Sickle Cell DiseasePhase 2/3INOSTANCT00748423
Effect of Inhaled Nitric Oxide in Acute Chest Syndrome (INOSTA Study)
A Phase 2/3 study testing Nitric Oxide in people with Sickle Cell Disease and Acute Chest Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease, Acute Chest Syndrome
- What is being tested?
- Nitric Oxide
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2008to Dec 2012
- Phase
- Phase 2/3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with sickle cell disease (Hemoglobin genotypes characterized by standard procedures as homozygous hemoglobin SS, hemoglobin SC, and S beta thalassemia)
- Diagnosis of acute chest syndrome based on the presence of fever, dyspnea or chest pain, associated with new pulmonary infiltrates on chest X-ray
Who cannot join
- Patient has been hospitalised < 14 days ago
- Patients presenting with clinically diagnosed bacterial infections
- Patients who have received an exchange transfusion in the last 30 days or are in a transfusion program.
- Current pregnancy or lactation
- Patient who is currently enrolled in any other investigational drug study
- Previous participation in this study
Show all 9Show fewer
- Any of the following medical conditions:
- Immediate need of ventilatory support wih orotracheal intubation
- Hemodynamic instability
Trial locations and contacts
1 site in 1 country.
France1 site
Réanimation Médicale, Hôpital A Chenevier-H Mondor
Créteil, 94 000
About this study
Official title: Bicentric Study of the Effect of Inhaled Nitric Oxide Compared to Placebo in Acute Chest Syndrome of Adult Sickle Cell Patients
Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults. Several pathologic processes are recognized causes of ACS, including infectious diseases, hypoventilation secondary to chest pain, in situ thrombosis and pulmonary fat embolism. Inhaled nitric oxide (iNO) has been shown to be a pulmonary vasodilatator with minimal systemic effects and has also been shown to improve gas exchange in both animal and human acute lung injury (ALI).
The combined effects of iNO gas of improving pulmonary ventilation to perfusion matching, reducing alveolar and systemic inflammation, modulate the course of acute chest syndrome, which combine the physiopathology of vaso-occlusive crisis and acute lung injury.
We hypothesise inhaled NO will improve oxygenation and clinical outcome of sickle cell disease patients with acute chest syndrome.
Read the detailed description
Objectives: To compare the outcome and duration of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) treated with iNO to that of similar episodes experienced by patients which receive a placebo.
Study design: Bi-center, prospective, randomized, controlled clinical trial
- Enrollment: 24 months
- Patients will be treated for 72 hours
- Patients will be followed for 15 days or until discharged home
Sample size:
- The study will accrue a maximum of 240 patients
- Progress of the trial will be reviewed by an independent data and safety monitoring committee to determine if randomization should stop for safety reasons.
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Being tested
- Nitric Oxide, Placebo
- Keywords
- Inhaled drug, Acute lung injury, Nitric oxide, Sickle cell
- Medical subject headings
- Anemia, Sickle Cell, Acute Chest Syndrome, Acute Lung Injury
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nitric OxideDrug
NO in inhalation for 3 days
PlaceboDrug
Also called "Nitrogen" placebo
Placebo in inhalation for 3 days
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Nitric Oxide in nitrogen Receives: Drug: Nitric Oxide |
| 2 | Placebo comparator | Nitrogen Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with treatment failure
Measured: at day 3
Secondary outcomes (5)
Proportion of hypoxemic patients defined by a PaO2/FiO2 ratio < 300
Measured: at day 3
Variation of pulmonary arterial systolic pressure evaluated by echocardiography
Measured: at day 1, day 3 and end of study
Length of hospitalisation
Measured: from day 0 to day 15 (max)
Pain assessment and the cumulative dose of parenteral opioids per body weight
Measured: during the first three days and during entire hospitalization
Proportion of patients requiring transfusion therapy (simple or exchange)
Measured: from day 1 to end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2008 (estimated)
- Study startDec 2008
- Primary completionDec 2012
- Study completionDec 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- MAITRE Bernard, MD, PHD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00748423
- Sponsor's protocol ID
- P060701
- Acronym
- INOSTA
- Status last verified
- Jul 2013
- First submitted
- 5 Sep 2008
- Last updated
- 2 Aug 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Maitre B, Djibre M, Katsahian S, Habibi A, Stankovic Stojanovic K, Khellaf M, Bourgeon I, Lionnet F, Charles-Nelson A, Brochard L, Lemaire F, Galacteros F, Brun-Buisson C, Fartoukh M, Mekontso Dessap A. Inhaled nitric oxide for acute chest syndrome in adult sickle cell patients: a randomized controlled study. Intensive Care Med. 2015 Dec;41(12):2121-9. doi: 10.1007/s00134-015-4060-2. Epub 2015 Oct 2.
