Sickle Cell DiseasePhase 3TOCIACSNCT06818266
Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease
A Phase 3 study testing Tocilizumab (RoActemra®, 20 mg/mL). in people with Sickle Cell Disease and Acute Chest Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Sickle Cell Disease, Acute Chest Syndrome
- What is being tested?
- Tocilizumab (RoActemra®, 20 mg/mL).
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 130 participants
- Where
- 1 siteFrance
- Study dates
- 27 Aug 2025to Jul 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- SCD patient of all genotypes (SS, SC, S/β0 and S/β+ or other major SCD syndrome)
- Age ≥ 2 years old
- Hospitalized for ACS, defined by the WHO as the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging, (X-ray, lung ultrasound, or CT scan)
- Requiring supplemental oxygen ≥ 2 L/min for SpO2 ≥ 95% or non-invasive respiratory support (high flow nasal oxygen or continuous positive airway pressure or bilevel non-invasive ventilation) or invasive mechanical ventilation or ECMO, for less than 48 hours
- Negative pregnancy test for girls or women of childbearing age
- Freely given, informed and written consent of patient or legal representatives
- Affiliation to the social security (or health insurance)
- Effective contraception up to 3 months after the administration of treatment (tocilizumab or placebo)
Who cannot join
- Impossibility to perform tocilizumab/placebo injection within the first 48 hours of supplemental oxygen ≥2L/min for SpO2≥95% and/or respiratory support (as defined in inclusion criteria n°4). If exchange transfusion is indicated at inclusion, it has to be performed before the injection of tocilizumab/placebo.
- Known hypersensitivity to tocilizumab or its excipients
- Known active current severe bacterial, viral, fungal, mycobacterial, or other infections (including but not limited to tuberculosis and atypical mycobacterial disease, hepatitis B and C, and herpes zoster)
- Immunization with a live/attenuated vaccine within the last 4 weeks
- Immunomodulatory therapy, anti-rejection therapy, cell depleting therapies and investigational agents within the last 3 months
- History of severe allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies
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- History of diverticulitis, diverticulosis requiring antibiotic treatment, or chronic ulcerative lower gastrointestinal disease such as Crohn's disease, ulcerative colitis, or other symptomatic lower gastrointestinal conditions that might predispose a patient to perforations
- Evidence of malignant disease or malignancies diagnosed within the last 3 years
- Pregnancy or breastfeeding
- Imminent and inevitable progression towards death in the opinion of the investigator
- Absolute neutrophil count < 1.0 G/L or platelets < 50 G/L
- ALT or AST > 5-fold the upper limit of normal
- Glomerular Filtration rate (GFR) < 60 mL/min/1,73 m²
- Current enrolment in another interventional research concerning a medicinal product for human use
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Aminata TRAORE, Project advisor contact
France1 site, 1 recruiting
- Recruiting
Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital
Paris, 75015
Slimane ALLALI, MD, PhD, 01 44 49 48 96 ext. +33, slimane.allali@aphp.fr
Aminata TRAORE, Project advisor
About this study
Official title: Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Pediatric and Adult Patients With Sickle Cell Disease
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).
Read the detailed description
SCD is a severe hemoglobinopathy, considered the first monogenic disease in the world. ACS, one of the most frequent and serious complications of SCD, is the first cause of hospitalization and mortality of SCD patients in intensive care unit. However, its pathophysiology has long been poorly understood and therapeutic options are limited.
A major increase has been recently reported in the level of interleukin-6 (IL-6), unlike other main pro-inflammatory cytokines, in the sputum (or bronchoalveolar fluid) from SCD children during ACS, positively correlated with the severity of ACS. Also, the observations of a very rapidly favorable outcome after administration of tocilizumab (anti-human IL-6 receptor monoclonal antibody) in SCD patients hospitalized for ACS with or without SARS-CoV-2 infection, suggest that tocilizumab may be a key therapy for ACS.
