A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML (TWT-202)

Treadwell Therapeutics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: CMML; AML; MDS; Myelodysplastic Syndromes; Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia

Treatments: Drug: Azacitidine; Drug: CFI-400945

Study type: Interventional

Funder types: Industry

Identifiers

NCT04730258

TWT-202

Details and patient eligibility

About

The purpose of this study is to test the safety of an investigational drug called CFI-400945 alone and in combination with azacitidine.

Full description

This study will be evaluating the safety and tolerability of CFI-400945 in subjects with Acute Myeloid Leukemia, Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia. The study is designed to build on encouraging data from another study and to obtain further safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) data of CFI-400945.

Enrollment: 72 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patients must be >18 years of age

2. For Parts 1A and 1B, the following malignancy types will be included:

   1. Relapsed or refractory AML.
   2. MDS, after prior hypomethylating agents.
   3. CMML, with progressive disease/lack of response after hypomethylating agents

   For Parts 1A and 1B, Patients may have relapsed or refractory disease.

3. For Parts 2A and 2B, the following malignancy types will be included:

   1. Relapsed or Refractory AML.
   2. MDS patients should be limited to high risk disease
   3. MDS or CMML should be previously untreated and patients with AML may have relapsed or refractory disease;

4. Have clinically acceptable laboratory screening results (i.e., clinical chemistry, hematology, and urinalysis) within certain limits per protocol.

5. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

Exclusion criteria

1. Patients who have received investigational therapy, radiotherapy, immunotherapy, monoclonal antibodies, or chemotherapy within 14 days or 5 half-lives (whichever is shorter)
2. Allogeneic or autologous transplant for AML with infusion of stem cells within 90 days before Cycle 1 Day 1, or on active immunosuppressive therapy for graft-versus-host disease (GVHD) or GVHD prophylaxis within 2 weeks of Cycle 1 Day 1.
3. Any Grade ≥ 2 persistent non-hematological toxicity related to allogeneic transplant, such as those requiring systemic immunosuppressive therapy.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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72 participants in 2 patient groups

1A: Monotherapy escalation and expansion

Experimental group

Description:

Dose escalation and expansion arm with CFI-400945

Treatment:

Drug: CFI-400945

2A: Combination escalation and expansion

Experimental group

Description:

Dose escalation and expansion arm with CFI-400945 and azacitidine

Treatment:

Drug: CFI-400945

Drug: Azacitidine