to assess the distribution of sleep disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to age, sex, body mass index (BMI), comorbidities in patients affected by LAM.
BMI will be calculated using height (mt) and weight (Kg) at the time of PSG and expressed in Kg/m^2.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2020
- Study start30 Jun 2020
- Primary completion8 Jul 2021 (estimated)
- Study completion1 Nov 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LymphangioleiomyomatosisStudies 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
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- cohort study, pilot
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- dr. Stefano Nava
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, dr. Stefano Nava, IRCCS Azienda Ospedaliero-Universitaria di Bologna
- Investigators
- Stefano Nava, MD-FERS, Principal investigator, AOSP - Azienda Ospedaliero Universitaria Di Bologna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04577937
- Sponsor's protocol ID
- 270/2016/O/Oss/AOUB
- Status last verified
- Mar 2021
- First submitted
- 30 Jun 2020
- Last updated
- 18 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sleep Patterns in Patients Affected by Lymphangioleiomiomatosis
- ClinicalTrials.gov ID
- NCT04577937
- Acronym / sponsor ID
- 270/2016/O/Oss/AOUB
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- dr. Stefano NavaUniversity, hospital or other
- Study start
- 30 Jun 2020 (actual)
- Primary completion
- 8 Jul 2021 (estimated)
- Study completion
- 1 Nov 2021 (estimated)
- First posted
- 8 Oct 2020 (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.
Lymphangioleiomyomatosis (LAM) is a rare and progressive pulmonary disease of unknown etiology that almost exclusively affects women. It is characterised by cystic radiological lung pattern and by the possible presence of angiomyolipomas in other sites or organs. Functionally LAM is associated with airway obstruction or restriction and progressive hypoxemia up to chronic respiratory failure.
There are no studies, so far, which have investigated whether during sleep these patients show changes in the sleep profile and gas exchange and if these changes are related to disease severity. Aim of the study, prospective and pilot, is to evaluate whether the physiological modification of respiratory mechanics during sleep is associated with polysomnographic alterations in LAM.
Detailed description
Lymphangioleiomiomatosis (LAM) is a rare progressive disease that affects primarily lungs with a cystic radiologic pattern and may be associated with angiomiolipomas in the kidneys or in other sites and an increased frequency of meningioma. Patients with sporadic LAM are usually female (incidence around 1/400.000 adult females). LAM associated with tuberous sclerosis may affect male, female and children.
LAM patients are functionally characterized by obstructive or restrictive syndrome that leads patients to hypoxemia and chronic respiratory failure, so that quality of Life (QoL) of these patients is affected by dyspnea. Hypoxemia contributes to the development of secondary pulmonary hypertension (PH) during progression of LAM history, worsening the damage.
The modifications in the neural control of ventilation during sleep in combination with anatomical disposition drive to sleep-related changes in upper airway resistance and physiologic desaturations. In healthy subjects these features are almost completely compensated but, in LAM patients, may lead to airway obstruction and/or pathologic oxygen desaturation that could be managed. So far, none have investigated the changes in sleep architecture and related blood gas exchange in patients with LAM relating these changes with disease severity.
This pilot study investigate if the physiologic modifications of breathing during sleep lead to sleep disorders in LAM patients, measured through abnormalities in polysomnography (PSG) and ih sleep disorders in LAM are associated with respiratory functional abnormalities and disease severity.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- cohort study, pilot
| Arm | Type | Receives |
|---|---|---|
| PSG in LAM patients | Experimental | Diagnostic Test: LAM patients underwent PSGPatients affected by LAM underwent whole-night PSG |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- confirmed diagnosis of LAM according to current guidelines
- sign of informed consent to participate
Key exclusion criteria (2 listed)
- the presence of either acute or chronic respiratory failure
- the use of long-term oxygen therapy
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 |
|---|---|---|
| LAM patients underwent PSGPatients with confirmed diagnosis of LAM underwent a whole-night polysomnography. The presence of either acute or chronic respiratory failure or the use of long-term oxygen therapy are considered exclusion criteria | Diagnostic test | PSG in LAM patients |
Primary outcome
prevalence of sleep disorders in LAM
Time frame: 1 year
Aim of the project is to assess the presence of sleep disorders (oxygen destaruration during sleep and/or sleep apnea or hypopnea) in patients affected by LAM.
correlation between sleep-related disorders and LAM severity measured by spirometry
Time frame: 1 year
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured through functional impairment. By spirometry performed at the time of PSG the study population will be divided in: subjects with obstructive impairment; subjects with restrictive abnormality and subjects with no alterations.
correlation between sleep-related disorders and LAM severity measured by annual decline of forced-expiratory volume at the first second (FEV1)
Time frame: 1 year
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured by annual decline of forced-expiratory volume at the first second (FEV1), expressed in ml/year. Annual decline of FEV1 will be measured subtracting the FEV1 value (ml) at the time of PSG test to the FEV1 value (ml) at the time of diagnosis and then, dividing it by disease duration in years.
correlation between sleep-related disorders and LAM severity measured by arterial blood gas exchange
Time frame: 1 year
Another aim of the project is to stratify the presence of sleep-related disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to the severity of LAM measured through arterial blood gas exchanges values: partial pressure of oxygen (PaO2 - mmHg), partial pressure of carbon dioxide (PaCO2 - mmHg), oxygen saturation (SatO2, %).
correlation between sleep-related disorders and clincal parameters
Time frame: 1 year
Moreover, another aim is to assess the distribution of sleep disorders (oxygen desaturation during sleep, sleep apnea or hypopnea) according to age, sex, body mass index (BMI), comorbidities in patients affected by LAM. BMI will be calculated using height (mt) and weight (Kg) at the time of PSG and expressed in Kg/m^2.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2021.
- 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.
Stefano Nava, MD-FERS principal investigator
AOSP - Azienda Ospedaliero Universitaria Di Bologna
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing LAM patients underwent PSG in people with Lymphangioleiomyomatosis and Sleep Disorder, run by dr. Stefano Nava at 1 site in Italy.
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 2 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 18 Mar 2021. 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 Italy. 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 Stefano Nava, MD-FERS (00390512143253, stefano.nava@aosp.bo.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
prevalence of sleep disorders in LAM (time frame: 1 year). The study lists 5 primary outcomes in all. 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 18 Mar 2021.