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Being tested
- Tocilizumab (RoActemra®, 20 mg/mL)., Placebo (NaCl 0.9%)
- Keywords
- Sickle cell disease, Acute chest syndrome, Tocilizumab
- Medical subject headings
- Anemia, Sickle Cell, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tocilizumab (RoActemra®, 20 mg/mL).Drug
One single intravenous infusion at 8 mg/kg (up to a maximum of 800 mg) for patients ≥ 30 kg and 12 mg/kg for patients < 30 kg
Placebo (NaCl 0.9%)Drug
One single intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Arms A : Tocilizumab (RoActemra®, 20 mg/mL) | Experimental | One single intravenous infusion at 8 mg/kg (up to a maximum of 800 mg) for patients ≥ 30 kg and 12 mg/kg for patients < 30 kg. Receives: Drug: Tocilizumab (RoActemra®, 20 mg/mL). |
| Arm B : Placebo (NaCl 0.9%) | Placebo comparator | One single intravenous infusion Receives: Drug: Placebo (NaCl 0.9%) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to successful weaning from both supplemental oxygen and any respiratory support
Successful weaning from both supplemental oxygen and any respiratory support, defined as SpO2 ≥ 95% without oxygen during the next 24 hours, and spontaneous breathing without any respiratory support (non-invasive or invasive) during the next 48 hours
Measured: During hospitalization for ACS, from randomization until day 28 after randomization
Secondary outcomes (15)
Adverse events during hospitalization and within 3 months following tocilizumab or placebo injection
Severe and not severe adverse events (including hypertension, hypersensitivity reactions, hypokalemia, neutropenia, thrombocytopenia, infections, pulmonary embolism/thrombosis, hepatic cytolysis, organ failure)
Measured: Within 3 months after randomization
Time to discharge
Length of hospital stay
Measured: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Mortality
Mortality
Measured: Within 3 months after randomization
Need for transfusion
Need for red blood cell transfusion
Measured: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Total number of red blood cell units received
Total number of red blood cell units received
Measured: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Need for non-invasive ventilation (for patients without ventilatory support at inclusion)
Need for non-invasive ventilation (high flow nasal oxygen, continuous positive airway pressure, or bilevel non-invasive ventilation), for patients without ventilatory support at inclusion
Measured: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Need for invasive ventilation (for patients without invasive ventilation at inclusion)
Need for invasive ventilation, for patients without invasive ventilation at inclusion
Measured: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Readmission for vaso-occlusive crisis or ACS within 3 months following tocilizumab or placebo injection
Readmission for vaso-occlusive crisis or ACS within 3 months following tocilizumab or placebo injection
Measured: Within 3 months after randomization
C-reactive protein (CRP), procalcitonin (PCT), plasma and sputum IL-6 levels 48 (+/- 12) hours after tocilizumab or placebo injection
C-reactive protein (CRP), procalcitonin (PCT), plasma and sputum IL-6 levels 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Procalcitonin (PCT) level 48 (+/- 12) hours after tocilizumab or placebo injection
Procalcitonin (PCT) level 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Plasma IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Plasma IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Sputum IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Sputum IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Chest imaging improvement 48 (+/- 12) hours after tocilizumab or placebo injection
Improvement of chest imaging (chest X-ray or lung ultrasound) will be assessed by an investigator, who will have to choose between 3 possible answers: worsening, stability or improvement of ACS images.
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the plasma 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the plasma 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the sputum (or in the tracheal aspirations in case of invasive mechanical ventilation) 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the sputum (or in the tracheal aspirations in case of invasive mechanical ventilation) 48 (+/- 12) hours after tocilizumab or placebo injection
Measured: 48 (+/- 12) hours after tocilizumab or placebo injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2025
- Study start27 Aug 2025
- Primary completionApr 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 130 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Slimane ALLALI, MD, PhD, Principal investigator, Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital, Paris, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06818266
- Sponsor's protocol ID
- APHP220797
- Other IDs
- 2023-505109-17-00, 2023
- Acronym
- TOCIACS
- Status last verified
- Jun 2026
- First submitted
- 9 Jan 2025
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Pediatric and Adult Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT06818266
- Acronym / sponsor ID
- TOCIACS / APHP220797
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Keywords
- Sickle cell disease, Acute chest syndrome, Tocilizumab
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 130 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 27 Aug 2025 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 10 Feb 2025 (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.
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).
Detailed description
SCD is a severe hemoglobinopathy, considered the first monogenic disease in the world. ACS, one of the most frequent and serious complications of SCD, is the first cause of hospitalization and mortality of SCD patients in intensive care unit. However, its pathophysiology has long been poorly understood and therapeutic options are limited.
