AMLPhase 1NCT06731699
A Phase 1 Study of FD-001 in Recurrent /Refractory (R/R)AML/NHL/MM/MDS
A Phase 1 study testing FD-001 in people with AML and NHL, run by Chengdu FenDi Pharmaceutical Co., Ltd. at 1 site in China.
Trial summary
This is a single-arm, open-label, dose-escalation and dose-expansion phase I clinical trial aimed at assessing the safety, tolerability, pharmacokinetic/pharmacodynamic profile, and preliminary efficacy of FD-001 capsules in the treatment of recurrent/refractory hematologic malignancies(AML/MDS/NHL/MM). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- What is being tested?
- FD-001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteChina
- Study dates
- 11 Mar 2024to 1 Mar 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing to voluntarily participate in this clinical study; possess a comprehensive understanding of, provide consent for, and sign the written informed consent form (ICF) for this study; demonstrate willingness to adhere to and complete all study procedures.
- Age range from 18 to 80 years (inclusive), with no gender restrictions.
- ECOG performance status ≤2 (refer to Appendix 1).
- Expected minimum survival period of at least 3 months.
- Patients diagnosed with hematological malignancies confirmed through pathological and/or cytological examinations, who have either failed or not received standard treatment due to lack of efficacy or intolerance. This includes patients with recurrent/refractory conditions.
- Presence of at least one measurable lesion:
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- For AML: bone marrow containing more than 5% primitive/immature cells (excluding regeneration after consolidation chemotherapy).
- For MDS: bone marrow aspirate or biopsy pathological examination revealing less than 20% primitive cells.
- For MM: meeting any of the following criteria:
- Monoclonal protein detected in blood ≥1 g/dL (10g/L) through serum protein electrophoresis.
- Urine monoclonal protein ≥200 mg over a span of 24 hours.
- If monoclonal protein is undetectable in blood or urine, affected-to-unaffected serum FLC ratio should be ≥100 (with affected serum FLC level being ≥100 mg/L).
- For NHL: presence of measurable lesions identified by CT, PET-CT, or PET-MRI scans such as lymph node lesions with major axis >1.5 cm or extranodal lesions with major axis >1.0 cm); CLL/SLL: peripheral blood monoclonal lymphocytes ≥5.0×10^9/L; WM: IgM >2×ULN.
- Adverse effects resulting from previous anti-cancer therapy should have recovered to grade ≤1 (continuous hair loss excluded along with laboratory tests specified in criterion 8).
- With sufficient organ function support, all the following criteria must be met during the laboratory tests in the screening period:
- The white blood cell (WBC) count should be less than or equal to 30×10^9/L before the first use of the investigational drug; (This criterion is only applicable to AML and MDS, and the use of hydroxyurea to lower the white blood cell count is permitted.) The absolute neutrophil count (ANC) should be greater than or equal to 1.0×10^9/L before the first use of the investigational drug, and it can be relaxed to 0.75×10^9/L in the case of bone marrow infiltration; the platelet count (PLT) should be greater than or equal to 70×10^9/L before the first use of the investigational drug, and it can be relaxed to 50×10^9/L in the case of bone marrow infiltration; the hemoglobin (HB) level should be greater than or equal to 70 g/L before the first use of the investigational drug, and it can be relaxed to 60 g/L in the case of bone marrow infiltration (This criterion is only applicable to MM and NHL; It is required that this test was not conducted within 7 days before blood collection after the administration of G-CSF (or GM-CSF), erythropoietin, thrombopoietin treatment or component blood transfusion)
Who cannot join
- Within 4 weeks prior to the first dose of the study drug or within 5 half-lives (whichever is shorter), the subject has received anti-cancer therapy, including chemotherapy, immunotherapy, targeted therapy (excluding hydroxyurea therapy and prophylactic intrathecal injection of chemotherapy drugs); received radiation therapy within 2 weeks; received anti-cancer therapy with Chinese herbal medicine within 2 weeks;
- Chronic myeloid leukemia (CML) with BCR/ABL positivity;
- The subject has central nervous system malignant tumor infiltration;
- The subject has another malignant tumor at the same time (excluding IB stage or lower-stage cervical cancer that has been cured, non-invasive basal cell or squamous cell skin cancer, and other malignant tumors that have achieved complete remission (CR) >10 years and >5 years, respectively);
- Within 4 weeks prior to the first dose of the study drug, the subject has received an active or attenuated live vaccine;
- The subject has a history of clear alcohol and drug abuse; a history of severe allergies in the past, or is allergic to any component of the study drug.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041
Ting Niu, MD, 86+02885423046, tingniu@sina.com
Ting Niu, MD
About this study
Official title: A Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Initial Efficacy of FD-001 in Patients with Relapsed or Refractory Hematological Malignancies.
