Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Cytarabine

Aclacinomycins

Antimetabolites

Antiviral Agents

Immunosuppressive Agents

Immunologic Factors

Adjuvants, Immunologic

Anti-Bacterial Agents

Antibiotics, Antitubercular

Aclacinomycin 20mg/d for 4 days combined with cytarabine 20mg bid for 10 days with/without G-CSF 6ug/m2 from 1 day before therapy to day 10 of therapy.

CAG regimen (Aclacinomycin, cytarabine, with/w/o G-CSF)

low dose cytarabine

Jianxiang Wang, MD

Chunlin Zhou, MD

This study focus on the comparison of CAG regimen to the low dose cytarabine therapy in elderly AML patients who are unfit or unwilling to receive intensive chemotherapy.

Low dose cytarabine remains to be the choice of standard for elderly AML patients who are unfit or unwilling to receive intensive treatment. CAG regimen, which is a combination of aclacinomycin，low dose cytarabine±G-CSF is used often in elderly AML patients in China. It is proved effective and safety in some pilot studies，while there is no prospective，randomized study yet.

Treatment of Elderly Chinese Acute Myeloid Leukemia Patients Aged 65 to 75 Years Old

A Multicenter，Open-label，Randomized Study on the Treatment of Elderly Chinese Acute Myeloid Leukemia Patients Aged 65 to 75 Years Old

Jianxiang Wang

vice-president

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Precancerous Conditions

Clofarabine

Clofarabine 10 mg/m2 by a 1-hr i.v infusion, once daily, from day 1 to 5. Ara-C 10 mg/m2, subcutaneously, twice a day (q12 hrs), from day 1 to 15. Clofarabine will be given 4 hours before the 2nd dose of Ara-C.

Clofarabine plus low dose Ara-C

Felicetto Ferrara, MD

This is an interventional, multicenter, open label, phase II study designed to evaluate the safety and efficacy of Clofarabine in combination with low dose Cytarabine in untreated patients with poor risk of Myelodisplastic Syndromes.

Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes

Phase II Study of Clofarabine in Combination With Low Dose Cytarabine for Untreated Patients With Poor Risk Myelodysplastic Syndromes

Fondazione Italiana Sindromi Mielodisplastiche-ETS

Azacitidine

Enzyme Inhibitors

Azacitidine daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

75 mg/m\^2 subcutaneous (SC) daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

Physician pre-selects prior to randomization from one of the following:

* Intensive chemotherapy (cytarabine 100-200 mg/m\^2 continuous intravenous infusion for 7 days + anthracycline IV x 3 days) + Best Supportive Care; induction with up to 2 consolidation cycles
* Low-dose cytarabine 20 mg subcutaneous (SC) twice a day (BID) for 10 days, for 28 day cycles + BSC; until disease progression or unacceptable toxicity
* Best Supportive Care only; until study end

Conventional Care Regimen

C L Beach, PharmD

The purpose of this study is to compare the effect of azacitidine (Vidaza) to conventional care regimens on overall survival in elderly AML patients.

Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)

A Phase 3, Multicenter, Randomized, Open-Label, Study of Azacitidine (Vidaza®) Versus Conventional Care Regimens for the Treatment of Older Subjects With Newly Diagnosed Acute Myeloid Leukemia

Overall Survival was defined as the time from randomization to death from any cause. Overall survival was calculated by the formula: date of death - date of randomization + 1. Participants surviving at the end of the follow-up period or who withdrew consent to follow-up were censored at the date of last contact. Participants who were lost to follow-up were censored at the date last known alive.

Kaplan Meier methods were used to estimate the 1-year survival probabilities for time to death from any cause. Estimates of the 1-year (365 day) survival probabilities and corresponding 95% confidence intervals (CI) were presented by treatment group. The CI for the difference in the 1-year survival probabilities was derived using Greenwoods variance estimate.

Event-free survival was defined as the interval from the date of randomization to the date of treatment failure, progressive disease, relapse after complete remission (CR) or complete remission with incomplete blood count recovery (CRi), death from any cause, or lost to follow-up, whichever occurs first. Participants who were still alive without any of these events were censored at the date of their last response assessment.

Relapse-free survival was defined as the interval from the date of first documented CR or CRi to the date of relapse, death from any cause, or lost to follow-up, whichever occurred first. Participants who were still alive and in continuous CR or CRi were censored at the date of their last response assessment.

