Acute Myelogenous Leukemia AMLPhase 2NCT02124174
Vidaza and Valproic Acid Post Allogeneic Transplant for High Risk AML and MDS
A Phase 2 study testing Vidaza and Valproic Acid in people with Acute Myelogenous Leukemia AML and Myelodysplastic Syndrome MDS, run by Patrick Stiff at 1 site in United States.
Trial summary
Phase II trial combining azacitidine with valproic acid as maintenance therapy post allogeneic stem cell transplantation in patients with high-risk MDS/AML. We hypothesize that adding valproic acid to azacitidine will improve outcomes via both direct anti-tumor and immunologically mediated antitumor response with… Read more about this study
- Who is it for?
- Ages 2 to 89 years
- Conditions the study is for
- Acute Myelogenous Leukemia AML, Myelodysplastic Syndrome MDS
- What is being tested?
- Vidaza and Valproic Acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2012to Jan 2022
- Phase
- Phase 2
- 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 2 to 89 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All allograft patients > 2 years of age.
- Patients will have one of the following malignancies:
- Patients with refractory or relapsed: acute myelogenous leukemia (AML) (including inv16, t(8;21) or t(15;17)) or high risk myelodysplastic syndrome (MDS) (defined as bone marrow blasts > or = 5%) are eligible. Patients may be in remission at the time of entry.
- Patients with adequate organ function and performance status criteria measured by:
- Karnofsky score greater than or equal to 70% or Performance status of < or = 2 by the Eastern Cooperative Oncology Group (ECOG) scale
- Adequate liver function (bilirubin of < 2mg/dL, serum glutamate pyruvate transaminase < 3 * ULN) and renal function (creatinine < 2mg/dL)
- Signed informed consent indicating that patients are aware of the investigational nature of this study in accordance with the regulations of Loyola University Medical Center
- Patients must have undergone allogeneic stem cell transplant within 40-60 days before starting treatment and be self-sufficient in caloric intake along with no active graft vs. host disease
Who cannot join
- Nursing and pregnant females are excluded.
- Active and uncontrolled infections will cause patients to be excluded.
- Patients already receiving valproic acid or receiving other anticonvulsants will be excluded.
- Low risk AML in complete remission 1, will not be candidates for this study.
- Patients with an absolute neutrophil count less than 1500 will be excluded
- Patients with platelets less than 50,000 will be excluded
- Children less than 2 years of age will be excluded due to increased hepatotoxicity from valproic acid in this age group
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Loyola University Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153
Mary Lee, BSN
Ceil Petrowsky, MSN, cpetrow@luc.edu
About this study
Official title: Maintenance Therapy With Azacitidine and Valproic Acid After Allogeneic Stem Cell Transplant in Patients With High-Risk Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)(Version 1_06 Jan 2012)
Phase II trial combining azacitidine with valproic acid as maintenance therapy post allogeneic stem cell transplantation in patients with high-risk MDS/AML. We hypothesize that adding valproic acid to azacitidine will improve outcomes via both direct anti-tumor and immunologically mediated antitumor response with alloreactive donor lymphocytes, having an additive effect and extending 1 year survival in patient with high-risk AML/MDS after hematopoietic stem cell transplant. Based on aforementioned data from the US Department of Health and Human Services, standard 1 year survival for AML after stem cell transplant is near 40%. We hypothesize that valproic acid and azacitidine will prolong survival, with a 1 year survival goal of 60%. In addition to assessing for 1 year survival, we will have secondary objectives of assessing progression-free survival, relapse, and toxicity. The primary toxicity endpoint from this will be cytopenias and infections.
Read the detailed description
To assess the combination of valproic acid and azacitidine in preventing relapse in patients with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant. The primary objective of this study will be determining the 1 year overall survival from combining valproic acid (VPA) with 5-azacytidine (5-aza).
To assess the effect that adding valproic acid to azacitidine will have in patient with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant on the following endpoints
- Being tested
- Vidaza and Valproic Acid
- Keywords
- acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS)
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vidaza and Valproic AcidDrug
Also called Vidaza, Valproic Acid, Azacitadine
Days 1-5: 5-Azacytidine 40 mg/m^2 daily Days 1-5: +Valproic acid 15 mg/kg daily Days 6-28: Valproic acid 15 mg/kg daily
*treatments will be repeated on the same days of each cycle for up to 4 total cycles. Each cycle will consist of 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Vidaza and Valproic Acid | Experimental | Vidaza and Valproic Acid Receives: Drug: Vidaza and Valproic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
Number of participants that survive post transplant for 1 year.
