Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies

A Phase 1/2a, Dose Escalation Study of FF-10501-01 for the Treatment of Advanced Hematologic Malignancies

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02193958

Enrollment

Registered

2014-07-18

Start date

2014-07-31

Completion date

2019-10-15

Last updated

2025-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

AML, CMML, MDS

Keywords

Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome

Brief summary

A Phase 1/2a Dose Escalation Study of FF-10501-01 in Patients with Relapsed or Refractory Hematological Malignancies to determine the safety and tolerability. A total of 6 cohorts will be enrolled in Phase 1 to establish the MTD. A total of 20 subjects with MDS/CMML treated at the RP2D are planned, including MDS/CMML subjects treated at the RP2D in Phase 1.

Detailed description

Subjects will receive FF-10501-01 orally on a twice daily schedule for 14, 21 or 28 days repeated every 28 days (=1 cycle). Disease assessments, including analysis of blood and bone marrow aspirates, will be performed at the end of Cycle 1 and every 2 cycles thereafter. Subjects who demonstrate objective response or stable disease will be allowed to continue therapy with FF-10501-01 until progression of disease, observation of unacceptable adverse events, intercurrent illness or changes in condition that prevent further study participation.

Interventions

DRUGFF-10501-01

FF-10501-01 will be administered orally on Days 1-14 of a 28-day cycle (Cohorts 1-6), Days 1-21 of a 28-say cycle (Cohort 7), Days 1-28 of a 28-day cycle (Cohort 8) or Days 1-21 of a 28-say cycle (Cohort 9). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Confirmed advanced hematologic malignancies; Phase 1: * High-risk MDS/CMML (defined as ≥ 10% peripheral blood or marrow blasts and/or IPSS score ≥ 1.5) and relapsed or refractory to prior therapy * AML relapsed or refractory to prior therapy, or ≥ 60 years of age and not a candidate for other therapies Phase 2a: * MDS/CMML, relapsed from, or refractory to, prior HMA therapy; the latter defined as failure to achieve clinical remission (CR), partial remission (PR) or hematologic improvement (HI) after previous HMA therapy (≥ 4 cycles of azacitidine or decitabine), or progression during, or toxicity to previous HMA therapy precluding further HMA treatment, and, * Bone marrow blast count ≥ 10% or peripheral blast count ≥ 5%, or IPSS-R score ≥ 3.5. * At least 3 weeks beyond the last chemotherapy, targeted anticancer agent, major surgery or experimental treatment and recovered from all acute toxicities (≤ Grade 1). Hydroxyurea used to control peripheral blast counts is permitted up to Day 7 of treatment on study. * Adequate performance status: ECOG ≤ 2; * Adequate renal and hepatic function: * creatinine ≤ 2.0 mg/dL, or calculated creatinine clearance ≥ 45 mL/min * total bilirubin ≤ 2 times the upper limit of normal (ULN) * ALT/AST ≤ 2 times ULN * Negative serum pregnancy test * Ability to provide written informed consent

Exclusion criteria

* Known history of coronary artery disease, angina, myocardial infarction, congestive heart failure, cardiac arrhythmia or any other type of heart disease present within the last 6 months * Known family history of hereditary heart disease * QT interval corrected for rate (QTc) \> 450 msec on the electrocardiogram (ECG) obtained at Screening * Concomitant medication(s) that may cause QTc prolongation or induce Torsades de Pointes, with the exception of anti-microbials that are used as standard of care to prevent or treat infections and other such drugs that are considered by the Investigator to be essential for the care of the patient. * Presence of active central nervous system (CNS) leukemia. Subjects adequately treated for CNS leukemia documented by 2 consecutive cerebrospinal fluid samples negative for leukemia cells are eligible. Subjects with no history of CNS leukemia will not be required to undergo cerebrospinal fluid sampling for eligibility. * Known positive for HIV, hepatitis B virus surface antigen (HBsAg), or hepatitis C virus (HCV). * Active infection requiring IV anti-infective usage within the last 7 days prior to study treatment. * Any other medical intervention or condition which could compromise adherence to study requirements or confound the interpretation of study results. * Pregnant or breast-feeding. * Treatment with any investigational product within 28 days prior to Screening.