A complete remission (CR) is defined as a leukemia-free state defined as less than 5% blasts in a BM aspirate with marrow spicules and with at least 200 nucleated cells (there should be no blasts with Auer rods), an absolute neutrophil count (ANC) of ≥ 1 x 10\^9/L, a platelet count ≥ 100 x 10\^9/L, and transfusion independence (no transfusions for 1 week prior to each assessment). No duration of these findings is required for confirmation of this response. A CR with incomplete blood count recovery (CRi) is defined as \<5% BM blasts with the ANC count \< 1 x 10\^9/L and/or the platelet count may be \< 100 x 10\^9/L. Where the date of the hematology assessment used is the earliest on or following the date of the BM sample up to 8 days after the BM date.

The time from the date CR or CRi was first documented until the date of documented relapse from CR/CRi. Duration of remission was defined only for those participants who achieved a CR or CRi, as determined by the IRC. Participants who were lost to follow-up without documented relapse, or were alive at last follow-up without documented relapse were censored at the date of their last response assessment.

The CRc is a normal karyotype defined as no clonal abnormalities after review of at least 10 metaphases using conventional cytogenetic techniques. Cytogenetic complete remission rate (CRc) is when the following criteria are met: 1) CR criteria met and 2) an abnormal karyotype is present at baseline and 3) there is reversion to normal karyotype at the time of CR (based on ≥ 10 metaphases), where date of cytogenetic sample = date of BM sample used for the CR assessment

AEs = any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the participant's health, regardless of cause. Serious AE (SAE) = any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. The severity of AEs were graded based upon the participants symptoms according to the Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0); AEs were evaluated for severity according to the following scale: Grade 1 = Mild - transient or mild discomfort; no medical intervention required; Grade 2 = Moderate - mild to moderate limitation in activity; Grade 3 = Severe; Grade 4 = Life threatening; Grade 5 = Death

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.

HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective.

HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective. The rate of inpatient hospitalizations per patient year was calculated as the total number of hospitalizations divided by the total number of patient-years followed in the study period. Patient-years (PY) were calculated as the duration from baseline to last available HRQL assessment for each patient.

Count of study participants who had transfusions during the treatment phase. HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective.

HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective. The rate of transfusions per patient year was calculated as the total number of transfusions divided by the total number of patient-years followed in the study period. Patient-years (PY) were calculated as the duration from baseline to last available HRQL assessment for each patient.

Celgene

3) discontinuation for BSC only. Median duration was 191.5, 65.0, 125.0, 124.5 and 360.0 days for each group respectively.

AEs reported for the Azacitidine group are those that occurred in the treatment phase, AEs reported in the Azacitidine extension group occurred during extension phase

Azacitidine 75 mg/m\^2/day by subcutaneous injection \[SC\] for 7 days every 28 days, with a 21 day rest period (optimally for at least 6 cycles) plus best supportive care as needed, including antibiotics and blood product transfusions, growth factors, per physician's discretion

Transfusion of blood products, antibiotics, antifungals and nutritional help

BSC Only

Low-dose cytarabine 20 mg subcutaneously twice a day (BID) for 10 days every 28 days, plus BSC

Low-dose Cytarabine

Cytarabine 100-200 mg/m\^2 as a continuous intravenous infusion (CIVI) for 7 days and daunorubicin 45 to 60 mg/m\^² daily (QD) IV on Days 1, 2 and 3 or Idarubicin 9-12 mg/m\^² IV QD for 3 days. Consolidation Therapy (Cycle 2 and 3) = Cytarabine 100-200 mg/m\^2 as a CIVI for 3 to 7 days and daunorubicin 45 to 60 mg/m\^² QD or Idarubicin 9-12 mg/m\^² IV QD on Days 1 and 2. The first consolidation therapy started between Day 28 and Day 70 from start of induction therapy, upon recovery of absolute neutrophil count (ANC) above 1.0 x 10\^9/L and platelets above 75 x 10\^9/L. The second cycle started between Day 28 and Day 70 from start of first consolidation therapy. Best supportive care (BSC) of antibiotics and transfusions, were given as needed

Intensive Chemotherapy

Azacitidine 75 mg/m\^2/day by subcutaneous injection \[SC\] for 7 days every 28 days, with a 21 day rest period (optimally for at least 6 cycles) plus best supportive care as needed, including antibiotics and blood product transfusions, growth factors, per physician's discretion.

Azacitidine-extension

Azacitidine 75 mg/m2/day by subcutaneous injection \[SC\] for 7 days every 28 days, with a 21 day rest period (optimally for at least 6 cycles) plus best supportive care as needed, including antibiotics and blood product transfusions, growth factors, per physician's discretion.

