Hemophagocytic SyndromesPhase 2JAKAHDINCT06244862
JAK Inhibitor in Acquired Hemophagocytic synDrome in the Intensive Care Unit
A Phase 2 study testing Ruxolitinib in people with Hemophagocytic Syndromes, run by Assistance Publique - Hôpitaux de Paris.
Trial summary
Hemophagocytic syndrome (HS) is a rare condition that can be responsible for severe organ failure. Therapeutic guidelines are mainly based on observational studies and expert opinions: no therapeutic advance has been developed for years, explaining why mortality in HS remains high (Intensive Care Unit mortality… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Hemophagocytic Syndromes
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- No sites listed yet
- Study dates
- 1 Feb 2024to 1 Feb 2026
- 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 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- adult patients older than 18 years
- acquired hemophagocytic syndrome, regardless of etiology, defined by the presence of 5 or 6 HLH-2004 criteria or HScore ≥ 200
- admission in the ICU
- need for symptomatic treatment of HS in relation with organ failure, as defined by SOFA score ≥ 4
- Informed consent signed:
- by the patient,
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- Or informed consent signed by a family members/trustworthy person if his condition does not allow him to express his consent in written
- Or in an emergency situation and in the absence of family members/trustworthy person, the patient can be enrolled. The consent to participate to the research will be requested as soon as the condition of the patient will allow).
- The inclusion of women of childbearing potential requires the use of a highly effective contraceptive measure. Contraception should be maintained during treatment and one day after
Who cannot join
- Moribund, defined by a life expectancy < 48 hours;
- Pregnant or lactating patients (women of childbearing potential must have a negative urine or blood Human Chorionic Gonadotropin pregnancy test prior to trial entry);
- No affiliation to health insurance;
- Known hypersensitivity to ruxolitinib;
- Lactose intolerance;
- Hypersensitivity to cellulose, microcrystalline; magnesium stearate; silica, colloidal anhydrous; sodium starch glycolate (Type A); povidone K30; hydroxypropylcellulose 300 to 600 cps,
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- Pre-existing decisions of withholding/withdrawing care,
- History of progressive multifocal leukoencephalopathy
- Uncontrolled cutaneous cancer
- Persons under psychiatric care that would impede understanding of informed consent and optimal treatment and follow-up
- Adults subject to a legal protection measure (guardianship, curatorship and safeguard of justice)
- Patients deprived of their liberty by a judicial or administrative decision
- Participation in another interventional research
Trial locations and contacts
No sites are listed yet.
About this study
Hemophagocytic syndrome (HS) is a rare condition that can be responsible for severe organ failure. Therapeutic guidelines are mainly based on observational studies and expert opinions: no therapeutic advance has been developed for years, explaining why mortality in HS remains high (Intensive Care Unit mortality ranging from 40 to 70%). If etoposide remains the gold standard in critically ill HS patients, nearly 20% of patients are refractory to this therapy: treatment escalation is common, most often requiring the administration of intensive treatments generating high toxicity. Ruxolitinib is the first approved JAK inhibitor. It has been associated with improvement of HS manifestations and survival in a pre-clinical murine model. Data in humans are scarce but promising.
The aim is to demonstrate that ruxolitinib, in association with standard of care, may reverse organ failure (as represented by Sequential Organ Failure Assessment (SOFA) score) better than standard of care alone in critically ill patients with acquired HS.
- Conditions
- Hemophagocytic Syndromes
- Being tested
- Ruxolitinib
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Oral ruxolitinib twice a day (10 mg x 2 during 28 days) in association with standard of care in HS.
| Study group | Role | What they receive |
|---|---|---|
| Interventional medicinal product | Experimental | Not described Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival with a decrease in SOFA score ≥ 3 points
Sequential Organ Failure Assessment Score varies from 0 to 4 and permit to assess organ failure. A higher score indicates better neurological function.
Measured: At day 7
Secondary outcomes (30)
Overall survival in HS critically ill patients
Measured: At 6 months
SOFA score
Sequential Organ Failure Assessment Score varies from 0 to 4 and permit to assess organ failure. A higher score indicates better neurological function.
Measured: At day 1
SOFA score
Sequential Organ Failure Assessment Score varies from 0 to 4 and permit to assess organ failure. A higher score indicates better neurological function.
Measured: At day 14
SOFA score
Sequential Organ Failure Assessment Score varies from 0 to 4 and permit to assess organ failure. A higher score indicates better neurological function.
