Acute Myeloid LeukemiaPhase 2NCT06180863

Oral Azacitidine (CC-486) Epigenetic Priming and Maintenance for Adult Acute Myeloid Leukemia (AML) Patients

A Phase 2 study testing oral azacitidine (CC-486) in people with Acute Myeloid Leukemia, run by Yale University at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

To investigate the feasibility of delivering oral azacitidine (CC-486) as a consolidation regimen from the time of first complete remission (CR1), in patients with acute myelogenous leukemia (AML) eligible for curative intent Allogeneic Stem Cell Transplant (ASCT). Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Acute Myeloid Leukemia
What is being tested?: oral azacitidine (CC-486)
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 1 siteUnited States
Study dates: Nov 2023to Apr 2029
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 18 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must have acute myeloid leukemia (AML) of Intermediate or High risk (IR or HR per National Comprehensive Cancer [NCCN] Criteria) in first CR/CRi achieved with 1 or 2 cycles of cytarabine based induction therapy. Zero to 1 cycle of cytarabine based consolidation will be allowed post CR1. OR Nucleophosmin 1(NPM 1) positive AML ((in the absence of FMS like tyrosine kinase 3 (FLT3) mutation)) at diagnosis with positive NPM1 PCR (measurable residual disease [MRD]) after at least 1 cycle of cytarabine-based consolidation chemotherapy OR Secondary AML(s-AML) based on prior MDS or CMML in first CR/CRi. OR Therapy-related AML (t-AML) with IR or HR criteria
Vyxeos induction (up to 2 cycles to reach CR1) and 0-1 cycle of vyxeos consolidation will be allowed.
Eligible to receive RIC or Non myeloablative (NMA) transplant preparative regimen with post-transplant cyclophosphamide (PTCY) based GVHD prophylaxis, at the discretion of study investigator.
Patients must have a calculated creatinine clearance (Cockcroft-Gault equation) > 50 mL/min.
Patients must have a related or unrelated donor. Sibling donor must be a 6/6 match for human leukocyte antigen A (HLA-A) and HLA-B at intermediate or higher resolution, and DRB1 at high resolution using DNA-based typing; must be willing to donate peripheral blood stem cells; and meet institutional criteria for donation. Unrelated donor must be a 7/8 or 8/8 match at HLA-A, -B, -C, and -DRB1 at high resolution using DNA-based typing; must be willing to donate peripheral blood mononuclear cells (PBSCs); and be medically eligible to donate stem cells according to National Marrow Donor Program (NMDP) criteria.
Patients must be willing to have peripheral blood stem cells as the graft source.

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Patients must have adequate hepatic function (i.e., serum bilirubin level ≤1.5 times the upper limit of normal [ULN], aspartate aminotransferase (AST), and alanine aminotransferase (ALT), defined as ≤2.5 × ULN). Higher levels of bilirubin are acceptable in the setting of Gilbert's disease with no other provable etiologies for altered liver function.
Patients must have a Karnofsky performance score (KPS) ≥70 and/or ECOG score of ≤2.
Patients must have recovered from the toxicities of the most recent induction/consolidation chemotherapy. Recovery is defined by the absence of persistent treatment-related AE of grade 2 or above. Persistent cytopenia meeting the criteria for a CRi and controlled infections on antimicrobials is acceptable.
Women of childbearing potential must be surgically sterile or agree to practice abstinence or utilize acceptable contraception (intrauterine, injectable, transdermal, or combination oral contraceptive) during the period while receiving study medication and for at least 6 months after the last dose of CC-486. Males who are sexually active with women of childbearing potential must be surgically sterile or using an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) from time of screening through at least 3 months after the last dose of CC-486.
Patients must be able to understand the study procedures, agree to participate in the study program, and voluntarily provide written Informed Consent
Patients must have no active COVID-19 infection symptoms at the time of enrollment. Those who tested positive in the past or made recovery post infection must be symptom free for at least 2 weeks prior to enrollment.
Patients must be able to start transplant preparative regimens no later than 35 days from Day 1 of the most recent CC-486 cycle administration.