Azacitidine (AZA)

#1 Intensive Chemotherapy: Cytarabine 100-200 mg/m\^2 as a continuous intravenous infusion (CIVI) for 7 days and daunorubicin 45 to 60 mg/m\^² daily (QD) IV on Days 1, 2 and 3 or Idarubicin 9-12 mg/m\^² IV QD for 3 days. Consolidation Therapy (Cycle 2 and 3) = Cytarabine 100-200 mg/m\^2 as a CIVI for 3 to 7 days and daunorubicin 45 to 60 mg/m\^² QD or Idarubicin 9-12 mg/m\^² IV QD on Days 1 and 2. Consolidation therapy started between Day 28 and Day 70 from start of induction therapy, upon recovery of absolute neutrophil count (ANC) above 1.0 x 10\^9/L and platelets above 75 x 10\^9/L. The second cycle started between Day 28 and Day 70 from start of first consolidation therapy. Best supportive care (BSC) of antibiotics and transfusions, were given as needed. # 2 Low-dose cytarabine 20 mg SC twice a day (BID) for 10 days every 28 days, plus BSC # 3 BSC only includes transfusion of blood products, antibiotics, antifungals and nutritional help.

Conventional Care Regimens (CCR)

Total

Age, Continuous

<75 years

≥75 years

Age, Customized

Sex: Female, Male

0 = Fully Active

1 = Restrictive but Ambulatory

2 = Ambulatory but unable to work

3 = Limited Self Care

4 = Completely disabled

ECOG performance status is used by doctors and researchers to assess how a participants disease is progressing, assess how the disease affects the daily living activities of the participant and determine appropriate treatment and prognosis. 0 = Fully Active (Most Favorable Activity), 1 = Restricted activity but ambulatory, 2 = Ambulatory but unable to carry out work activities, 3 = Limited Self Care; 4 = Completely Disabled, No self care (Least Favorable Activity)

Eastern Cooperative Oncology Group (ECOG) Performance Status

AML with myelodysplasia-related changes

Therapy-related myeloid neoplasms

AML with recurrent genetic abnormalities

AML not otherwise specified

WHO categories include:

1. Those with AML with recurrent genetic abnormalities. Includes subtypes with multiple chromosome translocations and mutations.
2. Those with AML with myelodysplasia-related changes. Includes those with prior myelodysplastic syndrome (MDS) or myeloproliferative disease and has transformed to AML.
3. Those with therapy related myeloid neoplasms and have had prior chemotherapy and/or radiation and subsequently develop AML or MDS
4. Those with AML not otherwise specified and include subtypes that do not fall into the above categories.

World Health Organization Acute Myeloid Leukemia (AML) Classification

Baseline clinical characteristics, including percentage of bone marrow blasts were assessed centrally and locally. Central values are included here. Baseline blasts were the last non-missing value on or prior to the date of randomization. The bone marrow blasts cells are not typically found in the circulating blood of healthy individuals. Abnormal immature white blood cells (blasts) fill the bone marrow and spill into the bloodstream.

Bone Marrow-Blasts Counts

The intent-to-treat (ITT) population is defined as all participants who were randomized, independent of whether they received study treatment or not.

Anne McClain, Senior Manager Clinical Trial Disclosure

CCRs included: #1 Intensive Chemotherapy: Cytarabine 100-200 mg/m\^2 as a continuous intravenous infusion (CIVI) for 7 days and daunorubicin 45 to 60 mg/m\^² daily (QD) IV on Days 1, 2 and 3 or Idarubicin 9-12 mg/m\^² IV QD for 3 days. Consolidation Therapy (Cycle 2 and 3) = Cytarabine 100-200 mg/m\^2 as a CIVI for 3 to 7 days and daunorubicin 45 to 60 mg/m\^² QD or Idarubicin 9-12 mg/m\^² IV QD on Days 1 and 2. The first consolidation therapy started between Day 28 and Day 70 from start of induction therapy, upon recovery of absolute neutrophil count (ANC) above 1.0 x 10\^9/L and platelets above 75 x 10\^9/L. The second cycle started between Day 28 and Day 70 from start of first consolidation therapy. Best supportive care (BSC) of antibiotics and transfusions, were given as needed # 2 Low-dose cytarabine 20 mg subcutaneously twice a day (BID) for 10 days every 28 days, plus BSC # 3 BSC only = transfusion of blood products, antibiotics, antifungals and nutritional help

The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored

Kaplan-Meier Estimates for Overall Survival

One-year Overall Survival Rate

Median is estimated from a Kaplan-Meier distribution of EFS

Event-free Survival (EFS)

Median is estimated from a Kaplan-Meier distribution of RFS

Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi)

Percentage of Participants Who Achieved a Morphologic CR + CRi as Determined by the Independent Review Committee (IRC) Based on International Working Group (IWG) Response Criteria for Acute Myeloid Leukemia (AML)

Includes those who achieved a CR or CRi and assessed by the IRC; numbers of ITT participants in each treatment group

Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates

Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC.

