---
title: A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML
nct_id: NCT04730258
overall_status: ACTIVE_NOT_RECRUITING
phase: PHASE1, PHASE2
sponsor: Treadwell Therapeutics, Inc
study_type: INTERVENTIONAL
primary_condition: Acute Myeloid Leukemia
countries: United States, Canada, Hong Kong
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04730258.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04730258"
ct_last_update_post_date: 2025-05-18
last_seen_at: "2026-05-12T06:44:03.185Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML

**Official Title:** Phase 1b/2 Clinical Study of the Safety, Tolerability, and Pharmacokinetic and Pharmacodynamic Profiles of CFI-400945 as a Single Agent or in Combination With Azacitidine in Patients With AML, MDS or CMML

**NCT ID:** [NCT04730258](https://clinicaltrials.gov/study/NCT04730258)

## Key Facts

- **Status:** ACTIVE_NOT_RECRUITING
- **Phase:** PHASE1, PHASE2
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 72
- **Lead Sponsor:** Treadwell Therapeutics, Inc
- **Conditions:** Acute Myeloid Leukemia, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, AML, MDS, CMML
- **Start Date:** 2021-04-16
- **Completion Date:** 2026-01
- **CT.gov Last Update:** 2025-05-18

## Brief Summary

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

## Detailed 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.

## Eligibility

- **Minimum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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.
```

## Arms

- **1A: Monotherapy escalation and expansion** (EXPERIMENTAL) — Dose escalation and expansion arm with CFI-400945
- **2A: Combination escalation and expansion** (EXPERIMENTAL) — Dose escalation and expansion arm with CFI-400945 and azacitidine

## Interventions

- **CFI-400945** (DRUG) — The starting dose is 32 mg/day for escalation arms and the recommended starting dose for the expansion arms.
- **Azacitidine** (DRUG) — Azacitidine will be given at its labeled dose and schedule

## Primary Outcomes

- **Incidence of treatment emergent AEs** _(time frame: 36 months)_ — The number of subjects who experience an adverse event that was possibly related to study drug
- **Treatment emergent changes in vital signs** _(time frame: 36 months)_ — The number of subjects who experience changes in blood pressure, heart rate, respiratory rate, body temperature that was possibly related to study drug.
- **Treatment emergent changes in clinical laboratory tests** _(time frame: 36 months)_ — The number of subjects who experience a change in laboratory parameters that was possibly related to study drug.
- **Treatment emergent changes in physical examinations, ECOG performance status, electrocardiograms (ECGs), echocardiograms and cardiac troponins** _(time frame: 36 months)_ — The number of subjects who experience changes in physical examinations, performance status, ECG, troponins that were possibly related to study drug.

## Secondary Outcomes

- **Composite Complete Remission Rate, CRc (complete remission + complete remission with incomplete blood count recovery + complete remission with incomplete platelet count recovery [CR + CRi + CRp])** _(time frame: 36 months)_
- **Overall response rate (ORR, defined as Complete remission + Marrow CR + Partial remission + Hematologic Improvement (CR + mCR+ PR + HI)** _(time frame: 36 months)_
- **The pharmacokinetics of CFI-400945 will be assessed through AUC.** _(time frame: 36 months)_
- **To assess the pharmacokinetic profile of CFI-400945 through Cmax.** _(time frame: 36 months)_
- **To assess the pharmacokinetic profile of CFI-400945 through T1/2.** _(time frame: 36 months)_

## Locations (9)

- City of Hope, Duarte, California, United States
- University of California Davis Comprehensive Cancer Center, Sacramento, California, United States
- Norton Cancer Institute - Saint Matthews, Louisville, Kentucky, United States
- New York Presbyterian Weill Cornell Medical Center, New York, New York, United States
- The Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States
- The University of Texas MD Anderson Cancer Centre, Houston, Texas, United States
- University of Alberta, Edmonton, Alberta, Canada
- Princess Margaret Cancer Center, Toronto, Ontario, Canada
- Queen Mary Hospital, Hong Kong, Hong Kong

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.city of hope|duarte|california|united states` — added _(2026-05-12)_
- `locations.university of california davis comprehensive cancer center|sacramento|california|united states` — added _(2026-05-12)_
- `locations.norton cancer institute - saint matthews|louisville|kentucky|united states` — added _(2026-05-12)_
- `locations.new york presbyterian weill cornell medical center|new york|new york|united states` — added _(2026-05-12)_
- `locations.the ohio state university comprehensive cancer center|columbus|ohio|united states` — added _(2026-05-12)_
- `locations.the university of texas md anderson cancer centre|houston|texas|united states` — added _(2026-05-12)_
- `locations.university of alberta|edmonton|alberta|canada` — added _(2026-05-12)_
- `locations.princess margaret cancer center|toronto|ontario|canada` — added _(2026-05-12)_
- `locations.queen mary hospital|hong kong||hong kong` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04730258.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04730258*  
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