Design outcomes

Primary

Measure	Time frame	Description
Safety Assessed by Adverse Events	12 months	Safety and tolerability assessed by adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicity (DLT), dose reductions, delays or withdrawals due to toxicity

Secondary

Measure	Time frame	Description
Determination of Objective Response (OR) Rates.	OR responses were assessed at end of Cycles 1 thru 3. Each cycle was 28 days in length.	The OR endpoint: proportion of subjects w/ OR as best response (CR, CRi or PR) assessed at the end of Cycles 1 and 3. AML: CR - free of all symptoms related to leukemia, absolute neutrophil count \> 1.0 x 10\^9/L, platelet count ≥ 100 x 10\^9/L, normal bone marrow with \< 5% blasts no Auer rods; CRi - CR with residual thrombocytopenia (platelet count \< 100 x 10\^9/L) or residual neutropenia (absolute neutrophil count \< 1.0 x 10\^9/L); PR - A ≥ 50% decrease in bone marrow blasts to 5 to 25% abnormal. MDS or CMML: CR - free of all symptoms related to leukemia, absolute neutrophil count ≥ 1.0 x 10\^9/L, platelet count ≥ 100 x 10\^9/L, bone marrow ≤ 5% myeloblasts, w/ normal maturation of all cell lines, hemoglobin ≥ 11g/dL, no blasts in the peripheral blood; PR - All CR criteria with ≥50% decrease in bone marrow blasts over pre-treatment (but still \> 5%); Marrow CR - In bone marrow, ≤ 5% myeloblasts and decrease by ≥ 50% over pre-treatment.

Countries

United States

Participant flow

Recruitment details

Participants were recruited based on physician referral at 2 academic medical centers between July 2014 and December 2018. The first participant was enrolled on August 21, 2014 and the last participant was enrolled on December 3, 2018. Study completion date was August 9, 2019.

Pre-assignment details

For Phase 1, anticipated enrollment was 48 subjects; 38 subjects were enrolled and treated with FF-10501-01. For Phase 2, anticipated enrollment was 20 subjects; 17 were enrolled and treated with FF-10501-01.

Participants by arm

Arm	Count
Phase 1 Cohort 1: 50mg/m2 FF-10501-01 tablets BID for 14 days of a 28 day cycle. FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	3
Phase 1 Cohort 2: 100mg/m2 FF-10501-01 tablets BID for 14 days of a 28 day cycle. FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	3
Phase 1 Cohort 3: 200mg/m2 FF-10501-01 tablets BID for 14 days of a 28-day cycle. FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	4
Phase 1 Cohort 4: 300mg/m2 FF-10501-01 tablets BID for 14 days of a 28-day cycle. FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	4
Phase 1 Cohort 5: 400mg/m2 FF-10501-01 tablets BID for 14 days of a 28-day cycle FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	7
Phase 1 Cohort 6: 500mg/m2 FF-10501-01 tablets BID for 14 days of a 28-day cycle. FF-10501-01: FF-10501-01 will be administered orally on Days 1-14 of a 28-day cycle (Cohorts 1-6). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	5
Phase 1 Cohort 7: 400mg/m2 FF-10501-01 tablets BID every 21 days of a 28 day cycle. FF-10501-01: FF-10501-01 will be administered orally on Days 1-21 of a 28-say cycle (Cohort 7). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	5
Phase 1 Cohort 8: 400mg/m2 FF-10501-01 tablets BID for 28 days of a 28-day cycle FF-10501-01: FF-10501-01 will be administered orally BID on Days 1-28 of a 28-day cycle (Cohort 8). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	7
Phase 2a Cohort 9: 400mg/m2 FF-10501-01 tablets BID for 21 days of a 28-day cycle. FF-10501-01 will be administered orally BID on Days 1-21 of a 28-day cycle (Cohorts 9). The dose-escalation will proceed until Maximum Tolerated Dose (MTD) is reached. The treatment will continue until disease progression, intolerable toxicity or investigation/subject decision.	17
Total	55