This is a single-arm, open-label, dose-escalation and dose-expansion phase I clinical trial aimed at assessing the safety, tolerability, pharmacokinetic/pharmacodynamic profile, and preliminary efficacy of FD-001 capsules in the treatment of recurrent/refractory hematologic malignancies(AML/MDS/NHL/MM). The trial consists of two phases: the initial phase (dose escalation) and the subsequent phase (dose expansion). The primary objectives are to evaluate the safety and tolerability of FD-001 in subjects with recurrent/refractory hematologic tumors and determine the maximum tolerated dose (MTD) as well as the recommended Phase II dose (RP2D) for FD-001 in this patient population.
Read the detailed description
This is a single-arm, open-label, dose-escalation and dose-expansion phase I clinical trial aimed at assessing the safety, tolerability, pharmacokinetic/pharmacodynamic profile, and preliminary efficacy of FD-001 capsules in the treatment of recurrent/refractory hematologic malignancies(AML/MDS/NHL/MM).The trial process consists of three periods: the screening period, the treatment period, and the follow-up period. The screening period occurred from D-28 to D-1. The treatment period (DLT observation period) includes a single administration phase (C0D1-C0D4) (oral administration on D1, followed by safety observation from D2-D4), as well as multiple administration phase 1 (C1D1-C1D28) (oral administration on D1-D2 and D7-D9, with withdrawal for the remaining time). Blood samples were collected in the first cycle (C1D1~C1D28) for PK study, and part of the PK test blood samples may be used for metabolite identification.After completion of the DLT evaluation, subjects without intolerable toxicity may enter the maintenance treatment period if determined by the investigator that continued treatment outweighs risks. The maintenance treatment period lasts for 28 days per cycle (CND1-CND28), with oral administration on D1-D3 and D8-D10 per cycle, while withdrawing for the rest of the time.
- Being tested
- FD-001
- Keywords
- FD-001, AML, NHL
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FD-001Drug
The initial dose was 0.4mg, and subsequent doses of 0.8mg, 1.6mg, 2.4mg, 3.2mg, and 4.0mg were proposed to be administered in a progressive manner using the "rapid titration" method combined with the traditional "3+3" experimental design. The trial process consists of three periods: screening period, treatment period, and follow-up period. The screening period lasted from D-28 to D-1 for treatment (DLT observation period): during this time frame, there was a single administration period (C0D1-C0D4) where oral administration occurred on day one followed by safety observations on days two to four; as well as multiple administration period one (C1D1-C1D28) where oral administration took place on days one to two and seven to nine with rest periods in between. The drug is available in capsule form.
| Study group | Role | What they receive |
|---|---|---|
| FD-001 | Experimental | Not described Receives: Drug: FD-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase Ⅰa:Dose limiting toxicity(DLT)
DLT are assessed according to NCI-CTCAE v5.0 during the first cycle and defined as occurrence of any of the toxicities in DLT definition if judged by the investigator to be possibly,probably or definitely related to study drug administration
Measured: From enrollment to the end of treatment at 32 days
Phase Ⅰa:Maximum tolerated dose(MTD)
MTD is defined as the highest dose level at which no more than 1 in 3 participants experienced a DLT during the first cycle
Measured: From enrollment to the end of treatment at 32 days
Phase Ⅰb:Recommended Phase Ⅱ Dose(RP2D)
The RP2D is defined as the dose level chosen by the sponsor (in consultation with the investigators) for phase Ⅱ study,based on safety,tolerability,efficacy,PK,and PD data collected during the dose escalation study of FD-001.