PubMed 26431718
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bicentric Study of the Effect of Inhaled Nitric Oxide Compared to Placebo in Acute Chest Syndrome of Adult Sickle Cell Patients
- ClinicalTrials.gov ID
- NCT00748423
- Acronym / sponsor ID
- INOSTA / P060701
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Keywords
- Inhaled drug, Acute lung injury, Nitric oxide, Sickle cell
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Dec 2008
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 8 Sep 2008 (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.
Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults. Several pathologic processes are recognized causes of ACS, including infectious diseases, hypoventilation secondary to chest pain, in situ thrombosis and pulmonary fat embolism. Inhaled nitric oxide (iNO) has been shown to be a pulmonary vasodilatator with minimal systemic effects and has also been shown to improve gas exchange in both animal and human acute lung injury (ALI).
The combined effects of iNO gas of improving pulmonary ventilation to perfusion matching, reducing alveolar and systemic inflammation, modulate the course of acute chest syndrome, which combine the physiopathology of vaso-occlusive crisis and acute lung injury.
We hypothesise inhaled NO will improve oxygenation and clinical outcome of sickle cell disease patients with acute chest syndrome.
Detailed description
Objectives: To compare the outcome and duration of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) treated with iNO to that of similar episodes experienced by patients which receive a placebo.
Study design: Bi-center, prospective, randomized, controlled clinical trial
- Enrollment: 24 months
- Patients will be treated for 72 hours
- Patients will be followed for 15 days or until discharged home
Sample size:
- The study will accrue a maximum of 240 patients
- Progress of the trial will be reviewed by an independent data and safety monitoring committee to determine if randomization should stop for safety reasons.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Nitric OxideNitric Oxide in nitrogen |
| 2 | Placebo comparator | Drug: PlaceboNitrogen |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients with sickle cell disease (Hemoglobin genotypes characterized by standard procedures as homozygous hemoglobin SS, hemoglobin SC, and S beta thalassemia)
- Diagnosis of acute chest syndrome based on the presence of fever, dyspnea or chest pain, associated with new pulmonary infiltrates on chest X-ray
Key exclusion criteria (9 listed)
- Patient has been hospitalised < 14 days ago
- Patients presenting with clinically diagnosed bacterial infections
- Patients who have received an exchange transfusion in the last 30 days or are in a transfusion program.
- Current pregnancy or lactation
- Patient who is currently enrolled in any other investigational drug study
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 |
|---|---|---|
| Nitric OxideNO in inhalation for 3 days | Drug | 1 |
| PlaceboAlso: "Nitrogen" placeboPlacebo in inhalation for 3 days | Drug | 2 |
Primary outcome
Percentage of patients with treatment failure
Time frame: at day 3
5 secondary outcomes
Proportion of hypoxemic patients defined by a PaO2/FiO2 ratio < 300
Time frame: at day 3
Variation of pulmonary arterial systolic pressure evaluated by echocardiography
Time frame: at day 1, day 3 and end of study
Length of hospitalisation
Time frame: from day 0 to day 15 (max)
Pain assessment and the cumulative dose of parenteral opioids per body weight
Time frame: during the first three days and during entire hospitalization
Proportion of patients requiring transfusion therapy (simple or exchange)
Time frame: from day 1 to end of 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 2 Aug 2013.
- Derived from this study: PubMed 26431718Maitre B, Djibre M, Katsahian S, Habibi A, Stankovic Stojanovic K, Khellaf M, Bourgeon I, Lionnet F, Charles-Nelson A, Brochard L, Lemaire F, Galacteros F, Brun-Buisson C, Fartoukh M, Mekontso Dessap A. Inhaled nitric oxide for acute chest syndrome in adult sickle cell patients: a randomized controlled study. Intensive Care Med. 2015 Dec;41(12):2121-9. doi: 10.1007/s00134-015-4060-2. Epub 2015 Oct 2.
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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.
MAITRE Bernard, MD, PHD 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 2/3 study testing Nitric Oxide in people with Sickle Cell Disease and Acute Chest Syndrome, 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 2 inclusion and 9 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 2 Aug 2013. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Percentage of patients with treatment failure (time frame: at day 3). 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 NCT00748423, 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 2 Aug 2013; AzTrials last refreshed its copy on 8 Nov 2026.