Baseline characteristics

Characteristic	Phase 1 Cohort 2: 100mg/m2	Phase 1 Cohort 3: 200mg/m2	Phase 1 Cohort 4: 300mg/m2	Phase 1 Cohort 5: 400mg/m2	Phase 1 Cohort 6: 500mg/m2	Phase 1 Cohort 7: 400mg/m2	Phase 1 Cohort 8: 400mg/m2	Phase 1 Cohort 1: 50mg/m2	Phase 2a Cohort 9: 400mg/m2	Total
Age, Categorical <=18 years	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Age, Categorical >=65 years	2 Participants	1 Participants	4 Participants	7 Participants	4 Participants	5 Participants	5 Participants	3 Participants	16 Participants	47 Participants
Age, Categorical Between 18 and 65 years	1 Participants	3 Participants	0 Participants	0 Participants	1 Participants	0 Participants	2 Participants	0 Participants	1 Participants	8 Participants
Age, Continuous	69.00 years STANDARD_DEVIATION 11.79	66.25 years STANDARD_DEVIATION 9.36	76.00 years STANDARD_DEVIATION 5.35	79.00 years STANDARD_DEVIATION 5.72	71.00 years STANDARD_DEVIATION 9.14	77.40 years STANDARD_DEVIATION 3.78	73.29 years STANDARD_DEVIATION 9.36	73.67 years STANDARD_DEVIATION 4.04	73.00 years STANDARD_DEVIATION 6.68	73.44 years STANDARD_DEVIATION 7.355
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants	0 Participants	1 Participants	3 Participants	5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants	3 Participants	3 Participants	2 Participants	4 Participants	3 Participants	7 Participants	2 Participants	14 Participants	41 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants	1 Participants	1 Participants	5 Participants	1 Participants	1 Participants	0 Participants	0 Participants	0 Participants	9 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Black or African American	0 Participants	0 Participants	0 Participants	2 Participants	0 Participants	1 Participants	1 Participants	0 Participants	0 Participants	4 Participants
Race (NIH/OMB) More than one race	0 Participants	1 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants	2 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) White	3 Participants	3 Participants	4 Participants	5 Participants	5 Participants	4 Participants	6 Participants	3 Participants	16 Participants	49 Participants
Region of Enrollment United States	3 participants	4 participants	4 participants	7 participants	5 participants	5 participants	7 participants	3 participants	17 participants	55 participants
Sex: Female, Male Female	2 Participants	2 Participants	1 Participants	2 Participants	3 Participants	2 Participants	4 Participants	2 Participants	3 Participants	21 Participants
Sex: Female, Male Male	1 Participants	2 Participants	3 Participants	5 Participants	2 Participants	3 Participants	3 Participants	1 Participants	14 Participants	34 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk	EG003 affected / at risk	EG004 affected / at risk	EG005 affected / at risk	EG006 affected / at risk	EG007 affected / at risk	EG008 affected / at risk
deaths Total, all-cause mortality	3 / 3	2 / 3	3 / 4	4 / 4	7 / 7	4 / 5	2 / 5	5 / 7	8 / 17
other Total, other adverse events	3 / 3	3 / 3	4 / 4	4 / 4	7 / 7	5 / 5	5 / 5	7 / 7	17 / 17
serious Total, serious adverse events	2 / 3	2 / 3	4 / 4	3 / 4	7 / 7	5 / 5	4 / 5	7 / 7	15 / 17

Outcome results

Primary

Safety Assessed by Adverse Events

Safety and tolerability assessed by adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicity (DLT), dose reductions, delays or withdrawals due to toxicity

