Sickle Cell DiseasePhase 3VOLCADREPNCT00560261
Evaluation of the Lung Capillary Blood Volume in Children With Sickle Cell Disease
A Phase 3 study testing NO-CO inhalation and expiration in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Sickle cell disease (SCD) is the most common inherited disease of the world affecting African and Caribbean populations. SCD is caused by the homozygous inheritance of the gene for sickle hemoglobin (HbS). Read more about this study
- Who is it for?
- Ages 6 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- NO-CO inhalation and expiration
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteFrance
- Study dates
- Feb 2008to Feb 2012
- Phase
- Phase 3
- 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 6 to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Children between 6 and 18 years
- Sickle cell disease( SS,SC, SBETA O, SDpunjab) and control without sickle cell disease
- Social insurance
- Signed informed consent
Who cannot join
- Respiratory disease other tha asthma
- Cardiac disease
- Encephalopathy
- G6PD deficiency
- Consent not signed
Trial locations and contacts
1 site in 1 country.
About this study
Sickle cell disease (SCD) is the most common inherited disease of the world affecting African and Caribbean populations. SCD is caused by the homozygous inheritance of the gene for sickle hemoglobin (HbS). Most patients with SCD develop abnormal pulmonary function characterized by airway obstruction, restrictive lung disease, abnormal diffusing capacity, hypoxemia and pulmonary hypertension In healthy subjects, lung capillary blood volume (Qc) and membrane diffusing capacity (Dm) can be accurately measured by the nitric oxide-carbon monoxide (NO-CO) method. We propose to study, for the first time, lung capillary blood volume and alveolar membrane diffusing capacity, using the NO-CO method, in children with SCD aged of at least 6 years Early determination of lung function and pulmonary circulation in children with SCD is very important, not only for the understanding of physiopathologic mechanisms of the disease but also for a better therapeutic management of these children.
Read the detailed description
We propose to study, for the first time, lung capillary blood volume and alveolar membrane diffusing capacity, using the NO-CO method, in children with SCD aged of at least 6 years. We will compare lung function and measurement of Qc and Dm in 2 groups of 120 subjects, one group of SCD children, and the other of normal children matched on age and ethnic origin. Measurement of lung capillary blood will be measured twice, to assess short term reproducibility. The measurement will be done in sitting position and lying down for one part of subjects, and at rest and during a moderate rectangular exercise for the other part of subjects. These different tests are designed to assess the physiological adaptation of pulmonary circulation in these two populations of children. Combined with complete lung function measurements, echocardiographic assessment of pulmonary hemodynamics, and measurement of exhaled nitric oxide, these evaluations will lead to a better understanding of pathophysiology of lung injury in SCD. The study will be completes at Robert Debré Hospital, in close collaboration with Sickle Cell Disease Center and Physiology Department. Children will be included after informed consent signed, as legally prescribed.
- Conditions
- Sickle Cell Disease
- Being tested
- NO-CO inhalation and expiration
- Keywords
- Sickle cell disease;, Lung capillary blood volume;, Children
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NO-CO inhalation and expirationOther
NO-CO inhalation and expiration
NO-CO inhalation and expirationOther
NO-CO inhalation and expiration
| Study group | Role | What they receive |
|---|---|---|
| 1:Children with sickle cell disease | Experimental | NO-CO inhalation and expiration:
Children with sickle cell disease Receives: Other: NO-CO inhalation and expiration |
| 2: Healthy volunteers | Active comparator | NO-CO inhalation and expiration:
Healthy volunteers Receives: Other: NO-CO inhalation and expiration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Study of lung capillary blood volume and alveolar membrane diffusing capacity using NO-CO method
Measured: The day of the measure
Secondary outcomes (4)
Respiratory physiopathology's study in sickle cell disease
Measured: At the induction of the study
Valid alveolar membrane diffusing capacity using NO-CO in children with or without sickle cell disease
Measured: At the induction of the study
Purpose respiratory function follow up in sickle cell disease child
Measured: At the induction of the study
Find relationship between these vascular abnormalities and NO metabolism
Measured: At the induction of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2007 (estimated)
- Study startFeb 2008
- Primary completionFeb 2012
- Study completionFeb 2012
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 Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Florence MISSUD, Md, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00560261
- Sponsor's protocol ID
- P061013
- Other IDs
- 2007-A00913-50
- Acronym
- VOLCADREP
- Status last verified
- Jul 2012
- First submitted
- 16 Nov 2007
- Last updated
- 3 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Bokov P, Boizeau P, Pautrat J, Missud F, Ba A, Haouari Z, Denjean A, Delclaux C, Benkerrou M. Altered pulmonary capillary blood volume in childhood sickle cell disease. Eur Respir J. 2020 Dec 10;56(6):2000379. doi: 10.1183/13993003.00379-2020. Print 2020 Dec. No abstract available.