A major increase has been recently reported in the level of interleukin-6 (IL-6), unlike other main pro-inflammatory cytokines, in the sputum (or bronchoalveolar fluid) from SCD children during ACS, positively correlated with the severity of ACS. Also, the observations of a very rapidly favorable outcome after administration of tocilizumab (anti-human IL-6 receptor monoclonal antibody) in SCD patients hospitalized for ACS with or without SARS-CoV-2 infection, suggest that tocilizumab may be a key therapy for ACS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arms A : Tocilizumab (RoActemra®, 20 mg/mL) | Experimental | Drug: Tocilizumab (RoActemra®, 20 mg/mL).One single intravenous infusion at 8 mg/kg (up to a maximum of 800 mg) for patients ≥ 30 kg and 12 mg/kg for patients < 30 kg. |
| Arm B : Placebo (NaCl 0.9%) | Placebo comparator | Drug: Placebo (NaCl 0.9%)One single intravenous infusion |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- SCD patient of all genotypes (SS, SC, S/β0 and S/β+ or other major SCD syndrome)
- Age ≥ 2 years old
- Hospitalized for ACS, defined by the WHO as the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging, (X-ray, lung ultrasound, or CT scan)
- Requiring supplemental oxygen ≥ 2 L/min for SpO2 ≥ 95% or non-invasive respiratory support (high flow nasal oxygen or continuous positive airway pressure or bilevel non-invasive ventilation) or invasive mechanical ventilation or ECMO, for less than 48 hours
- Negative pregnancy test for girls or women of childbearing age
Key exclusion criteria (14 listed)
- Impossibility to perform tocilizumab/placebo injection within the first 48 hours of supplemental oxygen ≥2L/min for SpO2≥95% and/or respiratory support (as defined in inclusion criteria n°4). If exchange transfusion is indicated at inclusion, it has to be performed before the injection of tocilizumab/placebo.
- Known hypersensitivity to tocilizumab or its excipients
- Known active current severe bacterial, viral, fungal, mycobacterial, or other infections (including but not limited to tuberculosis and atypical mycobacterial disease, hepatitis B and C, and herpes zoster)
- Immunization with a live/attenuated vaccine within the last 4 weeks
- Immunomodulatory therapy, anti-rejection therapy, cell depleting therapies and investigational agents within the last 3 months
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 |
|---|---|---|
| Tocilizumab (RoActemra®, 20 mg/mL).One single intravenous infusion at 8 mg/kg (up to a maximum of 800 mg) for patients ≥ 30 kg and 12 mg/kg for patients < 30 kg | Drug | Arms A : Tocilizumab (RoActemra®, 20 mg/mL) |
| Placebo (NaCl 0.9%)One single intravenous infusion | Drug | Arm B : Placebo (NaCl 0.9%) |
Primary outcome
Time to successful weaning from both supplemental oxygen and any respiratory support
Time frame: During hospitalization for ACS, from randomization until day 28 after randomization
Successful weaning from both supplemental oxygen and any respiratory support, defined as SpO2 ≥ 95% without oxygen during the next 24 hours, and spontaneous breathing without any respiratory support (non-invasive or invasive) during the next 48 hours
15 secondary outcomes
Adverse events during hospitalization and within 3 months following tocilizumab or placebo injection
Time frame: Within 3 months after randomization
Time to discharge
Time frame: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Mortality
Time frame: Within 3 months after randomization
Need for transfusion
Time frame: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Total number of red blood cell units received
Time frame: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Need for non-invasive ventilation (for patients without ventilatory support at inclusion)
Time frame: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Need for invasive ventilation (for patients without invasive ventilation at inclusion)
Time frame: From the date of randomization until the date of end of hospitalization, assessed up to 3 months
Readmission for vaso-occlusive crisis or ACS within 3 months following tocilizumab or placebo injection
Time frame: Within 3 months after randomization
C-reactive protein (CRP), procalcitonin (PCT), plasma and sputum IL-6 levels 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Procalcitonin (PCT) level 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Plasma IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Sputum IL-6 level 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Chest imaging improvement 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the plasma 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Tocilizumab level in the sputum (or in the tracheal aspirations in case of invasive mechanical ventilation) 48 (+/- 12) hours after tocilizumab or placebo injection
Time frame: 48 (+/- 12) hours after tocilizumab or placebo injection
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- 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.
Aminata TRAORE, Project advisor contact
Slimane ALLALI, MD, PhD principal investigator
Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital, Paris, France
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 Tocilizumab (RoActemra®, 20 mg/mL). 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 8 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 24 Jun 2026. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Slimane ALLALI, MD, PhD (01 44 49 48 96 ext. +33, slimane.allali@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to successful weaning from both supplemental oxygen and any respiratory support (time frame: During hospitalization for ACS, from randomization until day 28 after randomization). 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 24 Jun 2026; AzTrials last refreshed its copy on 8 Nov 2026.