Who cannot join

Patients may not have Acute Promyelocytic leukemia
Patients may not have favorable risk AML per National Comprehensive Cancer Network (NCCN) guidelines ((Core binding factor leukemias, CCAAT/enhancer-binding protein-alpha (CEBPA) double mutant, AML with concomitant mutations in Nucleophosmin 1 (NPM1) and FLT3))
Patients must not have received more than two rounds of chemotherapy to achieve first CR or CRi
Patients may not have had exposure to hypomethylating agent (HMA) to treat the leukemia or prior hematologic malignancy ((i.e., myelodysplastic syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML)) in the 6 months prior to study enrollment.
Patients may not be, per investigator evaluation, currently in need of other leukemia-directed therapy such as FLT3- or Isocitrate dehydrogenase (IDH)-directed therapy.
Patients may not have myeloproliferative neoplasm progressing to AML (except for CMML)

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With related and unrelated donors, patients may not have high pre-transplant donor-specific HLA antibodies warranting need for desensitization or other maneuvers per discretion of the treating physician.
Patients may not have previously received radiation to maximum tolerable limits to any critical normal organ.
Patients with prior Central Nervous System (CNS) involvement or extramedullary disease will be excluded.
Patients may not have previously received allogeneic Hematopoietic Cell Transplantation (HCT).
Patients may not have clinically significant cardiac disease ((New York Heart Association (NYHA) Class III or IV)); clinically significant arrhythmia i.e., ventricular tachycardia, ventricular fibrillation, or "Torsade de Pointes." Significant active cardiac disease within the previous 6 months including NYHA class 4 Congestive Heart Failure (CHF), Unstable angina, and Myocardial Infarction
Patients may not have abnormal QTcF (>480 msec) after electrolytes have been corrected (at least two different ECG readings and at least 15 minutes between readings). Subjects with paced rhythm or prolonged QTcF may be exempt from this exclusion if considered eligible for transplant per treating physician clinical judgement with optional cardiology consultation.
Patients may not have positive test results for human immunodeficiency virus (HIV) or hepatitis B (HBV) or C (HCV). Subjects with a past positive HBV test results due to previous exposure but who have cleared the virus or are vaccinated against hepatitis B, as evidenced by negative hepatitis B surface antigen (HbsAg), negative HBV viral load, and positive antibody to the HbsAg (anti-HBs) are not excluded. Subjects who have positive hepatitis test results with adequate organ function as defined in the protocol are not excluded.
Patients may not have uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment)
Patients may not have had an active malignancy within 1 year of entry. Active malignancy is defined as those malignancies requiring treatment with anti-cancer therapy or with a survival prognosis of less than 2 years at the time of inclusion. Exceptions to this exclusion include myelodysplastic syndrome/CMML, treated non-melanoma skin cancer, completely resected Stage 0 or 1 melanoma no less than 1 year from resection, cervical carcinoma in situ or cervical intraepithelial neoplasia, Breast cancers on long term hormonal therapy (1 year or more), and successfully treated organ-confined prostate cancer with no evidence of progressive disease based on prostate specific antigen (PSA) levels and not on active therapy.
Patients may not be currently receiving any other investigational agents for relapse reduction.
Patients seeking umbilical cord blood transplants, or bone marrow graft, or mismatched unrelated donor (MMUD) with <9/10 HLA match are ineligible.
Ex-vivo T cell depletion or use of campath, antithymocyte globulin (ATG), or other anti-T cell antibodies as GVHD prophylaxis are not allowed. Patients are allowed to receive abatacept as approved by FDA.
Patients may not have known uncontrolled and active alcohol or substance abuse
Patients may not have any ongoing medical and non-medical condition that may render the patient ineligible for ASCT
Pregnant or breast-feeding females are not eligible. (Lactating females must agree not to breast feed while taking CC-486).
Patients may not have a history of inflammatory bowel disease (e.g., Crohn's disease, ulcerative colitis), celiac disease (i.e., sprue), prior gastrectomy or upper bowel removal, or any other gastrointestinal disorder or defect that would interfere with the absorption, distribution, metabolism, or excretion of the study drug and/or predispose the subject to an increased risk of gastrointestinal toxicity
Patients may not have abnormal coagulation parameters (PT >15 seconds, PTT>40 seconds, and/or INR >1.5). Patients with chronic anticoagulation can be considered for inclusion after discussion with the PI
Patients may not have known or suspected hypersensitivity to azacitidine or mannitol
Patients may not have any significant medical, surgical, or mental health problem that would prevent appropriate patient participation in the study
Patients may not have any conditions including, but not limited to, laboratory abnormalities which put patients at unacceptable risk as per evaluation of treating physician
Patients may not have a history of idiopathic thrombocytopenic purpura, disseminated intravascular coagulation, thrombotic thrombocytopenic purpura (TTP) or hemolytic uremic syndrome (HUS)

