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NCT02193958 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE1, PHASE2
Status at capture
COMPLETED
Registry last update
2025-03-19

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Safety Assessed by Adverse Events

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outcome · POSTED

Determination of Objective Response (OR) Rates.

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
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.
healthy Volunteers
false
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 1: 50mg/m2
    type
    EXPERIMENTAL
  2. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 2: 100mg/m2
    type
    EXPERIMENTAL
  3. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 3: 200mg/m2
    type
    EXPERIMENTAL
  4. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 4: 300mg/m2
    type
    EXPERIMENTAL
  5. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 5: 400mg/m2
    type
    EXPERIMENTAL
  6. description
    FF-10501-01 tablets BID every 14 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 6: 500mg/m2
    type
    EXPERIMENTAL
  7. description
    FF-10501-01 tablets BID every 21 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 2a Cohort 7: 400mg/m2 in MDS/CMML
    type
    EXPERIMENTAL
  8. description
    FF-10501-01 tablets BID every 28 days of a 28 day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 1 Cohort 8: 400mg/m2
    type
    EXPERIMENTAL
  9. description
    FF-10501-01 tablets BID every 21 days of a 28-day cycle.
    intervention Names
    1. Drug: FF-10501-01
    label
    Phase 2a Cohort 9: 400mg/m2
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Phase 1 Cohort 1: 50mg/m2
    2. Phase 1 Cohort 2: 100mg/m2
    3. Phase 1 Cohort 3: 200mg/m2
    4. Phase 1 Cohort 4: 300mg/m2
    5. Phase 1 Cohort 5: 400mg/m2
    6. Phase 1 Cohort 6: 500mg/m2
    7. Phase 1 Cohort 8: 400mg/m2
    8. Phase 2a Cohort 7: 400mg/m2 in MDS/CMML
    9. Phase 2a Cohort 9: 400mg/m2
    description
    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.
    name
    FF-10501-01
    other Names
    1. Single Group Assignment
    2. Drug FF-10501-01
    type
    DRUG
Study design
allocation
NON_RANDOMIZED
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
55
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Safety and tolerability assessed by adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicity (DLT), dose reductions, delays or withdrawals due to toxicity
    measure
    Safety Assessed by Adverse Events
    time Frame
    12 months
secondary Outcomes
  1. description
    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.
    measure
    Determination of Objective Response (OR) Rates.
    time Frame
    OR responses were assessed at end of Cycles 1 thru 3. Each cycle was 28 days in length.
Full study description
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.
Source references
references
  1. citation
    Garcia-Manero G, Pemmaraju N, Alvarado Y, Naqvi K, Ravandi F, Jabbour E, De Lumpa R, Kantarjian H, Advani A, Mukherjee S, Gerds A, Carraway HE, Nazha A, Iwamura H, Murase M, Bavisotto L, Kurman M, Maier G, Johansen M, Sekeres MA. Results of a Phase 1/2a dose-escalation study of FF-10501-01, an IMPDH inhibitor, in patients with acute myeloid leukemia or myelodysplastic syndromes. Leuk Lymphoma. 2020 Aug;61(8):1943-1953. doi: 10.1080/10428194.2020.1747065. Epub 2020 Apr 7.
    pmid
    32264726
    type
    DERIVED
Source notices and limitations
    Discovery and provenance
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    Preserved source evidence · Not medical advice