A Phase 1 Study of FD-001 in Recurrent /Refractory (R/R)AML/NHL/MM/MDS

December 12, 2024 updated by: Chengdu FenDi Pharmaceutical Co., Ltd.

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
72

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Hospital of Sichuan University
        • Contact:
        • Contact:
          • Ting Niu, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. 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.
  2. Age range from 18 to 80 years (inclusive), with no gender restrictions.
  3. ECOG performance status ≤2 (refer to Appendix 1).
  4. Expected minimum survival period of at least 3 months.
  5. 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.

  6. Presence of at least one measurable lesion:

    • 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:

      1. Monoclonal protein detected in blood ≥1 g/dL (10g/L) through serum protein electrophoresis.
      2. Urine monoclonal protein ≥200 mg over a span of 24 hours.
      3. 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.
  7. 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).

  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)

Exclusion Criteria:

  1. 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;
  2. Chronic myeloid leukemia (CML) with BCR/ABL positivity;
  3. The subject has central nervous system malignant tumor infiltration;
  4. 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);
  5. Within 4 weeks prior to the first dose of the study drug, the subject has received an active or attenuated live vaccine;
  6. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: FD-001
Drug: FD-001
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
From enrollment to the end of treatment at 32 days
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
From enrollment to the end of treatment at 32 days
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.
From enrollment to the end of treatment at 32 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Objective Response Rate (ORR)
Time Frame: Up to approximately 12 months
ORR is defined as the percentage of participants,who has a CR or PR
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
Maximum serum concentration(Cmax) of FD-001 will be investigated
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
To characterize the pharmacokineties of FD-001by assessment of time to reach maximum plasma concentration
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
To characterize the pharmacokinetics of FD-001 by assessment of area under the plasma concentration time curve from zero to infinity
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
To characterize the pharmacokinetics of FD-001 by assessment of the duration required for the peak plasma concentration to be reduced by half
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Chengdu FenDi Pharmaceutical Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 11, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 24, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 6, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 9, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 12, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 12, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2024

More Information

Terms related to this study

Keywords

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • FD-MD-23001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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