Time frame: 12 months

Arm	Measure	Group	Value (NUMBER)
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	2 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	1 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Any TEAEs	3 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	0 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	3 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 1: 50mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	2 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Any TEAEs	3 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	1 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	2 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	1 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	1 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 2: 100mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	2 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	1 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	2 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	4 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	4 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	2 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Any TEAEs	4 participants
Phase 1 Cohort 3: 200mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	2 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	2 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Any TEAEs	4 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	3 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	3 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	2 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	2 participants
Phase 1 Cohort 4: 300mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	1 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	3 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	1 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	7 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	6 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	2 participants
Phase 1 Cohort 5: 400mg/m2	Safety Assessed by Adverse Events	Any TEAEs	7 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	5 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	2 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	3 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	2 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	2 participants
Phase 1 Cohort 6: 500mg/m2	Safety Assessed by Adverse Events	Any TEAEs	5 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	3 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Any TEAEs	5 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	1 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	4 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	4 participants
Phase 1 Cohort 7: 400mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	1 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	7 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	2 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	7 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	0 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Any TEAEs	7 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	3 participants
Phase 1 Cohort 8: 400mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	5 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Interruption of study drug due to TEAEs	7 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Any TEAEs	17 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 study drug related TEAEs	6 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Grade 3 or 4 TEAEs	15 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Death due to TEAEs	0 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Any study drug related TEAEs	14 participants
Phase 2a Cohort 9: 400mg/m2	Safety Assessed by Adverse Events	Any serious TEAEs	11 participants

Secondary

Determination of Objective Response (OR) Rates.

The OR endpoint: proportion of subjects w/ OR as best response (CR, CRi or PR) assessed at the end of Cycles 1 and 3. AML: CR - free of all symptoms related to leukemia, absolute neutrophil count \> 1.0 x 10\^9/L, platelet count ≥ 100 x 10\^9/L, normal bone marrow with \< 5% blasts no Auer rods; CRi - CR with residual thrombocytopenia (platelet count \< 100 x 10\^9/L) or residual neutropenia (absolute neutrophil count \< 1.0 x 10\^9/L); PR - A ≥ 50% decrease in bone marrow blasts to 5 to 25% abnormal. MDS or CMML: CR - free of all symptoms related to leukemia, absolute neutrophil count ≥ 1.0 x 10\^9/L, platelet count ≥ 100 x 10\^9/L, bone marrow ≤ 5% myeloblasts, w/ normal maturation of all cell lines, hemoglobin ≥ 11g/dL, no blasts in the peripheral blood; PR - All CR criteria with ≥50% decrease in bone marrow blasts over pre-treatment (but still \> 5%); Marrow CR - In bone marrow, ≤ 5% myeloblasts and decrease by ≥ 50% over pre-treatment.

Time frame: OR responses were assessed at end of Cycles 1 thru 3. Each cycle was 28 days in length.

Arm	Measure	Group	Value (NUMBER)
Phase 1 Cohort 1: 50mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	1 participants
Phase 1 Cohort 1: 50mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 2: 100mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	1 participants
Phase 1 Cohort 2: 100mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 3: 200mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 1 Cohort 3: 200mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 4: 300mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 1 Cohort 4: 300mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 5: 400mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 1 Cohort 5: 400mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	1 participants
Phase 1 Cohort 6: 500mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 6: 500mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 1 Cohort 7: 400mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 1 Cohort 7: 400mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	1 participants
Phase 1 Cohort 8: 400mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 1 Cohort 8: 400mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants
Phase 2a Cohort 9: 400mg/m2	Determination of Objective Response (OR) Rates.	Partial Response subjects	0 participants
Phase 2a Cohort 9: 400mg/m2	Determination of Objective Response (OR) Rates.	Complete bone marrow remission (mCR)	0 participants

Source: ClinicalTrials.gov · Data processed: Feb 18, 2026

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies

Conditions

Keywords

Brief summary

Detailed description

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Recruitment details

Pre-assignment details

Participants by arm

Baseline characteristics

Adverse events

Outcome results

Safety Assessed by Adverse Events

Determination of Objective Response (OR) Rates.