HLHNCT06048107
Teniposide Incorporating Bu/Cy Conditioning Regimen for HLH With Central Nervous System Involvemen
A study testing Teniposide in people with HLH, run by Beijing Friendship Hospital at 1 site in China.
Trial summary
Hemophagocytic lymphohistiocytosis (HLH) is a highly inflammatory disease involving cytokine storms and is characterised by rapid progression and high mortality. There are no uniform clinical criteria for the diagnosis of CNS-HLH.And there is still a lack of international consensus on the treatment for CNS-HLH. Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- HLH
- What is being tested?
- Teniposide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 22 Oct 2023to 30 Sep 2025
- Phase
- Interventional
- 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 14 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed as HLH according to HLH-04 diagnostic criteria.
- The indications for the presence of allogeneic hematopoietic stem cell transplantation (allo HSCT) include: I. Primary HLH; II. Refractory/recurrent HLH; III. Severe CNS-HLH.
- According to the different causes of HLH, meeting the following conditions is defined as CNS-HLH.
- Definition of CNS-HLH in EBV-HLH: a significant increase of EBV-DNA copies/ml in peripheral blood or tissue or EBER(+) in tissue specimens or peripheral blood. Meanwhile cerebrospinal fluid EBV-DNA ≥5×10^2 copies/ml.
- II.Definition of CNS-HLH in primary HLH: Nervous system symptom/sign or cerebrospinal fluid cell increase (> 5 cells/μ L) and/or cerebrospinal fluid protein abnormality (> 35mg/dL) and/or brain parenchyma or meningeal abnormality in cranial imaging during the onset or course of HLH.
- Definition of CNS-HLH of lymphoma-associated HLH: Cerebrospinal fluid flow cytology or brain histopathology confirmed that lymphoma involved CNS. Cerebrospinal fluid EBV-DNA ≥5×10^2 copies/ml are also defined as CNS-HLH for EBV positive lymphoma-associated HLH.
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- Estimated survival time ≥ 1 month.
- Age 14~60# gender is not limited.
- Total bilirubin ≤ 2 times the upper limit of normal; serum creatinine ≤normal value before study entry.
- Serum HIV antigen or antibody negative.
- HCV antibody negative.
- HBsAg negative, HBcAb negative. If any of the above is positive, then HBV-DNA titer in peripheral blood is required less than 1×10^2 copies/mL before enrollment.
- Cardiac ultrasound LVEF≥50%.
- Women in the child-bearing period must not be pregnant as determined by a pregnancy test and must be willing to use effective contraception during the trial and for ≥ 12 months after the last dose. Pregnant and lactating women cannot participate. All male subjects accept contraception during the trial and for ≥6 months after the last dose.
- Signed informed consent.
Who cannot join
- Heart function above grade II (NYHA).
- Active bleeding of the internal organs(digestive tract, lung, brain, etc.).
- Uncontrolled infection (pulmonary infection, intestinal infection, etc.).
- Severe mental illness.
- Patients are 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
Zhao Wang
Beijing, 100050, China
Zhao Wang, 01080838339, wangzhao@ccmu.edu.cn
About this study
Official title: Teniposide Incorporating Bu/Cy Conditioning Regimen Followed by HSCT for HLH With Central Nervous System Involvement: a Prospective Single-arm Clinical Study
Hemophagocytic lymphohistiocytosis (HLH) is a highly inflammatory disease involving cytokine storms and is characterised by rapid progression and high mortality. There are no uniform clinical criteria for the diagnosis of CNS-HLH.And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of Teniposide incorporating Bu/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for HLH with central nervous system involvement.
Read the detailed description
Hemophagocytic lymphohistiocytosis (HLH) is a highly inflammatory disease involving cytokine storms and is characterised by rapid progression and high mortality. HLH with central nervous system involvement (CNS-HLH) is a type of inflammatory factor storm that affects the blood-brain barrier and further infiltrates brain tissue, causing patients to exhibit corresponding neurological and/or psychiatric symptoms. It can be the first symptom of HLH or occur in the later stages of HLH, and can be an independent symptom or accompanied by other symptoms. In recent years, with the deepening of research on HLH, CNS-HLH has gradually received attention and is often associated with poor prognosis. There are no uniform clinical criteria for the diagnosis of CNS-HLH. And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of Teniposide incorporating Bu/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for CNS-HLH.
