RefractoryPhase 3NCT06038422
GTP Regimen in the Treatment of Refractory/Recurrent HLH
A Phase 3 study testing GTP regimen in people with Refractory and Recurrence, run by Beijing Friendship Hospital.
Trial summary
The goal of this clinical trial is to learn about efficacy and safety of GTP regimen in refractory/recurrent hemophagocytic lymphohistiocytosis. The main questions it aims to answer are: Overall remission rate of GTP regimen in R/R HLH Adverse effect of GTP regimen Participants will be treated with GTP regimen Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Refractory, Recurrence, Hemophagocytic Lymphohistiocytosis
- What is being tested?
- GTP regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 15 Sep 2023to 15 Sep 2025
- 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 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients ≥1 month of age with HLH
- Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a family history of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004.
- The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator:
- Previous conventional HLH treatment did not respond.
- The patients were not satisfied with the efficacy of conventional HLH treatment or their condition worsened.
- HLH reactivation occurs. Reactivation is defined as: worsening of two or more HLH clinical and laboratory criteria (after initial remission).
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- According to the researchers, the expected survival was more than 2 weeks.
- The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative.
- Women who are fertile are willing to use a highly effective contraceptive method from the start of the study until 6 months after the end of the last course of treatment.
Who cannot join
- There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection.
- There is uncontrolled active gastrointestinal bleeding.
- The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment.
- A history of hypersensitivity or hypersensitivity to any component of the drug in the study protocol, such as polysorbate.
- Had received the BCG vaccine within 12 weeks prior to screening.
- Have received live or attenuated vaccine (except BCG) within 4 weeks prior to screening.
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- Pregnancy patients.
- Within 4 weeks prior to enrollment in this study, another concurrent clinical intervention study was enrolled.
- There is any condition or circumstance that the investigator believes may cause the patient to be unable to complete the study or to comply with study procedures or requirements.
About this study
Official title: Clinical Study on Efficacy and Safety of GTP Regimen in the Treatment of Refractory/Recurrent Hemophagocytic Lymphohistiocytosis
The goal of this clinical trial is to learn about efficacy and safety of GTP regimen in refractory/recurrent hemophagocytic lymphohistiocytosis. The main questions it aims to answer are:
- Overall remission rate of GTP regimen in R/R HLH
- Adverse effect of GTP regimen Participants will be treated with GTP regimen
Read the detailed description
Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.
- Conditions
- Refractory, Recurrence, Hemophagocytic Lymphohistiocytosis
- Being tested
- GTP regimen
- Medical subject headings
- Recurrence, Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GTP regimenDrug
Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone)
| Study group | Role | What they receive |
|---|---|---|
| GTP regimen treated patients | Experimental | R/R HLH patients treated with GTP regimen Receives: Drug: GTP regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
Overall remission rate
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 posted14 Sep 2023
- Study start15 Sep 2023 (estimated)
- Primary completion15 Sep 2024 (estimated)
- Study completion15 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RefractoryStudies 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
- 15 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
- NCT06038422
- Sponsor's protocol ID
- BFH20230717001
- Status last verified
- Sep 2023
- First submitted
- 8 Sep 2023
- Last updated
- 14 Sep 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on Efficacy and Safety of GTP Regimen in the Treatment of Refractory/Recurrent Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT06038422
- Acronym / sponsor ID
- BFH20230717001
- Conditions
- Refractory, Recurrence, Hemophagocytic Lymphohistiocytosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 15 Sep 2023 (estimated)
- Primary completion
- 15 Sep 2024 (estimated)
- Study completion
- 15 Sep 2025 (estimated)
- First posted
- 14 Sep 2023 (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 goal of this clinical trial is to learn about efficacy and safety of GTP regimen in refractory/recurrent hemophagocytic lymphohistiocytosis. The main questions it aims to answer are:
- Overall remission rate of GTP regimen in R/R HLH
- Adverse effect of GTP regimen Participants will be treated with GTP regimen
Detailed description
Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GTP regimen treated patients | Experimental | Drug: GTP regimenR/R HLH patients treated with GTP regimen |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Male and female patients ≥1 month of age with HLH
- Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a family history of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004.
- The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator:
- According to the researchers, the expected survival was more than 2 weeks.
- The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative.
Key exclusion criteria (9 listed)
- There is active mycobacterium, histoplasma capsulatus, Salmonella, or leishmania infection.
- There is uncontrolled active gastrointestinal bleeding.
- The investigator believes that there are any serious comorbidities or any other conditions that make the patient unsuitable for treatment.
- A history of hypersensitivity or hypersensitivity to any component of the drug in the study protocol, such as polysorbate.
- Had received the BCG vaccine within 12 weeks prior to screening.
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 |
|---|---|---|
| GTP regimenRefractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone) | Drug | GTP regimen treated patients |
Primary outcome
ORR
Time frame: 1 year
Overall remission rate
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GTP regimen in people with Refractory and Recurrence, 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 9 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 14 Sep 2023. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR (time frame: 1 year). 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 14 Sep 2023.