Trial locations and contacts

1 site in 1 country.

United States1 site

Yale Comprehensive Cancer Center
New Haven, Connecticut, 06520

Other trials in United States

About this study

Official title: A Pilot Study of Oral Azacitidine (CC-486) Epigenetic Priming and Maintenance in Adult AML Patients Eligible for Reduced Intensity Allogeneic Stem Cell Transplant

Read the detailed description

A significant proportion of patients with Acute myeloid leukemia (AML) who are fit to receive intensive chemotherapy and reach first complete remission (CR), relapse despite further high or low intensity therapies. Relapse/refractory (R/R) AML generally portends poor outcomes and available agents to treat this condition have modest efficacy. Until the recent approval of CC-486 as a maintenance therapy, no other drug had shown overall survival (OS) advantage for patients with AML in first complete remission (CR1). In the CC-486 registration trial (QUAZAR AML-01), remission induction was achieved by intensive chemotherapy, but patients were ineligible to proceed to allogeneic stem cell transplant (ASCT) at the time of screening. Post CR1, CC-486 apart from prolonging remission was also successful in deepening the quality of remission by eradicating measurable residual disease (MRD). It is therefore logical to investigate the feasibility of CC-486 as a consolidation regimen from the time of CR1, in patients eligible for curative intent ASCT. It is anticipated that continued epigenetic priming peri-ASCT with CC-486 can favorably alter disease biology for AML patients in CR1, modulate immune surveillance to decrease relapse risk, mitigate adverse graft versus host disease (GVHD) biology without significant compromise on the quality of life. This pilot study will assess the suitability of CC-486 as a bridge to transplant, will help optimize the timing between CC-486 administration and the start of transplant preparative regimen, and will assess the utility of continued CC-486 maintenance post-transplant.

Conditions: Acute Myeloid Leukemia
Being tested: oral azacitidine (CC-486)
Keywords: Azacitidine, Reduced Intensity Allogeneic Stem Cell Transplant, Epigenetic priming, Complete remission
Medical subject headings: Leukemia, Myeloid, Acute, Pathologic Complete Response

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

oral azacitidine (CC-486)Drug
Also called Onureg
Oral azacitidine is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.

