AMLPhase 1/2NCT02193958
Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies
A Phase 1/2 study testing FF-10501-01 in people with AML and CMML, run by Fujifilm Pharmaceuticals U.S.A., Inc. at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- What is being tested?
- FF-10501-01
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2014to 15 Oct 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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,
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- 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
Who cannot join
- 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).
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- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cleveland Clinic at Taussig Cancer Center
Cleveland, Ohio, 44195
M D Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase 1/2a, Dose Escalation Study of FF-10501-01 for the Treatment of Advanced Hematologic Malignancies
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.
Read the 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.
- Being tested
- FF-10501-01
- Keywords
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FF-10501-01Drug
Also called Single Group Assignment, Drug FF-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 group | Role | What they receive |
|---|---|---|
| Phase 1 Cohort 1: 50mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 2: 100mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 3: 200mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 4: 300mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 5: 400mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 6: 500mg/m2 | Experimental | FF-10501-01 tablets BID every 14 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 2a Cohort 7: 400mg/m2 in MDS/CMML | Experimental | FF-10501-01 tablets BID every 21 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
| Phase 1 Cohort 8: 400mg/m2 | Experimental | FF-10501-01 tablets BID every 28 days of a 28 day cycle. Receives: Drug: FF-10501-01 |
| Phase 2a Cohort 9: 400mg/m2 | Experimental | FF-10501-01 tablets BID every 21 days of a 28-day cycle. Receives: Drug: FF-10501-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Secondary outcomes (1)
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.
Measured: OR responses were assessed at end of Cycles 1 thru 3. Each cycle was 28 days in length.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2014 (estimated)
- Study startJul 2014
- Primary completion9 Aug 2019
- Study completion15 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AMLStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fujifilm Pharmaceuticals U.S.A., Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Guillermo Garcia-Manero, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02193958
- Sponsor's protocol ID
- FF1050101US01
- Status last verified
- Mar 2025
- First submitted
- 14 Jul 2014
- Last updated
- 19 Mar 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 32264726
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a, Dose Escalation Study of FF-10501-01 for the Treatment of Advanced Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT02193958
- Acronym / sponsor ID
- FF1050101US01
- Keywords
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Fujifilm Pharmaceuticals U.S.A., Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2014 (actual)
- Primary completion
- 9 Aug 2019 (actual)
- Study completion
- 15 Oct 2019 (actual)
- First posted
- 18 Jul 2014 (estimated)
- Results
- Posted 19 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Phase 1 Cohort 1: 50mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 1 Cohort 2: 100mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 1 Cohort 3: 200mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 1 Cohort 4: 300mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 1 Cohort 5: 400mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 1 Cohort 6: 500mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 14 days of a 28-day cycle. |
| Phase 2a Cohort 7: 400mg/m2 in MDS/CMML | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 21 days of a 28-day cycle. |
| Phase 1 Cohort 8: 400mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 28 days of a 28 day cycle. |
| Phase 2a Cohort 9: 400mg/m2 | Experimental | Drug: FF-10501-01FF-10501-01 tablets BID every 21 days of a 28-day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Confirmed advanced hematologic malignancies;
- 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
- 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.
Key exclusion criteria (10 listed)
- 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.
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 |
|---|---|---|
| FF-10501-01Also: Single Group Assignment, Drug FF-10501-01FF-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. | Drug | Phase 1 Cohort 1: 50mg/m2, 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 8: 400mg/m2, Phase 2a Cohort 7: 400mg/m2 in MDS/CMML, Phase 2a Cohort 9: 400mg/m2 |
Primary outcome
Safety Assessed by Adverse Events
Time frame: 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
1 secondary outcome
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32264726Garcia-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.
- 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.
Guillermo Garcia-Manero, MD principal investigator
M.D. Anderson Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FF-10501-01 in people with AML and CMML, run by Fujifilm Pharmaceuticals U.S.A., Inc. at 2 sites 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 15 inclusion and 10 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: finished, as of the sponsor's last update on 19 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 19 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Safety Assessed by Adverse Events (time frame: 12 months). 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 NCT02193958, which also lists 1 linked publication.
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 19 Mar 2025.