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Dose Optimization and Expansion Study of DFV890 in Adult Patients With Myeloid Diseases

NCT ID: NCT05552469

Last Updated: 2025-11-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

105 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-05-08

Study Completion Date

2027-02-01

Brief Summary

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Study CDFV890G12101 is an open-label, phase 1b, multicenter study with a randomized two-dose optimization part, and a dose expansion part consisting of three groups evaluating DFV890 in patients with myeloid diseases. The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, efficacy and recommended dose for single agent DFV890 in patients with lower risk (LR: very low, low or intermediate risk) myelodysplastic syndromes (LR MDS), lower risk chronic myelomonocytic leukemia (LR CMML) and High-Risk Clonal Cytopenia of Undetermined Significance (HR CCUS).

Detailed Description

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This research study is to find out if study treatment DFV890 is safe and tolerable, and can help patients who were diagnosed with a myeloid disease such as: very low, low or intermediate risk myelodysplastic syndromes (MDS), very low, low or intermediate risk chronic myelomonocytic leukemia (CMML) and High-Risk Clonal Cytopenia of Undetermined Significance (HR CCUS). The study seeks to determine the optimal dose of DFV890 that is safe and efficacious in patients with myeloid disease. The effectiveness and safety/tolerability of the study treatment is not yet confirmed in this disease setting.

Eligible patients meeting all study entry requirements will be required to provide a sample from their bone marrow at screening and at select study timepoints. All enrolled patients will be dosed for a minimum of twenty-four weeks (6 cycles of treatment) unless they experience side effects related to the study treatment requiring dose interruption/discontinuation, worsening of the disease, and/or if treatment is discontinued at the discretion of the investigator or the patient.

Conditions

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Myeloid Diseases

Keywords

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MDS CMML CCUS CHRS NLRP3 Myelodysplastic Syndromes Chronic Myelomonocytic Leukemia inflammasome CYP2C9

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose escalation: DFV890 low dose

DFV890 given as single agent at a low dose

Group Type EXPERIMENTAL

DFV890

Intervention Type DRUG

DFV890 Single Agent

Dose escalation: DFV890 high dose

DFV890 given as single agent at a high dose

Group Type EXPERIMENTAL

DFV890

Intervention Type DRUG

DFV890 Single Agent

Dose expansion: DFV890 low dose

DFV890 given as single agent at a low dose

Group Type EXPERIMENTAL

DFV890

Intervention Type DRUG

DFV890 Single Agent

Interventions

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DFV890

DFV890 Single Agent

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1\. Patients must be ≥ 18 years of age at the time of signing the informed consent form (ICF) 2. The Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 2 3. Patient must be a candidate for serial bone marrow aspirate and/or biopsy according to the institutions guidelines and must be willing to undergo a bone marrow aspirate.

4\. Patients must have one of the following for eligibility into the study:

1. In dose optimization: IPSS-R defined very low, low or intermediate risk Myelodysplastic Syndrome (LR MDS) who failed to respond to or did not tolerate ESAs or luspatercept or HMAs and patients with del 5q who failed to respond to or did not tolerate lenalidomide; or
2. In dose optimization and expansion: IPSS-R defined very low, low or intermediate risk Chronic Myelomonocytic Leukemia (LR CMML) who failed to respond to or did not tolerate hydroxyurea or HMAs.
3. changes for dose expansion (applicable as of amendment 3):

1. LR MDS with ≤ 10% bone marrow blasts, IPSS-R score of ≤ 3.5, transfusion independent (TID) status as per IWG 2006 criteria (requiring \<4U pRBC in 8 weeks), clinically meaningful cytopenia(s) and no or limited (\<4 months) prior therapy for MDS.
2. LR CMML patients with symptomatic cytopenias and/or constitutional symptoms refractory, intolerant or unsuitable for standard first-line therapy.
3. HR-CCUS: Diagnosis of high-risk CCUS by clonal hematopoiesis risk score (CHRS) with clinically meaningful cytopenias and no prior therapy for a myeloid neoplasm.

Exclusion Criteria

1\. Systemic antineoplastic therapy (including cytotoxic chemotherapy, alpha-interferon, kinase inhibitors or other targeted small molecules, and toxin-immunoconjugates) or any experimental therapy within 28 days or 5 half-lives, whichever is longer, and recovered from the toxicities before the first dose of study treatment. For patients that received antibodies the washout period is 4 weeks prior to study treatment.

a. For TID LR MDS in Dose Expansion Phase only (applicable as of amendment 03):

1. Prior therapy for MDS administered for \>4 months (ESA and luspatercept administered for ≤4 months will be allowed if washout period followed)
2. Concurrent malignancy requiring active systemic therapy
3. Prior or concurrent cytotoxic chemotherapy for MDS at any time

2\. History of hypersensitivity to the study treatment or its excipients or to drugs of similar chemical classes.