Study groups in NCT06180863
Study group	Role	What they receive
Oral azacitidine (CC-486)	Experimental	Patients on study will receive 300 mg CC-486 for 14 out of 28 days per cycle with dose modification according to the Onureg United States Prescribing Information (USPI). Up to 3 cycles of CC-486 will be allowed, and a minimum of 1 cycle is to be given prior to transplant. Multiple cycles will be allowed only if donor-specific or logistical issues prevent transplantation after the first cycle. CC-486 will be dispensed on day 1 of each treatment cycle and only 1 cycle equivalent is to be dispensed at the beginning of each cycle. Post-transplant maintenance with CC-486 can be started 60 days (range 60-120 days) after hematopoietic progenitor cell infusion which should allow time for count and graft recovery after post-transplant cyclophosphamide (PTCY). The CC-486 maximum preferred pre-emptive dose post-ASCT is 200 mg daily, which will be administered for 14 of 28 days per cycle (max 12 cycles). Receives: Drug: oral azacitidine (CC-486)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of patients receiving oral azacitidine (CC 486) as post-remission consolidation therapy that are able to proceed to reduced intensity conditioning (RIC) allogeneic stem cell transplant (ASCT)
To identify the number of participants able to receive a minimum of 1 and a maximum of 3 cycles of CC-486 consolidation therapy as a bridge to Reduced Intensity Conditioning (RIC) ASCT while maintaining first complete remission- CR1.
Measured: With each cycle (1 cycle is equivalent to 28 days) of CC-486 pre-transplant, up to 3 months
Total number of participants that experience any adverse events with oral azacitidine (CC 486) use prior to RIC ASCT for patients with (AML) in first complete remission (CR1).
Adverse events (AEs) occurring in the participants will be graded according to NCI CTCAE v 5.0 with each cycle of CC_486 administered pre-transplant.
Measured: For a maximum of 3 cycles (1 cycle is equivalent to 28 days) and or up to 3 months from the start of CC-486

Secondary outcomes (8)

Disease status and response to each cycle of pre-transplant CC-486 administration as per European Leukemia Net (ELN) AML 2017 guidelines
With CC-486 consolidation prior to transplant best response to treatment and disease status will be measured. Response criteria or loss of response (including frank morphologic relapse) will be assessed by using peripheral blood or bone marrow samples as per European Leukemia Net (ELN) AML 2017 guidelines.
Measured: With each cycle (1 cycle is equivalent to 28 days) of CC-486 pre-transplant
Number of participants retaining remission post-transplant
Bone marrow biopsies will be performed longitudinally post-transplant. Prior to initiating first cycle of CC-486 maintenance post-transplant, disease status confirmation with a bone marrow biopsy in the prior 14 days would be needed. Response criteria or loss of response will be defined as per European Leukemia Net (ELN) AML 2017 guideline. Based on best response observed duration of remission, cumulative incidence of relapse, patterns of relapse will be described. Participants will be assessed at days 30, 60, 120, 180, 365 and 720 for disease relapse.
Measured: Up to 720 days
Total number of maintenance cycles administered post-transplant to assess compliance
As assessed by the number of cycles administered and the periods of interruptions.
Measured: Up to 2 years post transplant
Overall Survival
As assessed by the length of survival in months from the time of 1st cycle of CC-486 pretransplant, from the time of transplant and from the time of starting maintenance post-transplant.
Measured: Up to 2 years from the time of starting CC-486
Incidence of graft versus host disease (GVHD)
As assessed by cumulative incidence of acute and chronic graft versus host disease (GVHD) within 2 years from the date of transplant.
Measured: up to 2 years post-transplant
Hematopoiesis origins assessed in peripheral blood and bone marrow.
As assessed by kinetics of chimerism changes for those receiving CC-486. Based on institutional practices peripheral blood and or bone marrow samples would be used for chimerism testing. Donor/recipient hematopoiesis fraction will be reported across cellular subset per institutional standards.
Measured: Up to 2 years post transplant
Change in quality of life assessed by Foundation for the Accreditation of Cellular Therapy Bone Marrow Transplant (FACT-BMT) version 4.
Quality of life questionnaire administered pre- and post-transplant with CC-486 maintenance use. The Likert scale from zero to four [0-4] is used to measure the responses for each question after taking into account reverse scores for questions constructed in a negative form. The final score for FACT-BMT ranges from zero to 196. Higher scores for the scales and subscales indicate better quality of life.
Measured: Prior to transplant (at screening and immediately prior to transplant) and in post-transplant at days + 100, +180 & +365
Total number of participants that experience any adverse events with oral azacitidine (CC 486) use in post-transplant period.
Adverse events (AEs) occurring in the participants will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v 5.0 with each cycle of CC_486 administered post-transplant.
Measured: up to 2 years post transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Nov 2023Apr 2029