Measured: From enrollment to the end of treatment at 32 days
Secondary outcomes (5)
Objective Response Rate (ORR)
ORR is defined as the percentage of participants,who has a CR or PR
Measured: Up to approximately 12 months
Cmax
Maximum serum concentration(Cmax) of FD-001 will be investigated
Measured: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Tmax
To characterize the pharmacokineties of FD-001by assessment of time to reach maximum plasma concentration
Measured: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Area Under the Curve (AUC)0-t
To characterize the pharmacokinetics of FD-001 by assessment of area under the plasma concentration time curve from zero to infinity
Measured: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
T1/2
To characterize the pharmacokinetics of FD-001 by assessment of the duration required for the peak plasma concentration to be reduced by half
Measured: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2024
- Study start11 Mar 2024
- Primary completion24 Jan 2026 (estimated)
- Study completion1 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chengdu FenDi Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06731699
- Sponsor's protocol ID
- FD-MD-23001
- Status last verified
- Dec 2024
- First submitted
- 6 Dec 2024
- Last updated
- 17 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Initial Efficacy of FD-001 in Patients with Relapsed or Refractory Hematological Malignancies.
- ClinicalTrials.gov ID
- NCT06731699
- Acronym / sponsor ID
- FD-MD-23001
- Keywords
- FD-001, AML, NHL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Chengdu FenDi Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 11 Mar 2024 (actual)
- Primary completion
- 24 Jan 2026 (estimated)
- Study completion
- 1 Mar 2026 (estimated)
- First posted
- 12 Dec 2024 (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.
This is a single-arm, open-label, dose-escalation and dose-expansion phase I clinical trial aimed at assessing the safety, tolerability, pharmacokinetic/pharmacodynamic profile, and preliminary efficacy of FD-001 capsules in the treatment of recurrent/refractory hematologic malignancies(AML/MDS/NHL/MM). The trial consists of two phases: the initial phase (dose escalation) and the subsequent phase (dose expansion). The primary objectives are to evaluate the safety and tolerability of FD-001 in subjects with recurrent/refractory hematologic tumors and determine the maximum tolerated dose (MTD) as well as the recommended Phase II dose (RP2D) for FD-001 in this patient population.
Detailed description
This is a single-arm, open-label, dose-escalation and dose-expansion phase I clinical trial aimed at assessing the safety, tolerability, pharmacokinetic/pharmacodynamic profile, and preliminary efficacy of FD-001 capsules in the treatment of recurrent/refractory hematologic malignancies(AML/MDS/NHL/MM).The trial process consists of three periods: the screening period, the treatment period, and the follow-up period. The screening period occurred from D-28 to D-1. The treatment period (DLT observation period) includes a single administration phase (C0D1-C0D4) (oral administration on D1, followed by safety observation from D2-D4), as well as multiple administration phase 1 (C1D1-C1D28) (oral administration on D1-D2 and D7-D9, with withdrawal for the remaining time). Blood samples were collected in the first cycle (C1D1~C1D28) for PK study, and part of the PK test blood samples may be used for metabolite identification.After completion of the DLT evaluation, subjects without intolerable toxicity may enter the maintenance treatment period if determined by the investigator that continued treatment outweighs risks. The maintenance treatment period lasts for 28 days per cycle (CND1-CND28), with oral administration on D1-D3 and D8-D10 per cycle, while withdrawing for the rest of the time.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FD-001 | Experimental | Drug: FD-001 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Willing to voluntarily participate in this clinical study; possess a comprehensive understanding of, provide consent for, and sign the written informed consent form (ICF) for this study; demonstrate willingness to adhere to and complete all study procedures.
