APLPhase 2NCT05881265
Treatment of Chidamide and Venetoclax for Retinoic Acid and Arsenic Resistant Acute Promyelocytic Leukemia
A Phase 2 study testing Chidamide+venetoclax in people with APL, run by Shanghai Jiao Tong University School of Medicine at 5 sites in China.
Trial summary
Based on the current treatment with retinoic acid (ATRA) and arsenic (As), most patients with APL achieved long-term survival. There are few patients relapsed and became refractory to the RA and As treatment. Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- APL
- What is being tested?
- Chidamide+venetoclax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 5 sitesChina
- Study dates
- 15 May 2023to 1 Jan 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with PML-RARα+ APL
- Patients in non-remission status after treatment of RA combined with As
- Patients with life expectance >=3 months
- Inform consent provided
Who cannot join
- Patients with incontrollable infection
- Patients with life-expectancy less than 2 months
- Patients with abnormal liver (>3XN) and renal function (>3XN）
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
China5 sites, 4 recruiting
- Recruiting
NanFang Hospital
Guangzhou
Hongsheng Zhou, 8618665730280, zhs1@i.smu.edu.cn
- Not yet recruiting
The Affiliated Huai An No 1 Perople's Hospital of NanJing University
Huai'an, Jiangsu
Liang Yu, 8613405509177, yuliangha@163.com
- Not yet recruiting
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi
Wenhua Zhou
- Recruiting
Jiong HU
Shanghai, Shanghai Municipality, 200025
Jiong HU, M.D.,, 86-21-64370045, hj10709@rjh.com.cn
Ling Wang, M.D.,, 86-21-64370045, cclingjar@163.com
- Active, not recruiting
Zhaxin Hospital, Go Broad Health Care
Shanghai, Shanghai Municipality
About this study
Official title: Multi-center Phase II Prospective Study for the Treatment of Chidamide and Venetoclax in Patients With All-trans Retinoic Acid (ATRA) and Arsenic (As) Resistant Acute Promyelocytic Leukemia (APL)
Based on the current treatment with retinoic acid (ATRA) and arsenic (As), most patients with APL achieved long-term survival. There are few patients relapsed and became refractory to the RA and As treatment. In our pre-clinical study, we found that targeting histone deacetylase inhibitor 3 (HDAC3)degraded PML-RARa oncoprotein and induced differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor and venetoclax in patients with refractory APL.
Read the detailed description
For patients with newly-diagnosed acute promyelocytic leukemia (APL), the combination therapy retinoic acid (ATRA) combined with arsenic (As) is the mainstay upfront treatment. Most patients enjoy long-term survival with or without chemotherapy. Even though with such good prognosis, there are still some patients relapsed and eventually became refractory to RA and As treatment. In our pre-clinical study, we demonstrate that targeting histone deacetylase inhibitor 3 (HDAC3) could degrade PML-RARa oncoprotein and induce differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this multi-center prospective study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor (chidamide) and venetoclax in patients with refractory APL.
- Conditions
- APL
- Being tested
- Chidamide+venetoclax
- Keywords
- refractory，APL
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chidamide+venetoclaxDrug
Also called Chi-VEN
Chidamide 30mg BIW x 4 weeks; Venetoclax 100mg D1, 200mg D2 and 400mg D3-28
| Study group | Role | What they receive |
|---|---|---|
| Chi-Ven treatment | Experimental | Patients receive chidamide and venetoclax treatment Receives: Drug: Chidamide+venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
complete remission and complete remission with incomplete recovery of peripheral blood count
patients with <5% promyelocytes in bone marrow with or without full recovery of peripheral cell count
Measured: Day 42
Secondary outcomes (5)
Overall survival
Patients alive
Measured: 1 year
Leukemia-free survival
Patients alive without leukemia relapse
Measured: 1 year
Non-relapse mortality
Patients died without documentation of leukemia relapse
Measured: 1 year
relapse
Patients with documentation of leukemia relapse
Measured: 1 year
Early death
Patients died of any causes
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2023
- Study start15 May 2023
- Primary completion1 Jan 2026 (estimated)
- Study completion1 Jan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for APLStudies of the same condition, open and under way.
