Hemophagocytic LymphohistiocytosisPhase 3NCT04120090
Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis
A Phase 3 study testing low dose ruxolitinib and high dose ruxolitinib in people with Hemophagocytic Lymphohistiocytosis, run by Beijing Friendship Hospital at 1 site in China.
Trial summary
This study aimed to investigate the efficacy and safety of different doses of ruxolitinib as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis(HLH). Read more about this study
- Who is it for?
- Ages 1 year to 75 years
- Conditions the study is for
- Hemophagocytic Lymphohistiocytosis
- What is being tested?
- low dose ruxolitinib and high dose ruxolitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2019to 1 Jul 2020
- 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;
- treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
- Life expectancy exceeds 1 month;
- Age≥1 year old and ≤75 years old, gender is not limited；
- Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
- Serum human immunodeficiency virus(HIV) antigen or antibody negative；
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- Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；
- Both hepatitis B virus (HBV) surface antigen and HBV core antibody were negative. If any of the above is positive, peripheral blood hepatitis B virus DNA titer should be detected, and less than 1×103 copies/ml can enter the group；
- Informed consent.
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
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality, 100050
zhao wang
About this study
Official title: Clinical Study of Different Doses of Ruxolitinib as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis
This study aimed to investigate the efficacy and safety of different doses of ruxolitinib as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis(HLH).
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Being tested
- low dose ruxolitinib, high dose 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.
low dose ruxolitinibDrug
The dose for adult patients(Age>=14 years) is generally 10 mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 5mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 2.5mg twice daily.
high dose ruxolitinibDrug
The dose for adult patients(Age>=14 years) is generally 20mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 10mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 5mg twice daily
| Study group | Role | What they receive |
|---|---|---|
| Low dose | Active comparator | Not described Receives: Drug: low dose ruxolitinib |
| High dose | Experimental | Not described Receives: Drug: high dose ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
complete response (CR) and partial response (PR) rates
Measured: 1 years
Progression Free Survival
from date of inclusion to date of progression, relapse, or death from any cause Overall Survival
Measured: 1 years
Secondary outcomes (2)
Overall Survival
from the date of inclusion to date of death, irrespective of cause Adverse Events
Measured: 1 years
Adverse events
Adverse events including myelosuppression, infection, bleeding and so on.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2019
- Study start1 Jul 2019
- Primary completion1 Jul 2020 (estimated)
- Study completion1 Jul 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophagocytic LymphohistiocytosisStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 80 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
- NCT04120090
- Sponsor's protocol ID
- Ruxolitinib-HLH
- Status last verified
- Oct 2019
- First submitted
- 6 Oct 2019
- Last updated
- 29 Oct 2019
- 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 29 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Different Doses of Ruxolitinib as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT04120090
- Acronym / sponsor ID
- Ruxolitinib-HLH
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- 1 Jul 2020 (estimated)
- Study completion
- 1 Jul 2020 (estimated)
- First posted
- 9 Oct 2019 (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.
This study aimed to investigate the efficacy and safety of different doses of ruxolitinib as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis(HLH).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low dose | Active comparator | Drug: low dose ruxolitinib |
| High dose | Experimental | Drug: high dose ruxolitinib |
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;
- treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
- Life expectancy exceeds 1 month;
- Age≥1 year old and ≤75 years old, gender is not limited；
- Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
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 |
|---|---|---|
| low dose ruxolitinibThe dose for adult patients(Age>=14 years) is generally 10 mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 5mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 2.5mg twice daily. | Drug | Low dose |
| high dose ruxolitinibThe dose for adult patients(Age>=14 years) is generally 20mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 10mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 5mg twice daily | Drug | High dose |
Primary outcome
Response rate
Time frame: 1 years
complete response (CR) and partial response (PR) rates
Progression Free Survival
Time frame: 1 years
from date of inclusion to date of progression, relapse, or death from any cause Overall Survival
2 secondary outcomes
Overall Survival
Time frame: 1 years
Adverse events
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2019.
- 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. This study is not recruiting at the moment.
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 low dose ruxolitinib and high dose ruxolitinib in people with Hemophagocytic Lymphohistiocytosis, run by Beijing Friendship Hospital at 1 site in China.
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 29 Oct 2019. 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 China. 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. 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 jingshi wang (86-010-63139862, wangjingshi987@126.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: 1 years). The study lists 2 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 29 Oct 2019.