- Age range from 18 to 80 years (inclusive), with no gender restrictions.
- ECOG performance status ≤2 (refer to Appendix 1).
- Expected minimum survival period of at least 3 months.
- Patients diagnosed with hematological malignancies confirmed through pathological and/or cytological examinations, who have either failed or not received standard treatment due to lack of efficacy or intolerance. This includes patients with recurrent/refractory conditions.
Key exclusion criteria (6 listed)
- Within 4 weeks prior to the first dose of the study drug or within 5 half-lives (whichever is shorter), the subject has received anti-cancer therapy, including chemotherapy, immunotherapy, targeted therapy (excluding hydroxyurea therapy and prophylactic intrathecal injection of chemotherapy drugs); received radiation therapy within 2 weeks; received anti-cancer therapy with Chinese herbal medicine within 2 weeks;
- Chronic myeloid leukemia (CML) with BCR/ABL positivity;
- The subject has central nervous system malignant tumor infiltration;
- The subject has another malignant tumor at the same time (excluding IB stage or lower-stage cervical cancer that has been cured, non-invasive basal cell or squamous cell skin cancer, and other malignant tumors that have achieved complete remission (CR) >10 years and >5 years, respectively);
- Within 4 weeks prior to the first dose of the study drug, the subject has received an active or attenuated live vaccine;
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 |
|---|---|---|
| FD-001The initial dose was 0.4mg, and subsequent doses of 0.8mg, 1.6mg, 2.4mg, 3.2mg, and 4.0mg were proposed to be administered in a progressive manner using the "rapid titration" method combined with the traditional "3+3" experimental design. The trial process consists of three periods: screening period, treatment period, and follow-up period. The screening period lasted from D-28 to D-1 for treatment (DLT observation period): during this time frame, there was a single administration period (C0D1-C0D4) where oral administration occurred on day one followed by safety observations on days two to four; as well as multiple administration period one (C1D1-C1D28) where oral administration took place on days one to two and seven to nine with rest periods in between. The drug is available in capsule form. | Drug | FD-001 |
Primary outcome
Phase Ⅰa:Dose limiting toxicity(DLT)
Time frame: From enrollment to the end of treatment at 32 days
DLT are assessed according to NCI-CTCAE v5.0 during the first cycle and defined as occurrence of any of the toxicities in DLT definition if judged by the investigator to be possibly,probably or definitely related to study drug administration
Phase Ⅰa:Maximum tolerated dose(MTD)
Time frame: From enrollment to the end of treatment at 32 days
MTD is defined as the highest dose level at which no more than 1 in 3 participants experienced a DLT during the first cycle
Phase Ⅰb:Recommended Phase Ⅱ Dose(RP2D)
Time frame: From enrollment to the end of treatment at 32 days
The RP2D is defined as the dose level chosen by the sponsor (in consultation with the investigators) for phase Ⅱ study,based on safety,tolerability,efficacy,PK,and PD data collected during the dose escalation study of FD-001.
5 secondary outcomes
Objective Response Rate (ORR)
Time frame: Up to approximately 12 months
Cmax
Time frame: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Tmax
Time frame: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Area Under the Curve (AUC)0-t
Time frame: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
T1/2
Time frame: Hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on single dose;Hour 0 of day 7,day8 on multiple dose and hour 0,0.5,1,2,3,4,6,12,24,48,72hours post-dose on multiple dose of day 9
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2024.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FD-001 in people with AML and NHL, run by Chengdu FenDi Pharmaceutical Co., Ltd. at 1 site in China.
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 16 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 17 Dec 2024. 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 China. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Guobing Yang (86+13540819307, yangguobing@fendipharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase Ⅰa:Dose limiting toxicity(DLT) (time frame: From enrollment to the end of treatment at 32 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Dec 2024.