At least one Treatment Emergent AE

At least one TEAE related to study drug

Grade 3-4 adverse event

Grade 3-4 adverse event related to any study drug

At least one Grade 5 (leading to death) TEAE

Grade 5 adverse event related to any study drug

Serious TEAE

Serious TEAE related to any study drug

TEAE leading to discontinuation of study drug

Study drug-related TEAE leading to discontinuation

TEAE leading to study drug dose reduction

TEAE leading to study drug dose interruption

TEAE causing study drug dose reduction/disruption

Best supportive care included red blood cell (RBC) or whole blood transfusions, fresh frozen plasma transfusions, platelet transfusions, antibiotic and/or antifungal therapy, and nutritional support.

Conventional Care Regimen #3 Best Supportive Care Only

Low-dose cytarabine 20 mg SC injection twice a day (BID) for 10 days, every 28 days (optimally for at least 4 cycles) until the end of the study, unless participants were discontinued from the treatment. Best supportive care as needed, including antibiotics and transfusions, per physicians discretion.

Conventional Care Regimen #2 Low-dose Cytarabine

Induction Therapy included Cytarabine 100-200 mg/m\^2 as a continuous intravenous infusion (IV) for 7 days plus daunorubicin 45 to 60 mg/m\^² daily IV on Days 1, 2 and 3 or Idarubicin 9-12 mg/m\^² IV daily for 3 days as an alternative to daunorubicin (Cycle 1). Consolidation Therapy (Cycle 2 and 3) Cytarabine 100-200 mg/m\^2 as a continuous IV infusion for a total of 3 to 7 days plus daunorubicin 45 to 60 mg/m\^² daily or Idarubicin 9-12 mg/m\^² IV daily on Days 1 and 2. The first consolidation therapy started between Day 28 and Day 70 from commencement of induction therapy, upon recovery of absolute neutrophil count (ANC) to at least 1.0 x 10\^9/L and platelets to at least 75 x 10\^9/L. The second consolidation cycle, if given, started between Day 28 and Day 70 from commencement of the first consolidation therapy. Participants could receive BSC as needed, including antibiotics and transfusions, physicians discretion.

Conventional Care Regimen #1 Intensive Chemotherapy

Safety population = all randomized participants who received at least 1 dose of study drug and had 1 post-dose safety assessment. Because the BSC only regimen consisted of blood products or antibiotics given as needed, those assigned to BSC only were included in the safety population if they had at least 1 post-randomization safety assessment.

Number of Participants With Adverse Events (AEs)

The HRQoL Evaluable population included only participants with a baseline QoL assessment and at least 1 follow-up assessment. Time windows were applied post-hoc to increase the size of the analyzable population.

Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain

Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain

The HRQoL Evaluable population included only participants with a baseline QoL assessment and at least 1 follow-up assessment. Time windows were applied post-hoc to increase the size of the analyzable population. .

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

The HRQoL Evaluable population included only participants with a baseline QoL assessment and at least 1 follow-up assessment. Time windows were applied post-hoc to increase the size of the analyzable population. The analysis included 157 from the azacitidine group and 134 in the CCR group, a smaller number than the ITT population.

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

HRU was analyzed for the HRQoL Evaluable Population, a smaller sample than either the ITT population or safety population. Duration of therapy differed between treatment groups. Rate-per-patient year values adjust for these differences.

Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations

Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Per Year

HRU: Number of Participants Receiving Transfusions

HRU: Rate of Transfusions Per Patient Year

At least one Grade 3-4 adverse event

At least 1 Grade 3-4 TEAE related to study drug

At least 1 Grade 5 TEAE

At least 1 Grade 5 TEAE related to study drug

At least 1 serious TEAE

At least 1 serious TEAE related to study drug

At least one serious Grade 3-4 TEAE

TEAE leading to study drug dose interruption only

TEAE causing study dose reduction/interruption

Safety population includes those enrolled in the extension phase who received at least one dose of study drug.