3\. Patients who have previously been treated with agents that have the same mechanism of action as DFV890 as defined in Table 6-7, list of prohibited medications (e.g., drugs targeting the NLRP3 inflammasome pathway and the IL-1 pathway (canakinumab and anakinra)).

4\. Use of hematopoietic colony-stimulating growth factors (e.g., G-CSF, GM-CSF, M-CSF), thrombopoietin mimetics or erythroid stimulating agents anytime ≤ 1 week (or 5 half lives, whichever is longer) prior to start of study treatment.

5\. Patients receiving:

a. concomitant medications that are known to be modulators of cytochrome P450 enzymes CYP2C9 and/or CYP3A (specifically strong or moderate inducers of CYP2C9, strong inducers of CYP3A enzymes, strong inhibitors of CYP2C9 and/or strong or moderate dual inhibitors of CYP2C9/CYP3A); and b. patients, who are poor CYP2C9 metabolizers receiving concomitant medications known to be strong or moderate inhibitors of CYP3A, whose concomitant medications cannot be discontinued or switched to a different medication within 5 half-lives or 1 week (whichever is longer) prior to start of study treatment and for duration of the study. See Section 6.8 and list of prohibited drugs in Appendix 8 for more details.

6\. Dose expansion only: Poor CYP2C9 metabolizers defined as genotype of the CYP2C9 \*3/\*3 or CYP2C9 \*2/\*3 allele combinations are excluded.
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Stanford Cancer Center

Stanford, California, United States

Site Status RECRUITING

H Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, United States

Site Status RECRUITING

Emory University School of Medicine-Winship Cancer Institute

Atlanta, Georgia, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

Sidney Kimmel CCC At JH

Baltimore, Maryland, United States

Site Status RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status COMPLETED

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Weill Cornell Medicine NY-Presb

New York, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Ctr

New York, New York, United States

Site Status RECRUITING

Vanderbilt University Medical Ctr

Nashville, Tennessee, United States

Site Status RECRUITING

Univ of TX MD Anderson Cancer Cntr

Houston, Texas, United States

Site Status RECRUITING

Huntsman Cancer Institute

Salt Lake City, Utah, United States

Site Status RECRUITING

Novartis Investigative Site

Grenoble, , France

Site Status RECRUITING

Novartis Investigative Site

Marseille, , France

Site Status RECRUITING

Novartis Investigative Site

Nantes, , France

Site Status RECRUITING

Novartis Investigative Site

Paris, , France

Site Status RECRUITING

Novartis Investigative Site

Düsseldorf, North Rhine-Westphalia, Germany

Site Status RECRUITING

Novartis Investigative Site

Velbert, North Rhine-Westphalia, Germany

Site Status WITHDRAWN

Novartis Investigative Site

Dresden, Saxony, Germany

Site Status RECRUITING

Novartis Investigative Site

Leipzig, Saxony, Germany

Site Status RECRUITING

Novartis Investigative Site

Lübeck, , Germany

Site Status RECRUITING

Novartis Investigative Site

Hong Kong, , Hong Kong

Site Status RECRUITING

Novartis Investigative Site

Brescia, BS, Italy

Site Status RECRUITING

Novartis Investigative Site

Rozzano, MI, Italy

Site Status RECRUITING

Novartis Investigative Site

Singapore, , Singapore

Site Status RECRUITING

Novartis Investigative Site

Singapore, , Singapore

Site Status WITHDRAWN

Novartis Investigative Site

Madrid, , Spain

Site Status RECRUITING

Novartis Investigative Site

Madrid, , Spain

Site Status RECRUITING

Novartis Investigative Site

Cardiff, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States France Germany Hong Kong Italy Singapore Spain United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Mani Gupta

Role: primary

Cyril Patra

Role: primary

Ariel Kay

Role: primary

Alesi Monterroso

Role: primary

Matthew Gagaring

Role: primary

Misty Keller

Role: primary

Tania Curcio, NP

Role: primary

Marissa Giuliani

Role: primary

Role: primary

Role: primary

Phoenix Hines

Role: primary

Other Identifiers

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2022-501406-36-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

CDFV890G12101

Identifier Type: -

Identifier Source: org_study_id