About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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);
Trial design
Single group
Treatments tested in this trial
FD-001
DrugThe 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.
Treatment groups
Trial outcomes
Primary outcomes
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
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
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.
Secondary outcomes
Objective Response Rate (ORR)
ORR is defined as the percentage of participants,who has a CR or PR
Cmax
Maximum serum concentration(Cmax) of FD-001 will be investigated
Tmax
To characterize the pharmacokineties of FD-001by assessment of time to reach maximum plasma concentration
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
Sponsors and contacts
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