First posted26 Dec 2023
Study startNov 2023 (estimated)
Primary completionNov 2028 (estimated)
Study completionApr 2029 (estimated)

Why it stopped: Lack of funding.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
More trials run by Yale UniversityThe 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: A single-arm non-randomized pilot study
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06180863
Sponsor's protocol ID: 2000031065
Other IDs: 1T32CA233414-01A1
Status last verified: Dec 2023
First submitted: 17 Nov 2023
Last updated: 26 Dec 2023
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Acute Myeloid Leukemia trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Pilot Study of Oral Azacitidine (CC-486) Epigenetic Priming and Maintenance in Adult AML Patients Eligible for Reduced Intensity Allogeneic Stem Cell Transplant
ClinicalTrials.gov ID: NCT06180863
Acronym / sponsor ID: 2000031065
Conditions: Acute Myeloid Leukemia
Keywords: Azacitidine, Reduced Intensity Allogeneic Stem Cell Transplant, Epigenetic priming, Complete remission
Phase: Phase 2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Lack of funding.
Enrolment: 0 participants (actual)
Lead sponsor: Yale UniversityUniversity, hospital or other
Collaborators: Bristol-Myers Squibb, National Cancer Institute (NCI)
Study start: Nov 2023 (estimated)
Primary completion: Nov 2028 (estimated)
Study completion: Apr 2029 (estimated)
First posted: 26 Dec 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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: A single-arm non-randomized pilot study

Arm	Type	Receives
Oral azacitidine (CC-486)	Experimental	Drug: oral azacitidine (CC-486)Patients on study will receive 300 mg CC-486 for 14 out of 28 days per cycle with dose modification according to the Onureg United States Prescribing Information (USPI). Up to 3 cycles of CC-486 will be allowed, and a minimum of 1 cycle is to be given prior to transplant. Multiple cycles will be allowed only if donor-specific or logistical issues prevent transplantation after the first cycle. CC-486 will be dispensed on day 1 of each treatment cycle and only 1 cycle equivalent is to be dispensed at the beginning of each cycle. Post-transplant maintenance with CC-486 can be started 60 days (range 60-120 days) after hematopoietic progenitor cell infusion which should allow time for count and graft recovery after post-transplant cyclophosphamide (PTCY). The CC-486 maximum preferred pre-emptive dose post-ASCT is 200 mg daily, which will be administered for 14 of 28 days per cycle (max 12 cycles).

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (13 listed)

Patients must have acute myeloid leukemia (AML) of Intermediate or High risk (IR or HR per National Comprehensive Cancer [NCCN] Criteria) in first CR/CRi achieved with 1 or 2 cycles of cytarabine based induction therapy. Zero to 1 cycle of cytarabine based consolidation will be allowed post CR1. OR Nucleophosmin 1(NPM 1) positive AML ((in the absence of FMS like tyrosine kinase 3 (FLT3) mutation)) at diagnosis with positive NPM1 PCR (measurable residual disease [MRD]) after at least 1 cycle of cytarabine-based consolidation chemotherapy OR Secondary AML(s-AML) based on prior MDS or CMML in first CR/CRi. OR Therapy-related AML (t-AML) with IR or HR criteria
Vyxeos induction (up to 2 cycles to reach CR1) and 0-1 cycle of vyxeos consolidation will be allowed.
Eligible to receive RIC or Non myeloablative (NMA) transplant preparative regimen with post-transplant cyclophosphamide (PTCY) based GVHD prophylaxis, at the discretion of study investigator.
Patients must have a calculated creatinine clearance (Cockcroft-Gault equation) > 50 mL/min.
Patients must have a related or unrelated donor. Sibling donor must be a 6/6 match for human leukocyte antigen A (HLA-A) and HLA-B at intermediate or higher resolution, and DRB1 at high resolution using DNA-based typing; must be willing to donate peripheral blood stem cells; and meet institutional criteria for donation. Unrelated donor must be a 7/8 or 8/8 match at HLA-A, -B, -C, and -DRB1 at high resolution using DNA-based typing; must be willing to donate peripheral blood mononuclear cells (PBSCs); and be medically eligible to donate stem cells according to National Marrow Donor Program (NMDP) criteria.

