A Phase 1/1b Study of Pevonedistat in Combination With Select Standard of Care Agents in Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Acute Myelogenous Leukemia, or Advanced Solid Tumors With Severe Renal Impairment or Mild or Moderate Hepatic Impairment

Who is this study for? Patients with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, acute myelogenous leukemia, or advanced solid tumors with severe renal impairment or mild or moderate hepatic impairment
What treatments are being studied? Pevonedistat
Status: Completed
Location: See all (8) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

Pevonedistat is a medicine to treat people with blood cancers or solid tumors. The main aim of the study is to learn about the levels of pevonedistat in the blood of participants with blood cancers or solid tumors, who also have severe kidney problems or mild to moderate liver problems. The information from this study will be used to work out the best dose of pevonedistat to give people with these conditions in future studies. At the first visit, the study doctor will check who can take part in the study. This study is in 2 parts: A and B. Part A Participants will be placed into 1 of 4 treatment groups depending on how severe their kidney and liver problems are. All participants will receive 1 dose of pevonedistat as a slow injection in their vein (infusion). Then, the study doctors will check the levels of pevonedistat in the blood of the participants for 3 days after the infusion. They will also check if the participants have any side effects from pevonedistat. Participants will be asked to continue to Part B. Those who don't want to continue will visit the clinic 30 days later for a final check-up. Part B Participants who agree to participate into Part B will receive an infusion of pevonedistat on specific days during a 21-day or 28-day cycle. The cycle time will depend on what type of cancer the participants have. Participants will also be treated with standard of care medicines for their kidney and liver problems during this time. In the first cycle, the study doctors will also check the levels of pevonedistat in the blood and urine of participants for 3 days after the infusion. Participants will continue with cycles of treatment together with standard of care medicines until their condition gets worse or they have too many side effects from the treatment. When treatment has finished, participants will visit the clinic 10 days later for a final check-up.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• All participants:

• Has expected survival of at least 3 months from the date of enrollment in the study.

• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

• Has recovered (that is, Grade \<=1 toxicity) from the reversible effects of prior anticancer therapy.

• Prothrombin time (PT) and activated partial thromboplastin time (aPTT) \<=1.5 \* upper limit of the normal range (ULN) at screening or within 7 days before the first dose of study drug.

• Suitable venous access for the study-required blood sampling (that is, PK sampling).

• For hematologic malignancies:

• Previously untreated hematologic malignancies not suitable for induction therapy.

• Morphologically confirmed diagnosis of MDS or nonproliferative CMML (that is, with white blood cell \[WBC\] \<13,000 /mcL) at the study entry, based on one of the following:

• French-American-British (FAB) Classifications:

⁃ Refractory anemia with excess blasts (RAEB), defined as having 5% to 20% myeloblasts in the bone marrow.

⁃ CMML with 10% to 19% myeloblasts in the bone marrow and/or 5% to 19% blasts in the blood.

• OR

• World Health Organization (WHO) Classifications:

⁃ RAEB-1, defined as having 5% to 9% myeloblasts in the bone marrow.

⁃ RAEB-2, defined as having 10% to 19% myeloblasts in the bone marrow and/or 5% to 19% blasts in the blood.

⁃ CMML-2, defined as having 10% to 19% myeloblasts in the bone marrow and/or 5% to 19% blasts in the blood.

⁃ CMML-1 (although CMML-1 is defined as having \<10% myeloblasts in the bone marrow and/or \<5% blasts in the blood, these participants may enroll only if bone marrow blasts \>=5%).

• With MDS or CMML and must also have one of the following Prognostic Risk Categories, based on the Revised International Prognostic Scoring System (IPSS-R):

‣ Very high (\>6 points).

⁃ High (\>4.5-6 points).

⁃ Intermediate (\>3-4.5 points): a participant determined to be in the Intermediate Prognostic Risk Category is only allowable in the setting of \>=5% bone marrow myeloblasts.

• With WHO-defined AML at study entry, including leukemia secondary to prior chemotherapy or resulting from an antecedent hematologic disorder, have failed to achieve CR or have relapsed after prior therapy and are not candidates for potentially curative treatment.

⁃ With relapsed or refractory MDS, have previously been treated with an hypomethylating agent.

⁃ Laboratory value requirements per study arms are:

∙ Estimated glomerular filtration rate (eGFR) (milliliter per minute per 1.73 square meter \[mL/min/1.73\^m\]) \>=90 (Control arm), \<30 (Renal arm) , \>=60 (Mild and Moderate hepatic arm).

‣ Total Bilirubin \<= ULN (Control arm), \<= ULN (Renal arm), ULN \<Bilirubin \<=1.5 \* ULN (not secondary to transfusions) (Mild hepatic arm) and 1.5 \* ULN \<bilirubin \<=3.0 \* ULN (not secondary to transfusions) (Moderate hepatic arm).

‣ Alanine aminotransferase (ALT) \<= ULN (Control arm), \<=2.5 \* ULN (Renal arm) and any value (for mild and moderate hepatic arm).

⁃ For advanced solid tumors:

⁃ Have a histologically or cytologically confirmed metastatic or locally advanced solid tumor that is appropriate for treatment with pevonedistat in combination with either docetaxel or carboplatin plus paclitaxel in Part B of this study, or have progressed despite standard therapy, or whom conventional therapy is not considered effective.

⁃ Computerized tomography (CT) scan or magnetic resonance imaging (MRI) of the chest, abdomen, and pelvis within 28 days of the first dose of the study drug.

⁃ Laboratory value requirements per study arms are:

∙ eGFR (mL/min/1.73m\^2) \<30 (Renal arm) and \>=60 (mild and moderate hepatic arm).

‣ Total bilirubin \<=ULN (Renal arm), ULN \<bilirubin \<=1.5 \* ULN (Mild hepatic arm) and 1.5 \* ULN \<bilirubin \<=3.0 \* ULN (Moderate hepatic arm).

‣ ALT \<=1.5 \* ULN (for participants who receive pevonedistat plus docetaxel only) or \<=2.5 \* ULN (for participants who receive pevonedistat plus carboplatin plus paclitaxel only) (Renal arm) and any value (Mild and Moderate hepatic arm).

Locations
United States
North Carolina
University of North Carolina at Chapel Hill
Chapel Hill
New York
Icahn School of Medicine at Mount Sinai
New York
Other Locations
Spain
Hospital Universitario Vall d'Hebron - PPDS
Barcelona
Hospital de San Pedro de Alcantara
Caceres
C.H. Regional Reina Sofia
Cordoba
ICO lHospitalet Hospital Duran i Reynals
Lhospitalet De Llobregat
Complejo Asistencial Universitario de Salamanca H. Clinico
Salamanca
Hospital Universitario Virgen del Rocio - PPDS
Sevilla
Time Frame
Start Date: 2019-07-10
Completion Date: 2022-04-19
Participants
Target number of participants: 17
Treatments
Experimental: Control Arm
Pevonedistat 20 milligram per square meter (mg/m\^2), infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days, further followed by azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
Experimental: Renal Arm
Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days, further followed by azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8-9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 15 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
Experimental: Mild Hepatic Arm
Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days, further followed by azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
Sponsors
Leads: Takeda

This content was sourced from clinicaltrials.gov

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