Hemophagocytic LymphohistiocytosisPhase 1/2NCT03795909
Ruxolitinib Combined With Dexamethasone for HLH
A Phase 1/2 study testing Ruxolitinib and Dexamethasone in people with Hemophagocytic Lymphohistiocytosis, run by Capital Research Institute of Pediatrics at 2 sites in China.
Trial summary
A protocol named as "HLH-DR" for patients with refractory and secondary hemophagocytic lymphohistiocytosismay. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Hemophagocytic Lymphohistiocytosis
- What is being tested?
- Ruxolitinib and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 2 sitesChina
- Study dates
- 1 Mar 2017to 1 Mar 2022
- Phase
- Phase 1/2
- 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
- Secondary and refractory HLH.
Who cannot join
- Family HLH.
Trial locations and contacts
2 sites in 1 country.
China2 sites, 2 recruiting
- Recruiting
Yan Yue
Beijing, Chaoyang District, 100020
Yan Yue, MD, 8618515238169 ext. 8618515238169, 18611196921@163.com
Yan Yue, MD, 8618515238169 ext. 8618515238169, adaile123@sina.com
- Recruiting
Yan Yue
Beijing
xiaodong Shi, MD, 008613911601076 ext. 8618515238169, adaile123@sina.com
Yan Yue, MD, 8618515238169 ext. 8618515238169, adaile123@sina.com
About this study
Official title: Ruxolitinib Combined With Dexamethasone for Pediatric Refractory and Secondary Hemophagocytic Lymphohistiocytosis
A protocol named as "HLH-DR" for patients with refractory and secondary hemophagocytic lymphohistiocytosismay.
Read the detailed description
A modified protocol about ruxolitinib combined with dexamethasone which includes oral ruxolitinib (2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）. Dexamethasone (10mg/m2.d) delayes for the first 2 weeks （form week 1 to 2），(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8. Every 2 weeks, patients can be evaluated based the diagnosed index
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Being tested
- Ruxolitinib, Dexamethasone
- 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
Also called Jakafi
Ruxolitinib （2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）
DexamethasoneDrug
Also called Hormone
Dexamethasone (10mg/m2.d) delayes for the first 2 weeks（form week 1 to 2),(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib and Placebo | Experimental | Ruxolitinib 2.5 mg twice daily by oral Receives: Drug: Ruxolitinib; Drug: Dexamethasone |
| Placebo and Ruxolitinib | Placebo comparator | Sugar pill 2.5 mg twice daily by oral Receives: Drug: Ruxolitinib; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Severity of disease
Serum ferritin>2000ng/ml, disease actvie; serum ferritin<2000ng/ml, disease control;
Measured: 2 weeks
Secondary outcomes (1)
Axillary temperature
36<Axillary temperature<37.2, disease control; 37.2<Axillary temperature<38.2, disease partly control; Axillary temperature>38.2, disease active
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2019
- Study start1 Mar 2017
- Primary completion1 Mar 2020 (estimated)
- Study completion1 Mar 2022 (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.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Capital Research Institute of Pediatrics
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Yan Yue, Capital Research Institute of Pediatrics
- Investigators
- Yan Yue, MD, Principal investigator, Capital Institute of Pediatrics, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03795909
- Sponsor's protocol ID
- HLH-DR
- Status last verified
- Jan 2019
- First submitted
- 3 Jan 2019
- Last updated
- 8 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ruxolitinib Combined With Dexamethasone for Pediatric Refractory and Secondary Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT03795909
- Acronym / sponsor ID
- HLH-DR
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Capital Research Institute of PediatricsOther government
- Study start
- 1 Mar 2017 (actual)
- Primary completion
- 1 Mar 2020 (estimated)
- Study completion
- 1 Mar 2022 (estimated)
- First posted
- 8 Jan 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.
A protocol named as "HLH-DR" for patients with refractory and secondary hemophagocytic lymphohistiocytosismay.
Detailed description
A modified protocol about ruxolitinib combined with dexamethasone which includes oral ruxolitinib (2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）. Dexamethasone (10mg/m2.d) delayes for the first 2 weeks （form week 1 to 2），(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8. Every 2 weeks, patients can be evaluated based the diagnosed index
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib and Placebo | Experimental | Drug: Ruxolitinib, Drug: DexamethasoneRuxolitinib 2.5 mg twice daily by oral |
| Placebo and Ruxolitinib | Placebo comparator | Drug: Ruxolitinib, Drug: DexamethasoneSugar pill 2.5 mg twice daily by oral |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Secondary and refractory HLH.
Key exclusion criteria (1 listed)
- Family HLH.
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 |
|---|---|---|
| RuxolitinibAlso: JakafiRuxolitinib （2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years） | Drug | Placebo and Ruxolitinib, Ruxolitinib and Placebo |
| DexamethasoneAlso: HormoneDexamethasone (10mg/m2.d) delayes for the first 2 weeks（form week 1 to 2),(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8. | Drug | Placebo and Ruxolitinib, Ruxolitinib and Placebo |
Primary outcome
Severity of disease
Time frame: 2 weeks
Serum ferritin>2000ng/ml, disease actvie; serum ferritin<2000ng/ml, disease control;
1 secondary outcome
Axillary temperature
Time frame: 2 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2019.
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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.
Yan Yue, MD principal investigator
Capital Institute of Pediatrics, China
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ruxolitinib and Dexamethasone in people with Hemophagocytic Lymphohistiocytosis, run by Capital Research Institute of Pediatrics at 2 sites 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 1 inclusion and 1 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 8 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Yan Yue, MD (8618515238169 ext. 8618515238169, adaile123@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Severity of disease (time frame: 2 weeks). 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 8 Jan 2019.