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

NCT06731699 | Recruiting Phase 1

• 1 other identifier: FD-MD-23001
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief 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). 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.

Conditions

AML; NHL

Keywords

FD-001; AML; NHL

Outcome Measures

Primary Outcomes (3)

• 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

  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

  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.

  From enrollment to the end of treatment at 32 days

Secondary Outcomes (5)

• Objective Response Rate (ORR)

  Up to approximately 12 months

• Cmax

  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

  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

  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

  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

Study Arms (1)

FD-001

EXPERIMENTAL

Drug: FD-001

Interventions

FD-001 DRUG

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.

FD-001

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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:

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chengdu FenDi Pharmaceutical Co., Ltd.: lead

Study Sites (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

RECRUITING

Central Study Contacts

Guobing Yang

CONTACT

86+13540819307; yangguobing@fendipharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2024
• First Posted: December 12, 2024
• Study Start: March 11, 2024
• Primary Completion: January 24, 2026
• Study Completion: March 1, 2026
• Last Updated: December 17, 2024

Record last verified: 2024-12

Locations

CN (1)