Key exclusion criteria (27 listed)

Patients may not have Acute Promyelocytic leukemia
Patients may not have favorable risk AML per National Comprehensive Cancer Network (NCCN) guidelines ((Core binding factor leukemias, CCAAT/enhancer-binding protein-alpha (CEBPA) double mutant, AML with concomitant mutations in Nucleophosmin 1 (NPM1) and FLT3))
Patients must not have received more than two rounds of chemotherapy to achieve first CR or CRi
Patients may not have had exposure to hypomethylating agent (HMA) to treat the leukemia or prior hematologic malignancy ((i.e., myelodysplastic syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML)) in the 6 months prior to study enrollment.
Patients may not be, per investigator evaluation, currently in need of other leukemia-directed therapy such as FLT3- or Isocitrate dehydrogenase (IDH)-directed therapy.

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
oral azacitidine (CC-486)Also: OnuregOral azacitidine is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.	Drug	Oral azacitidine (CC-486)

Primary outcome

Number of patients receiving oral azacitidine (CC 486) as post-remission consolidation therapy that are able to proceed to reduced intensity conditioning (RIC) allogeneic stem cell transplant (ASCT)
Time frame: With each cycle (1 cycle is equivalent to 28 days) of CC-486 pre-transplant, up to 3 months
To identify the number of participants able to receive a minimum of 1 and a maximum of 3 cycles of CC-486 consolidation therapy as a bridge to Reduced Intensity Conditioning (RIC) ASCT while maintaining first complete remission- CR1.
Total number of participants that experience any adverse events with oral azacitidine (CC 486) use prior to RIC ASCT for patients with (AML) in first complete remission (CR1).
Time frame: For a maximum of 3 cycles (1 cycle is equivalent to 28 days) and or up to 3 months from the start of CC-486
Adverse events (AEs) occurring in the participants will be graded according to NCI CTCAE v 5.0 with each cycle of CC_486 administered pre-transplant.

8 secondary outcomes

Disease status and response to each cycle of pre-transplant CC-486 administration as per European Leukemia Net (ELN) AML 2017 guidelines
Time frame: With each cycle (1 cycle is equivalent to 28 days) of CC-486 pre-transplant
Number of participants retaining remission post-transplant
Time frame: Up to 720 days
Total number of maintenance cycles administered post-transplant to assess compliance
Time frame: Up to 2 years post transplant
Overall Survival
Time frame: Up to 2 years from the time of starting CC-486
Incidence of graft versus host disease (GVHD)
Time frame: up to 2 years post-transplant
Hematopoiesis origins assessed in peripheral blood and bone marrow.
Time frame: Up to 2 years post transplant
Change in quality of life assessed by Foundation for the Accreditation of Cellular Therapy Bone Marrow Transplant (FACT-BMT) version 4.
Time frame: Prior to transplant (at screening and immediately prior to transplant) and in post-transplant at days + 100, +180 & +365
Total number of participants that experience any adverse events with oral azacitidine (CC 486) use in post-transplant period.
Time frame: up to 2 years post transplant

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Dec 2023.
Individual participant dataSharing planned: No

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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.

Lohith Gowda, MD principal investigator

Yale University

Marcos de Lima, MD principal investigator

Ohio State University

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 oral azacitidine (CC-486) in people with Acute Myeloid Leukemia, run by Yale University 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 13 inclusion and 27 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: withdrawn, as of the sponsor's last update on 26 Dec 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 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?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients receiving oral azacitidine (CC 486) as post-remission consolidation therapy that are able to proceed to reduced intensity conditioning (RIC) allogeneic stem cell transplant (ASCT) (time frame: With each cycle (1 cycle is equivalent to 28 days) of CC-486 pre-transplant, up to 3 months). 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?

On the ClinicalTrials.gov record for NCT06180863.

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 Dec 2023.