Measured: At day 28
Length of stay in Intensive Care Unit
number of days in the ICU from inclusion to ICU discharge or death
Measured: Up to 6 months
Hospital length of stay
number of days in the hospital from inclusion to hospital discharge or death
Measured: Up to 6 months
Measurement of clinical and biological manifestations
Measurements of temperature, ferritin level, CD25 soluble receptor dosage, fibrinogen level, triglycerides level, haemoglobin level, white blood cells count, platelets count
Measured: At day 1
Measurement of clinical and biological manifestations
Measurements of temperature, ferritin level, CD25 soluble receptor dosage, fibrinogen level, triglycerides level, haemoglobin level, white blood cells count, platelets count
Measured: At day 7
Measurement of temperature
Measured: At day 14
Measurement of temperature
Measured: At day 28
Measurement of ferritin level
Measured: At day 14
Measurement of ferritin level
Measured: At day 28
Measurement of CD25 soluble receptor dosage
Measured: At day 14
Measurement of CD25 soluble receptor dosage
Measured: At day 28
Measurement of fibrinogen level
Measured: At day 14
Measurement of fibrinogen level
Measured: At day 28
Measurement of triglycerides level
Measured: At day 14
Measurement of triglycerides level
Measured: At day 28
Measurement of haemoglobin level
Measured: At day 14
Measurement of haemoglobin level
Measured: At day 28
Measurement of white blood cells count
Measured: At day 14
Measurement of white blood cells count
Measured: At day 28
Platelets count
Measured: At day 14
Platelets count
Measured: At day 28
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Measured: At day 1
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Measured: At day 7
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Measured: At day 14
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Measured: At day 28
Incidence of nosocomial infections (viral and bacterial)
Measured: Until day 28
Incidence of adverse event, severe adverse event
Intensity and frequency of adverse event and severe adverse event according to the CTCAE Toxicity Grading Scale for Determining The Severity of Adverse Events
Measured: Until day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2024
- Study start1 Feb 2024 (estimated)
- Primary completion8 Aug 2025 (estimated)
- Study completion1 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophagocytic SyndromesStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06244862
- Sponsor's protocol ID
- APHP220919
- Acronym
- JAKAHDI
- Status last verified
- Dec 2023
- First submitted
- 26 Dec 2023
- Last updated
- 6 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- JAK Inhibitor in Acquired Hemophagocytic synDrome in the Intensive Care Unit
- ClinicalTrials.gov ID
- NCT06244862
- Acronym / sponsor ID
- JAKAHDI / APHP220919
- Conditions
- Hemophagocytic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 1 Feb 2024 (estimated)
- Primary completion
- 8 Aug 2025 (estimated)
- Study completion
- 1 Feb 2026 (estimated)
- First posted
- 6 Feb 2024 (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.
Hemophagocytic syndrome (HS) is a rare condition that can be responsible for severe organ failure. Therapeutic guidelines are mainly based on observational studies and expert opinions: no therapeutic advance has been developed for years, explaining why mortality in HS remains high (Intensive Care Unit mortality ranging from 40 to 70%). If etoposide remains the gold standard in critically ill HS patients, nearly 20% of patients are refractory to this therapy: treatment escalation is common, most often requiring the administration of intensive treatments generating high toxicity. Ruxolitinib is the first approved JAK inhibitor. It has been associated with improvement of HS manifestations and survival in a pre-clinical murine model. Data in humans are scarce but promising.
The aim is to demonstrate that ruxolitinib, in association with standard of care, may reverse organ failure (as represented by Sequential Organ Failure Assessment (SOFA) score) better than standard of care alone in critically ill patients with acquired HS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventional medicinal product | Experimental | Drug: Ruxolitinib |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- adult patients older than 18 years
- acquired hemophagocytic syndrome, regardless of etiology, defined by the presence of 5 or 6 HLH-2004 criteria or HScore ≥ 200
- admission in the ICU
- need for symptomatic treatment of HS in relation with organ failure, as defined by SOFA score ≥ 4
- Informed consent signed:
Key exclusion criteria (13 listed)
- Moribund, defined by a life expectancy < 48 hours;
- Pregnant or lactating patients (women of childbearing potential must have a negative urine or blood Human Chorionic Gonadotropin pregnancy test prior to trial entry);
- No affiliation to health insurance;
- Known hypersensitivity to ruxolitinib;
- Lactose intolerance;
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 |
|---|---|---|
| RuxolitinibOral ruxolitinib twice a day (10 mg x 2 during 28 days) in association with standard of care in HS. | Drug | Interventional medicinal product |
Primary outcome
Survival with a decrease in SOFA score ≥ 3 points
Time frame: At day 7
Sequential Organ Failure Assessment Score varies from 0 to 4 and permit to assess organ failure. A higher score indicates better neurological function.
30 secondary outcomes
Overall survival in HS critically ill patients
Time frame: At 6 months
SOFA score
Time frame: At day 1
SOFA score
Time frame: At day 14
SOFA score
Time frame: At day 28
Length of stay in Intensive Care Unit
Time frame: Up to 6 months
Hospital length of stay
Time frame: Up to 6 months
Measurement of clinical and biological manifestations
Time frame: At day 1
Measurement of clinical and biological manifestations
Time frame: At day 7
Measurement of temperature
Time frame: At day 14
Measurement of temperature
Time frame: At day 28
Measurement of ferritin level
Time frame: At day 14
Measurement of ferritin level
Time frame: At day 28
Measurement of CD25 soluble receptor dosage
Time frame: At day 14
Measurement of CD25 soluble receptor dosage
Time frame: At day 28
Measurement of fibrinogen level
Time frame: At day 14
Measurement of fibrinogen level
Time frame: At day 28
Measurement of triglycerides level
Time frame: At day 14
Measurement of triglycerides level
Time frame: At day 28
Measurement of haemoglobin level
Time frame: At day 14
Measurement of haemoglobin level
Time frame: At day 28
Measurement of white blood cells count
Time frame: At day 14
Measurement of white blood cells count
Time frame: At day 28
Platelets count
Time frame: At day 14
Platelets count
Time frame: At day 28
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Time frame: At day 1
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Time frame: At day 7
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Time frame: At day 14
Dosages of IL2, IL6, IL10, IL12, GM-CSF, IFN gamma, TNF alpha
Time frame: At day 28
Incidence of nosocomial infections (viral and bacterial)
Time frame: Until day 28
Incidence of adverse event, severe adverse event
Time frame: Until day 28
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2024.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib in people with Hemophagocytic Syndromes, run by Assistance Publique - Hôpitaux de Paris.
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 9 inclusion and 13 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 6 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sandrine Valade, Dr (+33142499419, sandrine.valade@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival with a decrease in SOFA score ≥ 3 points (time frame: At day 7). 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 6 Feb 2024.