Hemophagocytic LymphohistiocytosisPhase 1NCT05546060
Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in HLH
A Phase 1 study testing Venetoclax, Dexamethasone and Etoposide 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 venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophagocytic Lymphohistiocytosis
- What is being tested?
- Venetoclax, Dexamethasone and Etoposide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2022to 1 Oct 2025
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- age ≥18 years old, expected survival time more than 3 months;
- met HLH-2004 diagnostic criteria；
- ECOG score 0-2；
- ECG QTcF interval: male ≤450ms, female ≤470ms;
- AST and ALT ≤3.0 ULN, TB ≤1.5×ULN；serum creatinine≤1.5×ULN，or CrCL ≥ 50mL/min；INR、APTT、PT ≤1.5×ULN；
- without pregnancy or lactation, and agree to contraception during and for at least 6 months after the study；
- signed informed consent.
Who cannot join
- patients with malignancies unrelated to HLH, except for fully recovered non-melanoma skin cancer and carcinoma in situ;
- patients participated in other clinical trials within 4 weeks；
- previously treated with Bcl-2 inhibitors；
- unable to take oral medication；
- history of substance abuse or patients with mental illness；
- severe infection；
- cardiovascular disease，NYHA II-IV；
- allergic to venetoclax or etoposide.
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, MD, 63138303, wangzhao@ccmu.edu.cn
About this study
Official title: Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in the Treatment of Hemophagocytic Lymphohistiocytosis
This study aimed to investigate the efficacy and safety of venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis.
Read the detailed description
Hemophagocytic lymphohistiocytosis (HLH) is a rare and aggressive disease with high mortality and poor prognosis. The HLH-94 and HLH-04 regimens are most commonly used, but approximately 30% of the patients remain unresponsive. Currently, there is no unified evidence-based salvage treatment. There is no unified salvage regimen. This study plans to enroll 20 patients with refractory and recurrent hemophagocytic lymphohistiocytosis. Subjects will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.The safety was evaluated once a week, and the efficacy was evaluated once every 2 weeks. Overall response rate will be evaluated at 8 weeks. Patients were followed up every 3 months thereafter.
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Being tested
- Venetoclax, Dexamethasone, Etoposide
- Keywords
- Hemophagocytic Lymphohistiocytosis, Venetoclax
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenetoclaxDrug
Venetoclax 100mg qd d1，200mg qd d2，400mg qd d3-56
DexamethasoneDrug
Dexamethasone 10mg/m2 qd d1-14, 5mg/m2 qd d15-28, 2.5 mg/m2 qd d29-42, and 1.25mg/m2 qd43-56
EtoposideDrug
Etoposide 75mg/m2 qw d1-56
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax Combined With Dexamethasone and Etoposide | Experimental | Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks. Receives: Drug: Venetoclax; Drug: Dexamethasone; Drug: Etoposide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases
Measured: 1 year
Incidence and severity of adverse effects
The percentage of cases with adverse effects and its severity.
Measured: 1 years
Secondary outcomes (1)
Overall survival
OS is defined as the time from the initiaion of treatment to death from any cause or July 2025.
Measured: 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2022
- Study start1 Jul 2022
- Primary completion1 Jul 2025 (estimated)
- Study completion1 Oct 2025 (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 1
- 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
- Principal investigator, Zhao Wang, Beijing Friendship Hospital
- Investigators
- Zhao Wang, MD, Principal investigator, Beijing Friendship Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05546060
- Sponsor's protocol ID
- BFH20220425006/BFHHZS20220026
- Status last verified
- Sep 2022
- First submitted
- 15 Sep 2022
- Last updated
- 19 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- 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 19 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in the Treatment of Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT05546060
- Acronym / sponsor ID
- BFH20220425006/BFHHZS20220026
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Keywords
- Hemophagocytic Lymphohistiocytosis, Venetoclax
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Study start
- 1 Jul 2022 (actual)
- Primary completion
- 1 Jul 2025 (estimated)
- Study completion
- 1 Oct 2025 (estimated)
- First posted
- 19 Sep 2022 (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 venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis.
Detailed description
Hemophagocytic lymphohistiocytosis (HLH) is a rare and aggressive disease with high mortality and poor prognosis. The HLH-94 and HLH-04 regimens are most commonly used, but approximately 30% of the patients remain unresponsive. Currently, there is no unified evidence-based salvage treatment. There is no unified salvage regimen. This study plans to enroll 20 patients with refractory and recurrent hemophagocytic lymphohistiocytosis. Subjects will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.The safety was evaluated once a week, and the efficacy was evaluated once every 2 weeks. Overall response rate will be evaluated at 8 weeks. Patients were followed up every 3 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax Combined With Dexamethasone and Etoposide | Experimental | Drug: Venetoclax, Drug: Dexamethasone, Drug: EtoposidePatients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- age ≥18 years old, expected survival time more than 3 months;
- met HLH-2004 diagnostic criteria；
- ECOG score 0-2；
- ECG QTcF interval: male ≤450ms, female ≤470ms;
- AST and ALT ≤3.0 ULN, TB ≤1.5×ULN；serum creatinine≤1.5×ULN，or CrCL ≥ 50mL/min；INR、APTT、PT ≤1.5×ULN；
Key exclusion criteria (8 listed)
- patients with malignancies unrelated to HLH, except for fully recovered non-melanoma skin cancer and carcinoma in situ;
- patients participated in other clinical trials within 4 weeks；
- previously treated with Bcl-2 inhibitors；
- unable to take oral medication；
- history of substance abuse or patients with 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 |
|---|---|---|
| VenetoclaxVenetoclax 100mg qd d1，200mg qd d2，400mg qd d3-56 | Drug | Venetoclax Combined With Dexamethasone and Etoposide |
| DexamethasoneDexamethasone 10mg/m2 qd d1-14, 5mg/m2 qd d15-28, 2.5 mg/m2 qd d29-42, and 1.25mg/m2 qd43-56 | Drug | Venetoclax Combined With Dexamethasone and Etoposide |
| EtoposideEtoposide 75mg/m2 qw d1-56 | Drug | Venetoclax Combined With Dexamethasone and Etoposide |
Primary outcome
Overall response rate
Time frame: 1 year
The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases
Incidence and severity of adverse effects
Time frame: 1 years
The percentage of cases with adverse effects and its severity.
1 secondary outcome
Overall survival
Time frame: 1 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2022.
- Individual participant dataSharing planned: No
Related trials for Hemophagocytic Lymphohistiocytosis
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study
Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis
Not yet recruitingObservational study
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis After Allogeneic Stem Cell Transplantation
Hemophagocytic Lymphohistiocytosis (HLH)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
CytOSorb TreatMent Of Critically Ill PatientS Registry
Septic Shock, Acute Respiratory Distress Syndrome
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, MD principal investigator
Beijing Friendship Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Venetoclax, Dexamethasone and Etoposide 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 7 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 19 Sep 2022. 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 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.
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 Zhao Wang, MD (63138303, wangzhao@ccmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: 1 year). 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 19 Sep 2022.