- More trials run by Shanghai Jiao Tong University School of MedicineThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Chi-Ven treatment
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Clinical Research Center
- Responsible party
- Principal investigator, Jiong HU, Shanghai Jiao Tong University School of Medicine
- Investigators
- Chun Wang, Study director, Zhaxin Hospital, Go Broad Health Care
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05881265
- Sponsor's protocol ID
- R/R-APL-2023
- Status last verified
- Feb 2025
- First submitted
- 16 May 2023
- Last updated
- 18 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data is available only based on request to study director or principle investigator
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center Phase II Prospective Study for the Treatment of Chidamide and Venetoclax in Patients With All-trans Retinoic Acid (ATRA) and Arsenic (As) Resistant Acute Promyelocytic Leukemia (APL)
- ClinicalTrials.gov ID
- NCT05881265
- Acronym / sponsor ID
- R/R-APL-2023
- Conditions
- APL
- Keywords
- refractory，APL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Shanghai Clinical Research Center
- Study start
- 15 May 2023 (actual)
- Primary completion
- 1 Jan 2026 (estimated)
- Study completion
- 1 Jan 2026 (estimated)
- First posted
- 31 May 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.
Based on the current treatment with retinoic acid (ATRA) and arsenic (As), most patients with APL achieved long-term survival. There are few patients relapsed and became refractory to the RA and As treatment. In our pre-clinical study, we found that targeting histone deacetylase inhibitor 3 (HDAC3)degraded PML-RARa oncoprotein and induced differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor and venetoclax in patients with refractory APL.
Detailed description
For patients with newly-diagnosed acute promyelocytic leukemia (APL), the combination therapy retinoic acid (ATRA) combined with arsenic (As) is the mainstay upfront treatment. Most patients enjoy long-term survival with or without chemotherapy. Even though with such good prognosis, there are still some patients relapsed and eventually became refractory to RA and As treatment. In our pre-clinical study, we demonstrate that targeting histone deacetylase inhibitor 3 (HDAC3) could degrade PML-RARa oncoprotein and induce differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this multi-center prospective study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor (chidamide) and venetoclax in patients with refractory APL.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Chi-Ven treatment
| Arm | Type | Receives |
|---|---|---|
| Chi-Ven treatment | Experimental | Drug: Chidamide+venetoclaxPatients receive chidamide and venetoclax treatment |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with PML-RARα+ APL
- Patients in non-remission status after treatment of RA combined with As
- Patients with life expectance >=3 months
- Inform consent provided
Key exclusion criteria (3 listed)
- Patients with incontrollable infection
- Patients with life-expectancy less than 2 months
- Patients with abnormal liver (>3XN) and renal function (>3XN）
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 |
|---|---|---|
| Chidamide+venetoclaxAlso: Chi-VENChidamide 30mg BIW x 4 weeks; Venetoclax 100mg D1, 200mg D2 and 400mg D3-28 | Drug | Chi-Ven treatment |
Primary outcome
complete remission and complete remission with incomplete recovery of peripheral blood count
Time frame: Day 42
patients with <5% promyelocytes in bone marrow with or without full recovery of peripheral cell count
5 secondary outcomes
Overall survival
Time frame: 1 year
Leukemia-free survival
Time frame: 1 year
Non-relapse mortality
Time frame: 1 year
relapse
Time frame: 1 year
Early death
Time frame: 42 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2025.
- Individual participant dataSharing planned: No. Individual participant data is available only based on request to study director or principle investigator
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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.
Chun Wang study director
Zhaxin Hospital, Go Broad Health Care
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Chidamide+venetoclax in people with APL, run by Shanghai Jiao Tong University School of Medicine at 5 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 4 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 18 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chun Wang, M.D., (8613386259777, wangchunsh@medmail.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
complete remission and complete remission with incomplete recovery of peripheral blood count (time frame: Day 42). 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 18 Feb 2025.