Acute Chest SyndromePhase 2INNOSTAPEDNCT01089439
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
A Phase 2 study testing Nitric oxide by inhalation INOMAX in people with Acute Chest Syndrome and Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Acute Chest Syndrome, Sickle Cell Disease
- What is being tested?
- Nitric oxide by inhalation INOMAX
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2010to Nov 2013
- Phase
- Phase 2
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- child between 1 and 18 years old
- Sickle cell anemia or equivalent (sickle beta 0 thalassemia)whoe weight is between 10kg and 65kg
- presenting acute chest syndrome as defined a new radiological infiltrate with tachypnea, respiratory discomfort, cough, chest wall pain and fever more than 38.5°C
- hypoxaemia with transcutaneous oxygen saturation equal or less than 92%
- informed consent signed by parents and approved by the child able to express his consent
- insured by the National social security system or by the universal medical insurance
- previous medical physical examination
Who cannot join
- respiratory distress with hypoxaemia with transcutaneous oxygen saturation equal or less than 92% under more than 5l/min of oxygen or 40% oxygen inhaled, hypercapnia signs 'sweating, altered consciousness, paCO2 more than 60mmHg) with need of emergency exchange transfusion and/or tracheal intubation with mechanical ventilation
- Isolated acute asthmatic crisis
- stroke or priapism with emergency acute transfusion needed
- acute anemia with hemoglobin drop of more than 20% as compared to steady state hemoglobin
- chronic long term transfusion therapy
- nitric oxyde hypersensitivity
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- patients with right-left extra-pulmonary cardiac shunt
- patient previously included in the protocol
- patient participating in another interventional protocol
- pregnancy or breast feeding
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis.
Read the detailed description
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis in a prospective randomized double-blind placebo controlled study. 50 children in two years will be included: 25 in each arm.
- Conditions
- Acute Chest Syndrome, Sickle Cell Disease
- Being tested
- Nitric oxide by inhalation INOMAX, Placebo
- Keywords
- Nitric oxide
- Medical subject headings
- Acute Chest Syndrome, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nitric oxide by inhalation INOMAXDrug
Also called INOMAX
Nitric oxide by inhalation INOMAX 800 ppm
40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| 1: INOMAX | Active comparator | Nitric oxide by inhalation INOMAX:
active arm treated with nitric oxide Receives: Drug: Nitric oxide by inhalation INOMAX |
| 2: Placebo | Placebo comparator | placebo arm treated with placebo at the same conditions Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )
Measured: Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge
Secondary outcomes (4)
Number of blood transfusions and total transfused blood volume
Measured: 7 to 10 days
Quantity of Pain-killer drugs required and particularly OPIOIDS
Measured: 7 to 10 days
Duration of Nitric oxide therapy
Measured: after 7 to 10 days
Duration of OXYGENOTHERAPY
Measured: 7 to 10 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2010 (estimated)
- Study startJun 2010
- Primary completionNov 2013
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Chest SyndromeStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- Malika Benkerrou, Dr., Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01089439
- Sponsor's protocol ID
- P071003
- Acronym
- INNOSTAPED
- Status last verified
- Aug 2013
- First submitted
- 21 Oct 2009
- Last updated
- 4 Dec 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
- ClinicalTrials.gov ID
- NCT01089439
- Acronym / sponsor ID
- INNOSTAPED / P071003
- Conditions
- Acute Chest Syndrome, Sickle Cell Disease
- Keywords
- Nitric oxide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Jun 2010
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 18 Mar 2010 (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 is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis.
Detailed description
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis in a prospective randomized double-blind placebo controlled study. 50 children in two years will be included: 25 in each arm.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1: INOMAX | Active comparator | Drug: Nitric oxide by inhalation INOMAXNitric oxide by inhalation INOMAX: active arm treated with nitric oxide |
| 2: Placebo | Placebo comparator | Drug: Placeboplacebo arm treated with placebo at the same conditions |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- child between 1 and 18 years old
- Sickle cell anemia or equivalent (sickle beta 0 thalassemia)whoe weight is between 10kg and 65kg
- presenting acute chest syndrome as defined a new radiological infiltrate with tachypnea, respiratory discomfort, cough, chest wall pain and fever more than 38.5°C
- hypoxaemia with transcutaneous oxygen saturation equal or less than 92%
- informed consent signed by parents and approved by the child able to express his consent
Key exclusion criteria (10 listed)
- respiratory distress with hypoxaemia with transcutaneous oxygen saturation equal or less than 92% under more than 5l/min of oxygen or 40% oxygen inhaled, hypercapnia signs 'sweating, altered consciousness, paCO2 more than 60mmHg) with need of emergency exchange transfusion and/or tracheal intubation with mechanical ventilation
- Isolated acute asthmatic crisis
- stroke or priapism with emergency acute transfusion needed
- acute anemia with hemoglobin drop of more than 20% as compared to steady state hemoglobin
- chronic long term transfusion 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 |
|---|---|---|
| Nitric oxide by inhalation INOMAXAlso: INOMAXNitric oxide by inhalation INOMAX 800 ppm 40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours | Drug | 1: INOMAX |
| Placeboplacebo | Drug | 2: Placebo |
Primary outcome
We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )
Time frame: Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge
4 secondary outcomes
Number of blood transfusions and total transfused blood volume
Time frame: 7 to 10 days
Quantity of Pain-killer drugs required and particularly OPIOIDS
Time frame: 7 to 10 days
Duration of Nitric oxide therapy
Time frame: after 7 to 10 days
Duration of OXYGENOTHERAPY
Time frame: 7 to 10 days
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 4 Dec 2014.
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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.
Malika Benkerrou, Dr. 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 study testing Nitric oxide by inhalation INOMAX in people with Acute Chest Syndrome and 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 7 inclusion and 10 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 4 Dec 2014. 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 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.
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?
We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% ) (time frame: Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge). 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 4 Dec 2014.