PubMed 32616593
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Lung Capillary Blood Volume in Children With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00560261
- Acronym / sponsor ID
- VOLCADREP / P061013
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease;, Lung capillary blood volume;, Children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Feb 2008
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 19 Nov 2007 (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.
Sickle cell disease (SCD) is the most common inherited disease of the world affecting African and Caribbean populations. SCD is caused by the homozygous inheritance of the gene for sickle hemoglobin (HbS). Most patients with SCD develop abnormal pulmonary function characterized by airway obstruction, restrictive lung disease, abnormal diffusing capacity, hypoxemia and pulmonary hypertension In healthy subjects, lung capillary blood volume (Qc) and membrane diffusing capacity (Dm) can be accurately measured by the nitric oxide-carbon monoxide (NO-CO) method. We propose to study, for the first time, lung capillary blood volume and alveolar membrane diffusing capacity, using the NO-CO method, in children with SCD aged of at least 6 years Early determination of lung function and pulmonary circulation in children with SCD is very important, not only for the understanding of physiopathologic mechanisms of the disease but also for a better therapeutic management of these children.
Detailed description
We propose to study, for the first time, lung capillary blood volume and alveolar membrane diffusing capacity, using the NO-CO method, in children with SCD aged of at least 6 years. We will compare lung function and measurement of Qc and Dm in 2 groups of 120 subjects, one group of SCD children, and the other of normal children matched on age and ethnic origin. Measurement of lung capillary blood will be measured twice, to assess short term reproducibility. The measurement will be done in sitting position and lying down for one part of subjects, and at rest and during a moderate rectangular exercise for the other part of subjects. These different tests are designed to assess the physiological adaptation of pulmonary circulation in these two populations of children. Combined with complete lung function measurements, echocardiographic assessment of pulmonary hemodynamics, and measurement of exhaled nitric oxide, these evaluations will lead to a better understanding of pathophysiology of lung injury in SCD. The study will be completes at Robert Debré Hospital, in close collaboration with Sickle Cell Disease Center and Physiology Department. Children will be included after informed consent signed, as legally prescribed.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1:Children with sickle cell disease | Experimental | Other: NO-CO inhalation and expirationNO-CO inhalation and expiration: Children with sickle cell disease |
| 2: Healthy volunteers | Active comparator | Other: NO-CO inhalation and expirationNO-CO inhalation and expiration: Healthy volunteers |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Children between 6 and 18 years
- Sickle cell disease( SS,SC, SBETA O, SDpunjab) and control without sickle cell disease
- Social insurance
- Signed informed consent
Key exclusion criteria (5 listed)
- Respiratory disease other tha asthma
- Cardiac disease
- Encephalopathy
- G6PD deficiency
- Consent not signed
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 |
|---|---|---|
| NO-CO inhalation and expirationNO-CO inhalation and expiration | Other | 1:Children with sickle cell disease |
| NO-CO inhalation and expirationNO-CO inhalation and expiration | Other | 2: Healthy volunteers |
Primary outcome
Study of lung capillary blood volume and alveolar membrane diffusing capacity using NO-CO method
Time frame: The day of the measure
4 secondary outcomes
Respiratory physiopathology's study in sickle cell disease
Time frame: At the induction of the study
Valid alveolar membrane diffusing capacity using NO-CO in children with or without sickle cell disease
Time frame: At the induction of the study
Purpose respiratory function follow up in sickle cell disease child
Time frame: At the induction of the study
Find relationship between these vascular abnormalities and NO metabolism
Time frame: At the induction of the study
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 3 Feb 2023.
- Results publication: PubMed 32616593Bokov P, Boizeau P, Pautrat J, Missud F, Ba A, Haouari Z, Denjean A, Delclaux C, Benkerrou M. Altered pulmonary capillary blood volume in childhood sickle cell disease. Eur Respir J. 2020 Dec 10;56(6):2000379. doi: 10.1183/13993003.00379-2020. Print 2020 Dec. No abstract available.
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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.
Florence MISSUD, Md principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NO-CO inhalation and expiration in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 4 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 3 Feb 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 France. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Study of lung capillary blood volume and alveolar membrane diffusing capacity using NO-CO method (time frame: The day of the measure). 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 NCT00560261, 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 3 Feb 2023.