To Assess the Safety and Tolerability of INCB000928 in Participants With Myelodysplastic Syndromes or Multiple Myeloma

NCT04582539 | Terminated Phase 1 Results FDA Drug

• 2 other identifiers: INCB 00928-1052020-002771-35
• Study Type: interventional
• Target: 22
• Locations: 3 countries
• Sites: 15

Brief Summary

This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

Conditions

Myelodysplastic Syndromes; Multiple Myeloma; Anemia

Keywords

Myelodysplastic Syndromes; Multiple Myeloma; Anemia; LIMBER

Outcome Measures

Primary Outcomes (5)

• Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

  An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE is an AE reported for the first time or the worsening of a pre-existing event after the first dose of study drug.

  up to 950 days

• Number of Participants With Any ≥Grade 3 TEAE

  The severity of AEs was assessed using Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0) Grades 1 through 5. The investigator made an assessment of intensity for each AE and SAE reported during the study and assigned it to one of the following categories. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.

  up to 950 days

• Number of Participants With Dose-limiting Toxicities (DLTs)

  A DLT was defined as the occurrence of any protocol-defined toxicities occurring during the first study drug treatment cycle, from C1D1 up to and including Cycle 1 Day 28 (per regimen cycle schedule), except those with a clear alternative explanation (e.g., disease progression) or transient (≤72 hours) abnormal laboratory values without associated clinically significant signs or symptoms based on investigator determination.

  up to Day 28

• Maximum Tolerated Dose (MTD)

  The MTD was defined as the dose at which the observed DLT rate was closest to the target DLT rate of 28% using an isotonical method that took the assumption of a monotonic dose-toxicity relationship into account. Bayesian optimal interval (BOIN) design was used to determine the MTD for this study. Per the protocol, the stopping rule was either (a) reaching a certain number of participants at one dose level under the early stopping rule or (b) reaching the pre-defined maximum sample size. Dose escalation was to be considered complete only when one of these conditions was met. After completion, the MTD was to be defined as the dose level closest to the target DLT rate. The MTD could not be concluded until the stopping rule was met.

  up to Day 28

• Recommended Dose for Expansion (RDE)

  RDE doses were defined as pharmacodynamically active. RDE doses were not to have exceeded the MTD defined in each treatment group.

  up to Day 28

Secondary Outcomes (18)

• Percentage of Participants With Anemia Response

  up to Week 24

• Duration of Anemia Response

  up to 920 days

• Percentage of Participants With RBC-transfusion Independence (TI)

  up to Week 24

• Duration of RBC-transfusion Independence (TI) Period for Participants Achieving RBC-TI for at Least 8 Consecutive Weeks During the First 24 Weeks of Treatment

  up to 920 days

• Rate of Red Blood Cell (RBC) Transfusion From Week 12 Through Week 24

  from Week 12 through Week 24

Study Arms (1)

INCB000928

EXPERIMENTAL

INCB000928 will be administered in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

Drug: INCB000928

Interventions

INCB000928 DRUG

INCB000928 will be administered once daily.

INCB000928

Eligibility Criteria

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

You may qualify if:

• Agreement to avoid pregnancy or fathering children.
• Participants who are transfusion-dependent or present with symptomatic anemia
• For MDS participants:
• Ineligible to receive or have not responded to available therapies for anemia such as ESAs or lenalidomide.
• Not requiring cytoreductive therapy other than hydroxyurea.
• BM and peripheral blood myeloblast count \< 10%.
• Histologically confirmed diagnosis of the MDS, CMML and unclassifiable MDS/MPN overlap syndromes.
• For MM participants:
• Histologically confirmed diagnosis of MM.
• After failure of available standard treatments such as alkylating agents, glucocorticoids, immunomodulatory drugs (lenalidomide,pomalidomide, or thalidomide), proteasome inhibitors (bortezomib or carfilzomib), and daratumumab.

You may not qualify if:

• Any prior allogeneic stem cell transplantation or a candidate for such transplantation.
• Any major surgery within 28 days before the first dose of study drug.
• Any prior chemotherapy, immunomodulatory drug therapy, immunosuppressive therapy, biological therapy, endocrine therapy, targeted therapy, or antibody or hypomethylating agent to treat the participant's disease within 5 half-lives or 28 days (whichever is shorter) before the first dose of study drug.
• Undergoing treatment with another investigational medication or having been treated with an investigational medication within 28 days before the first dose of study drug. -Undergoing treatment with ESAs, granulocyte colony-stimulating factor or granulocyte/macrophage colony-stimulating factor, romiplostin, or eltrombopag at any time within 28 days before the first dose of study drug.
• Undergoing treatment with a strong or potent inhibitor or inducer of CYP3A4/5 within 28 days or 5 half-lives (whichever is longer) before the first dose of study drug or expected to receive such treatment during the study.
• History of clinically significant or uncontrolled cardiac disease.
• History or presence of an abnormal ECG that, in the investigator's opinion, is clinically Meaningful.
• Presence of chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment.
• Diagnosis of chronic liver disease.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (15)

Stanford Cancer Center

Palo Alto, California, 94304, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Florida Cancer Specialists

Sarasota, Florida, 34232, United States

Location

Tulane Comprehensive Cancer Center

New Orleans, Louisiana, 70112, United States

Location

Barbara Ann Karmanos Cancer Hospital

Detroit, Michigan, 48201, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45219, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Md Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu

Nantes, 44093, France

Location

Hospices Civils de Lyon Centre Hospitalier Lyon Sud

Pierre-Bénite, 69310, France

Location

Institut Gustave Roussy

Villejuif, 94800, France

Location

L Azienda Ospedaliero-Universitaria Di Bologna Policlinico S. Orsola - Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliero-Universitaria Careggi (Aouc)

Florence, 50134, Italy

Location

Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo

Pavia, 27100, Italy

Location

Irccs Istituto Clinico Humanitas

Rozzano, 20089, Italy

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Multiple Myeloma; Anemia

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Limitations and Caveats

The sponsor terminated study enrollment as a strategic decision. Due to early study termination, no participants were enrolled in the expansion stage of the study.

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Ekatarine Asatiani, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: October 6, 2020
• First Posted: October 9, 2020
• Study Start: August 19, 2021
• Primary Completion: August 15, 2024
• Study Completion: August 15, 2024
• Last Updated: October 22, 2025
• Results First Posted: October 22, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.

Locations

US (8); IT (4); FR (3)