JMMLNCT04505995
Azacitidine and Homoharringtonine in JMML
A study testing Azacitidine and homoharringtonine in people with JMML, run by Air Force Military Medical University, China at 1 site in China.
Trial summary
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia… Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- JMML
- What is being tested?
- Azacitidine and homoharringtonine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2020to 1 Jan 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 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent signature, with one of the following:
- somatic mutation in PTPN11
- somatic mutation in KRAS
- somatic mutation in NRAS and HbF % > 5x normal value for age
- clinical diagnosis of neurofibromatosis Type 1.
Who cannot join
- 1. Any condition that would prevent the subject from participating in the study.
- 2. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study.
- 3. Any condition that confounds the ability to interpret data from the study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
Xiao-Fan Zhu, M.D., +86-22-23909001, xfzhu@ihcams.ac.cn
About this study
Official title: Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these agents has not been elucidated in children with JMML. The primary objective is to assess the treatment effect on response rate in this subject population.
- Conditions
- JMML
- Being tested
- Azacitidine and homoharringtonine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine and homoharringtonineDrug
Concommitant use of azacitidine and homoharringtonine
| Study group | Role | What they receive |
|---|---|---|
| Intervention group | Experimental | Not described Receives: Drug: Azacitidine and homoharringtonine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
Defined as proportion of patients with sustained clinical complete remission [cCR] or clinical partial remission [cPR] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).
Measured: Up to 4 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2020
- Study start1 Jan 2020
- Primary completion1 Jan 2023 (estimated)
- Study completion1 Jan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for JMMLStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Air Force Military Medical University, China
- Sponsor type
- University, hospital or other
- Collaborators
- Qilu Hospital of Shandong University, Taian City Central Hospital, The Affiliated Hospital of Qingdao University, The Second Hospital of Hebei Medical University, Children's Hospital of Hebei Province
- Responsible party
- Principal investigator, Lipeng Liu, Air Force Military Medical University, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04505995
- Sponsor's protocol ID
- JMML-CAMS-2020
- Status last verified
- Aug 2020
- First submitted
- 6 Aug 2020
- Last updated
- 10 Aug 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia
- ClinicalTrials.gov ID
- NCT04505995
- Acronym / sponsor ID
- JMML-CAMS-2020
- Conditions
- JMML
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Air Force Military Medical University, ChinaUniversity, hospital or other
- Collaborators
- Qilu Hospital of Shandong University, Taian City Central Hospital, The Affiliated Hospital of Qingdao University, The Second Hospital of Hebei Medical University, Children's Hospital of Hebei Province
- Study start
- 1 Jan 2020 (actual)
- Primary completion
- 1 Jan 2023 (estimated)
- Study completion
- 1 Jan 2025 (estimated)
- First posted
- 10 Aug 2020 (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.
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these agents has not been elucidated in children with JMML. The primary objective is to assess the treatment effect on response rate in this subject population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention group | Experimental | Drug: Azacitidine and homoharringtonine |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent signature, with one of the following:
Key exclusion criteria (3 listed)
- 1. Any condition that would prevent the subject from participating in the study.
- 2. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study.
- 3. Any condition that confounds the ability to interpret data from the study.
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 |
|---|---|---|
| Azacitidine and homoharringtonineConcommitant use of azacitidine and homoharringtonine | Drug | Intervention group |
Primary outcome
Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
Time frame: Up to 4 Months
Defined as proportion of patients with sustained clinical complete remission [cCR] or clinical partial remission [cPR] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2020.
- Individual participant dataSharing planned: Undecided
Related trials for JMML
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 2
Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia
Atypical Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia
RecruitingPhase 1
Phase 1 Study of 6MW5311 in Adults With Relapsed or Refractory Blood Cancers
AML, Multiple Myeloma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingPhase 2
Personalized NK Cell Therapy in CBT
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Azacitidine and homoharringtonine in people with JMML, run by Air Force Military Medical University, China 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 5 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: status not confirmed, as of the sponsor's last update on 10 Aug 2020. 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 Wen-Yu Yang, M.D. (+86-22-23909138, yangwenyu@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months (time frame: Up to 4 Months). 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 10 Aug 2020.