Measured: 1 year
Secondary outcomes (1)
Disease Relapse
The time to relapse is from Day 0 to the day of first hematologic, cytogenetic, or radiological evidence of recurrent disease.
Measured: Day 0 to the day of first recurrance
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2014 (estimated)
- Study startJan 2012
- Primary completionJan 2022 (estimated)
- Study completionJan 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myelogenous Leukemia AMLStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Patrick Stiff
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Patrick Stiff, Loyola University
- Investigators
- Patrick Stiff, MD, Principal investigator, Faculty
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02124174
- Sponsor's protocol ID
- 203835
- Status last verified
- Apr 2021
- First submitted
- 24 Apr 2014
- Last updated
- 26 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Maintenance Therapy With Azacitidine and Valproic Acid After Allogeneic Stem Cell Transplant in Patients With High-Risk Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)(Version 1_06 Jan 2012)
- ClinicalTrials.gov ID
- NCT02124174
- Acronym / sponsor ID
- 203835
- Keywords
- acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Patrick StiffUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- Jan 2022 (estimated)
- Study completion
- Jan 2022 (estimated)
- First posted
- 28 Apr 2014 (estimated)
- 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.
Phase II trial combining azacitidine with valproic acid as maintenance therapy post allogeneic stem cell transplantation in patients with high-risk MDS/AML. We hypothesize that adding valproic acid to azacitidine will improve outcomes via both direct anti-tumor and immunologically mediated antitumor response with alloreactive donor lymphocytes, having an additive effect and extending 1 year survival in patient with high-risk AML/MDS after hematopoietic stem cell transplant. Based on aforementioned data from the US Department of Health and Human Services, standard 1 year survival for AML after stem cell transplant is near 40%. We hypothesize that valproic acid and azacitidine will prolong survival, with a 1 year survival goal of 60%. In addition to assessing for 1 year survival, we will have secondary objectives of assessing progression-free survival, relapse, and toxicity. The primary toxicity endpoint from this will be cytopenias and infections.
Detailed description
To assess the combination of valproic acid and azacitidine in preventing relapse in patients with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant. The primary objective of this study will be determining the 1 year overall survival from combining valproic acid (VPA) with 5-azacytidine (5-aza).
To assess the effect that adding valproic acid to azacitidine will have in patient with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant on the following endpoints
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vidaza and Valproic Acid | Experimental | Drug: Vidaza and Valproic AcidVidaza and Valproic Acid |
Who may qualify
- Ages
- 2 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- All allograft patients > 2 years of age.
- Patients will have one of the following malignancies:
- Patients with adequate organ function and performance status criteria measured by:
- Signed informed consent indicating that patients are aware of the investigational nature of this study in accordance with the regulations of Loyola University Medical Center
- Patients must have undergone allogeneic stem cell transplant within 40-60 days before starting treatment and be self-sufficient in caloric intake along with no active graft vs. host disease
Key exclusion criteria (7 listed)
- Nursing and pregnant females are excluded.
- Active and uncontrolled infections will cause patients to be excluded.
- Patients already receiving valproic acid or receiving other anticonvulsants will be excluded.
- Low risk AML in complete remission 1, will not be candidates for this study.
- Patients with an absolute neutrophil count less than 1500 will be excluded
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 |
|---|---|---|
| Vidaza and Valproic AcidAlso: Vidaza, Valproic Acid, AzacitadineDays 1-5: 5-Azacytidine 40 mg/m^2 daily Days 1-5: +Valproic acid 15 mg/kg daily Days 6-28: Valproic acid 15 mg/kg daily *treatments will be repeated on the same days of each cycle for up to 4 total cycles. Each cycle will consist of 28 days. | Drug | Vidaza and Valproic Acid |
Primary outcome
Survival
Time frame: 1 year
Number of participants that survive post transplant for 1 year.
1 secondary outcome
Disease Relapse
Time frame: Day 0 to the day of first recurrance
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2021.
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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.
Patrick Stiff, MD principal investigator
Faculty
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vidaza and Valproic Acid in people with Acute Myelogenous Leukemia AML and Myelodysplastic Syndrome MDS, run by Patrick Stiff at 1 site in United States.
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 8 inclusion and 7 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 26 Apr 2021. 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 United States. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mary Lee, BSN (708-327-2241, mlee@luc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (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 26 Apr 2021; AzTrials last refreshed its copy on 26 Oct 2026.