- Conditions
- HLH
- Being tested
- Teniposide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TeniposideDrug
Also called VM-26
Teniposide is semisynthetic analogues of podophyllotoxin, and they are used in the treatment of leukemia, lymphoma, and certain sorts of solid tumors.In addition, tiniposide has central permeability and is commonly used in the treatment of central nervous system tumors.
| Study group | Role | What they receive |
|---|---|---|
| a prospective single-arm clinical study | Experimental | Not described Receives: Drug: Teniposide |
What is being measured
The primary outcome is the main question the study is designed to answer.
The clinical remission rate
The number of cases with complete response (CR) and partial response (PR) after ransplantation as a percentage of the total cases.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2023
- Study start22 Oct 2023
- Primary completion30 Sep 2024 (estimated)
- Study completion30 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HLHStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Friendship Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Zhao Wang, Principal investigator, Department of Hematology, Affiliated Beijing Friendship Hospital, Capital Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06048107
- Sponsor's protocol ID
- BFH20230727002/BFHHZS20230169
- Status last verified
- Sep 2023
- First submitted
- 15 Sep 2023
- Last updated
- 2 Nov 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 2 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Teniposide Incorporating Bu/Cy Conditioning Regimen Followed by HSCT for HLH With Central Nervous System Involvement: a Prospective Single-arm Clinical Study
- ClinicalTrials.gov ID
- NCT06048107
- Acronym / sponsor ID
- BFH20230727002/BFHHZS20230169
- Conditions
- HLH
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 22 Oct 2023 (actual)
- Primary completion
- 30 Sep 2024 (estimated)
- Study completion
- 30 Sep 2025 (estimated)
- First posted
- 21 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.
Hemophagocytic lymphohistiocytosis (HLH) is a highly inflammatory disease involving cytokine storms and is characterised by rapid progression and high mortality. There are no uniform clinical criteria for the diagnosis of CNS-HLH.And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of Teniposide incorporating Bu/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for HLH with central nervous system involvement.
Detailed description
Hemophagocytic lymphohistiocytosis (HLH) is a highly inflammatory disease involving cytokine storms and is characterised by rapid progression and high mortality. HLH with central nervous system involvement (CNS-HLH) is a type of inflammatory factor storm that affects the blood-brain barrier and further infiltrates brain tissue, causing patients to exhibit corresponding neurological and/or psychiatric symptoms. It can be the first symptom of HLH or occur in the later stages of HLH, and can be an independent symptom or accompanied by other symptoms. In recent years, with the deepening of research on HLH, CNS-HLH has gradually received attention and is often associated with poor prognosis. There are no uniform clinical criteria for the diagnosis of CNS-HLH. And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of Teniposide incorporating Bu/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for CNS-HLH.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| a prospective single-arm clinical study | Experimental | Drug: Teniposide |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Diagnosed as HLH according to HLH-04 diagnostic criteria.
- The indications for the presence of allogeneic hematopoietic stem cell transplantation (allo HSCT) include: I. Primary HLH; II. Refractory/recurrent HLH; III. Severe CNS-HLH.
- According to the different causes of HLH, meeting the following conditions is defined as CNS-HLH.
- II.Definition of CNS-HLH in primary HLH: Nervous system symptom/sign or cerebrospinal fluid cell increase (> 5 cells/μ L) and/or cerebrospinal fluid protein abnormality (> 35mg/dL) and/or brain parenchyma or meningeal abnormality in cranial imaging during the onset or course of HLH.
- Estimated survival time ≥ 1 month.
Key exclusion criteria (6 listed)
- Heart function above grade II (NYHA).
- Active bleeding of the internal organs(digestive tract, lung, brain, etc.).
- Uncontrolled infection (pulmonary infection, intestinal infection, etc.).
- Severe mental illness.
- Patients are unable to comply during the trial and/or follow-up phase.
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 |
|---|---|---|
| TeniposideAlso: VM-26Teniposide is semisynthetic analogues of podophyllotoxin, and they are used in the treatment of leukemia, lymphoma, and certain sorts of solid tumors.In addition, tiniposide has central permeability and is commonly used in the treatment of central nervous system tumors. | Drug | a prospective single-arm clinical study |
Primary outcome
The clinical remission rate
Time frame: 24 weeks
The number of cases with complete response (CR) and partial response (PR) after ransplantation as a percentage of the total cases.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 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.
Zhao Wang principal investigator
Department of Hematology, Affiliated Beijing Friendship Hospital, Capital Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Teniposide in people with HLH, 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 15 inclusion and 6 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 2 Nov 2023. 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.
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 Zhao Wang (010-80838339, wangzhao@ccmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The clinical remission rate (time frame: 24 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 2 Nov 2023.