Clinical trial NCT04582539: To Assess the Safety and Tolerability of INCB000928 in Participants With Myelodysplastic Syndromes or Multiple Myeloma
Official title: A Phase 1/2, Open-Label, Multicenter Study of INCB000928 Administered as a Monotherapy in Participants With Anemia Due to Myelodysplastic Syndromes or Multiple Myeloma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Myelodysplastic Syndromes
Multiple Myeloma
Anemia
Keywords: Myelodysplastic Syndromes
Multiple Myeloma
Anemia
LIMBER
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2020-10-09
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Number of treatment-related adverse events
timeFrame: Approximately up to 7 months
Secondary outcomes:
measure: Proportion of participants with anemia response (for TI patients at baseline)
timeFrame: Approximately up to 7 months
measure: Duration of anemia response
timeFrame: Approximately up to 7 months
measure: Proportion of participants with RBC-TI (for TD at baseline)
timeFrame: Approximately up to 7 months
measure: Duration of RBC-TI period
timeFrame: Approximately up to 7 months
measure: Rate of RBC transfusion
timeFrame: Through weeks 12 and 24
measure: Increase in mean Hgb
timeFrame: Approximately up to 7 months
measure: MDS Participants only : Overall Response Rate
timeFrame: Approximately up to 7 months
measure: MDS Participants only : Progression Free Survival
timeFrame: Approximately up to 7 months
measure: MDS Participants only : Leukemia Free Survival
timeFrame: Approximately up to 7 months
measure: MM participants only : Overall Response Rate
timeFrame: Approximately up to 7 months
measure: MM Participants only : Progression Free Survival
timeFrame: Approximately up to 7 months
measure: Cmax
timeFrame: C1D1 and C1D15
measure: Tmax
timeFrame: C1D1 and C1D15
measure: AUC0-t
timeFrame: C1D1 and C1D15
measure: Hepcidin levels
timeFrame: Approximately upto 7 months
measure: Iron Homeostasis
timeFrame: Approximately upto 7 months
measure: Erythropoiesis
timeFrame: Approximately upto 7 months
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Cancer Center
state: California
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Sarasota
country: United States
facility: Florida Cancer Specialists
state: Florida
city: New Orleans
country: United States
facility: Tulane Comprehensive Cancer Center
state: Louisiana
city: Detroit
country: United States
facility: Barbara Ann Karmanos Cancer Hospital
state: Michigan
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Md Anderson Cancer Center
state: Texas
city: Nantes
country: France
facility: Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu
city: Pierre Benite
country: France
facility: Hospices Civils de Lyon Centre Hospitalier Lyon Sud
city: Villejuif
country: France
facility: Institut Gustave Roussy
city: Bologna
country: Italy
facility: L Azienda Ospedaliero-Universitaria Di Bologna Policlinico S. Orsola - Malpighi
city: Firenze
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi (Aouc)
city: Pavia
country: Italy
facility: Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo
city: Rozzano
country: Italy
facility: Irccs Istituto Clinico Humanitas
Study officials:
affiliation: Incyte Corporation
name: Ekatarine Asatiani, MD
role: STUDY_DIRECTOR
References: