Macrophage Activation SyndromePhase 3NCT05137496
Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
A Phase 3 study testing Ruxolitinib and methylprednisolone in people with Macrophage Activation Syndrome, run by Beijing Friendship Hospital.
Trial summary
The present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy . Read more about this study
- Who is it for?
- Ages 1 year to 75 years
- Conditions the study is for
- Macrophage Activation Syndrome
- What is being tested?
- Ruxolitinib and methylprednisolone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2022to 1 Jun 2024
- 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 1 year to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
- No HLH induction therapy was performed.
- The expected survival time is more than 1 month.
- Serum creatinine ≤ 1.5 times normal；Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；HBV copies less than 1E+03 copies/ml.
- Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
- The left ventricular ejection fraction (LVEF) was normal.
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- No uncontrollable infection.
- Contraception for both male or female.
- Informed consent obtained.
Who cannot join
- Pregnancy or lactating Women；
- Allergic to ruxolitinib；
- Active bleeding of the internal organs；
- uncontrollable infection；
- Serious mental illness;
- Non-melanoma skin cancer history;
- Patients unable to comply during the trial and/or follow-up phase;
- Participate in other clinical research at the same time.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
The present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy .
- Conditions
- Macrophage Activation Syndrome
- Being tested
- Ruxolitinib, methylprednisolone
- Medical subject headings
- Macrophage Activation Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
0.3mg/kg/d, iv.gtt, for at least 2 weeks
2mg/kg, d1-d5, gradually reduced, for at least 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib+methylprednisolone | Experimental | Ruxolitinib and methylprednisolone administered as the first-line therapy Receives: Drug: Ruxolitinib; Drug: methylprednisolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT).
A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of<0.5 ×109/L, a response was defined as an increase by at least 100% to>0.5× 109/L; for patients with a neutrophil count of 0.5 to 2.0 × 109/L, an increase by at least 100% to >2.0 × 109/L was considered a response; and for patients with ALT >400 U/L, response was defined as an ALT decrease of at least 50%.
Measured: Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy
Progression Free Survival
from date of inclusion to date of progression, relapse, or death from any cause
Measured: 2 years
Adverse events
Adverse events including myelosuppression, infection, hemorrhage
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Nov 2021
- Study start1 Jun 2022 (estimated)
- Primary completion1 Jun 2023 (estimated)
- Study completion1 Jun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Macrophage Activation SyndromeStudies of the same condition, open and under way.
- More trials run by Beijing Friendship HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Friendship Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhao Wang, Beijing Friendship Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05137496
- Sponsor's protocol ID
- Ruxolitinib-steroids-MAS
- Status last verified
- Mar 2022
- First submitted
- 24 Nov 2021
- Last updated
- 17 Mar 2022
- Results posted
- No
- 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 17 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
- ClinicalTrials.gov ID
- NCT05137496
- Acronym / sponsor ID
- Ruxolitinib-steroids-MAS
- Conditions
- Macrophage Activation Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 1 Jun 2022 (estimated)
- Primary completion
- 1 Jun 2023 (estimated)
- Study completion
- 1 Jun 2024 (estimated)
- First posted
- 30 Nov 2021 (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 present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy .
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib+methylprednisolone | Experimental | Drug: Ruxolitinib, Drug: methylprednisoloneRuxolitinib and methylprednisolone administered as the first-line therapy |
Who may qualify
- Ages
- 1 year to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
- No HLH induction therapy was performed.
- The expected survival time is more than 1 month.
- Serum creatinine ≤ 1.5 times normal；Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；HBV copies less than 1E+03 copies/ml.
- Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
Key exclusion criteria (8 listed)
- Pregnancy or lactating Women；
- Allergic to ruxolitinib；
- Active bleeding of the internal organs；
- uncontrollable infection；
- Serious mental illness;
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 |
|---|---|---|
| Ruxolitinib0.3mg/kg/d, iv.gtt, for at least 2 weeks | Drug | Ruxolitinib+methylprednisolone |
| methylprednisolone2mg/kg, d1-d5, gradually reduced, for at least 2 weeks | Drug | Ruxolitinib+methylprednisolone |
Primary outcome
Response rate
Time frame: Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy
A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of<0.5 ×109/L, a response was defined as an increase by at least 100% to>0.5× 109/L; for patients with a neutrophil count of 0.5 to 2.0 × 109/L, an increase by at least 100% to >2.0 × 109/L was considered a response; and for patients with ALT >400 U/L, response was defined as an ALT decrease of at least 50%.
Progression Free Survival
Time frame: 2 years
from date of inclusion to date of progression, relapse, or death from any cause
Adverse events
Time frame: 2 years
Adverse events including myelosuppression, infection, hemorrhage
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2022.
- 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 3 study testing Ruxolitinib and methylprednisolone in people with Macrophage Activation Syndrome, run by Beijing Friendship Hospital.
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 8 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 17 Mar 2022. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yue Song (86-010-63139862, xueqifeng1992@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response rate (time frame: Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy). The study lists 3 primary outcomes in all. 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 17 Mar 2022.