Number of Participants in the Extension Phase With Treatment Emergent Adverse Events (TEAEs)

CCRs included:

* 1 Intensive Chemotherapy: Cytarabine 100-200 mg/m2 as a continuous intravenous infusion (CIVI) for 7 days and daunorubicin 45 to 60 mg/m² daily (QD) IV on Days 1, 2 and 3 or Idarubicin 9-12 mg/m² IV QD for 3 days. Consolidation Therapy (Cycle 2 and 3) = Cytarabine 100-200 mg/m2 as a CIVI for 3 to 7 days and daunorubicin 45 to 60 mg/m² QD or Idarubicin 9-12 mg/m² IV QD on Days 1 and 2. The first consolidation therapy started between Day 28 and Day 70 from start of induction therapy, upon recovery of absolute neutrophil count (ANC) above 1.0 x 10\^9/L and platelets above 75 x 10\^9/L. The second cycle started between Day 28 and Day 70 from start of first consolidation therapy. Best supportive care (BSC) of antibiotics and transfusions, were given as needed
* 2 Low-dose cytarabine 20 mg subcutaneously twice a day (BID) for 10 days every 28 days, plus BSC
* 3 BSC only = transfusion of blood products, antibiotics, antifungals and nutritional help

Treatment Phase

Survival Follow-Up Phase

Extension Phase

Hematologic Neoplasms

Leukemia, Myelomonocytic, Chronic

Leukemia, Myelomonocytic, Juvenile

Leukemia, Myelomonocytic, Acute

Myeloproliferative Disorders

Myelodysplastic-Myeloproliferative Diseases

Chronic Disease

Disease Attributes

Chronic Graft Versus Host Disease

Chronic Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia

Chronic Myeloproliferative Disorders

Myelodysplastic/myeloproliferative Disease

Decitabine

FHD-286 administered orally + LDAC administered subcutaneously

FHD-286 administered orally + decitabine administered intravenously (IV)

FHD-286

Low Dose Cytarabine

Foghorn Clinical Trials

Brian Ball, MD

Richard Stone, MD

Eytan Stein, MD

Michael Savona, MD

Courtney DiNardo, MD

Sarah Reilly, MD

This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as monotherapy or combination therapy, in subjects with advanced hematologic malignancies.

This study is primarily intended to evaluate the safety and tolerability of FHD-286 when administered orally as monotherapy or in combination with either LDAC or decitabine to subjects with R/R AML, R/R MDS, and R/R CMML not in blast crisis. In each arm of the study, successive cohorts of participants will receive increasing oral doses of FHD-286 as a single agent or in combination with LDAC or decitabine to determine the RP2D(s) in this population.

The data from this study in subjects with advanced hematologic malignancies, including safety, tolerability, PK/PD findings, and antitumor activity, will form the basis for subsequent clinical development of FHD-286.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Treatment of Elderly Chinese Acute Myeloid Leukemia Patients Aged 65 to 75 Years Old	NCT02432911	Acute Myeloid L...	CAG regimen (Ac... low dose cytara...	65 Years - 75 Years	ChineseAMS
Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes	NCT01302106	Myelodysplastic...	Clofarabine plu...	55 Years - 80 Years	Fondazione Italiana Sindromi Mielodisplastiche-ETS
Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)	NCT01074047	Acute Myeloid L...	Azacitidine Conventional Ca...	65 Years -	Celgene
Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes	NCT01302106	Myelodysplastic...	Clofarabine plu...	55 Years - 80 Years	Fondazione Italiana Sindromi Mielodisplastiche-ETS
FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies	NCT04891757	Advanced Hemato... Relapsed Acute ... Refractory Acut... Relapsed Myelod... Refractory Myel... Relapsed Chroni... Refractory Chro...	FHD-286 Low Dose Cytara... Decitabine	16 Years -	Foghorn Therapeutics Inc.
Bortezomib and Low Dose Cytarabine in the Treatment of High-Risk Myelodysplastic Syndromes	NCT00411905	Myelodysplastic...	Bortezomib	18 Years -	Groupe Francophone des Myelodysplasies
Treatment of Elderly Chinese Acute Myeloid Leukemia Patients Aged 65 to 75 Years Old	NCT02432911	Acute Myeloid L...	CAG regimen (Ac... low dose cytara...	65 Years - 75 Years	ChineseAMS

Spots Global Cancer Trial Database for low dose cytarabine