Testing FD-001 for recurrent or refractory blood cancers

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 study is testing a new medication called FD-001 to see if it can help people with tough-to-treat blood cancers like acute myeloid leukemia and non-Hodgkin lymphoma.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 72 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Chengdu FenDi Pharmaceutical Co., Ltd. (industry)
• Drugs / interventions: chemotherapy, immunotherapy, radiation
• Locations: 1 site (Chengdu, Sichuan)
• Trial ID: NCT06731699 on ClinicalTrials.gov

What this trial studies

This is a Phase 1 clinical trial that evaluates the safety, tolerability, and preliminary efficacy of FD-001 capsules in patients with recurrent or refractory hematologic malignancies, including acute myeloid leukemia (AML) and non-Hodgkin lymphoma (NHL). The trial consists of a dose-escalation phase to determine the maximum tolerated dose and a dose-expansion phase for further assessment. Participants will undergo a screening period followed by treatment and follow-up phases, with blood samples collected for pharmacokinetic studies.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 80 with recurrent or refractory hematologic malignancies who have failed standard treatments.

Not a fit: Patients with hematologic malignancies that are not recurrent or refractory, or those who have not failed standard treatments, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat blood cancers.

How similar studies have performed: Other studies have shown promise with similar investigational therapies in treating hematologic malignancies, suggesting potential for success.

Eligibility criteria

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.

Where this trial is running

Chengdu, Sichuan

• West China Hospital of Sichuan University — Chengdu, Sichuan, China (RECRUITING)

Study contacts

• Study coordinator: Guobing Yang

Conditions: AML, NHL, FD-001