NCT05107856

Brief Summary

This is a Phase 1 dose-escalation study of PRT1419, a myeloid cell leukemia-1 (MCL-1) inhibitor, in participants with selected relapsed/refractory myeloid or B-cell malignancies. The purpose of this study is to evaluate the safety and tolerability of PRT1419 monotherapy and in combination with either azacitidine or venetoclax, describe any dose limiting toxicities (DLTs), define the dosing schedule, and to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Mar 2022

Geographic Reach
1 country

9 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 25, 2021

Completed
10 days until next milestone

First Posted

Study publicly available on registry

November 4, 2021

Completed
5 months until next milestone

Study Start

First participant enrolled

March 22, 2022

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 19, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 19, 2024

Completed
Last Updated

January 31, 2024

Status Verified

January 1, 2024

Enrollment Period

1.8 years

First QC Date

October 25, 2021

Last Update Submit

January 29, 2024

Conditions

Keywords

Acute Myeloid Leukemia (AML)AzacitidineB-cell MalignanciesB-cell Non-Hodgkin lymphomaChronic lymphocytic leukemia (CLL)Chronic Myelomonocytic Leukemia (CMML)Follicular LymphomaHematologic MalignanciesMantle Cell LymphomaMarginal Zone LymphomaMyelodysplastic Syndromes (MDS)Myeloid cell leukemia-1 (MCL-1)Myeloproliferative Neoplasm (MPN)PRT1419Relapsed/Refractory MyeloidSmall lymphocytic lymphoma (SLL)Venetoclax

Outcome Measures

Primary Outcomes (3)

  • Dose limiting toxicities (DLT) of PRT1419

    Dose limiting toxicities will be evaluated over the 28-day observation period

    Baseline through Day 28

  • Safety and tolerability of PRT1419: AEs, SAEs, CTCAE assessments

    Safety and tolerability will be assessed by recording adverse events (AEs), serious adverse events (SAEs), and DLTs according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)

    Baseline through approximately 3 years

  • Maximum tolerated dose (MTD)/Recommended phase 2 dose (RP2D) and schedule of PRT1419

    The MTD/RP2D will be established for further investigation in participants with advanced hematologic malignancies

    Baseline through approximately 2 years

Secondary Outcomes (8)

  • Pharmacokinetic profile of PRT1419 monotherapy and in combination with AZA or VEN: maximum observed plasma concentration

    Baseline through approximately 3.5 years

  • Pharmacokinetic profile of PRT1419 monotherapy and in combination with AZA or VEN: Time to maximal plasma concentration

    Baseline through approximately 3.5 years

  • Pharmacokinetic profile of PRT1419 monotherapy and in combination with AZA or VEN: Area under the curve

    Baseline through approximately 3.5 years

  • Safety and tolerability of PRT1419 in combination with AZA and VEN: AEs, SAEs, CTCAE assessments

    Baseline through approximately 3 years

  • Anti-tumor activity of PRT1419 monotherapy and in combination with AZA and VEN: Overall response rate (ORR)

    Baseline through approximately 3.5 years

  • +3 more secondary outcomes

Study Arms (3)

PRT1419 Monotherapy

EXPERIMENTAL

PRT1419 will be administered by intravenous infusion once weekly on a 28-day treatment cycle at the dose level assigned.

Drug: PRT1419

PRT1419/Azacitidine Combination

EXPERIMENTAL

PRT1419 will be administered by intravenous infusion once weekly on a 28-day treatment cycle at the dose level assigned and Azacitidine will be administered by intravenous or subcutaneous on Days 1 through 7 (or alternatively on Days 1 through 5, 8 and 9) of each 28-day treatment cycle.

Drug: PRT1419Drug: Azacitidine

PRT1419/Venetoclax Combination

EXPERIMENTAL

PRT1419 will be administered by intravenous infusion once weekly on a 28-day treatment cycle at the dose level assigned and Venetoclax will be administered orally after either a 3-day or 5-week ramp-up period to reach 400 mg daily administration, prior to commencing PRT1419 administration.

Drug: PRT1419Drug: Venetoclax

Interventions

PRT1419 will be administered by intravenous infusion

PRT1419 MonotherapyPRT1419/Azacitidine CombinationPRT1419/Venetoclax Combination

Azacitidine will be administered by intravenous or subcutaneous

PRT1419/Azacitidine Combination

Venetoclax will be administered orally

PRT1419/Venetoclax Combination

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations (including contraception requirements), and other study procedures
  • Refractory/relapsed disease, having progressed on prior treatment, and without access to further approved therapies or ineligible for approved therapies, in one of the following disease categories: AML, CMML, MDS, MDS/MPN Overlap Syndrome, CLL/SLL, and B-cell NHLs
  • Eastern Cooperative Oncology Group (ECOG) Performance Score of 0 to 2
  • Adequate organ function (hematology, hepatic, renal, and coagulation)

You may not qualify if:

  • Active inflammatory disorders of the gastrointestinal tract, a history of bariatric surgery or other disorders with the potential for GI malabsorption
  • Cardiac function compromise, as assessed by echocardiogram or protocol-specified biochemical markers of cardiac damage, or protocol-defined clinically significant heart disease
  • History of cerebrovascular accident or transient ischemic attack, within 6 months of screening. Participants with a history of pulmonary embolism must not be symptomatic at enrollment
  • Undergone hematopoietic stem-cell transplantation (HSCT) within the last 90 days or have graft-versus-host disease (GVHD) Grade \> 1 at study entry
  • Uncontrolled intercurrent illnesses, poorly controlled hypertension or dyslipidemias, Unstable central nervous system (CNS) metastases
  • Treatment with either OATP1B1, OATP1B3 substrates or strong inhibitors of CYP2C8, CYP3A4, and any medication contraindicated in combination with AZA or VEN
  • Prior exposure to an MCL-1 inhibitor
  • Within 5 half-lives or 14 days (whichever is longer) following the last systemic anti-cancer therapy
  • History of another malignancy except for:
  • Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease
  • Adequately treated cervical or breast carcinoma in situ without evidence of disease
  • Asymptomatic prostate cancer without known metastatic disease and no requirement for therapy or requiring only hormonal therapy and with normal prostate specific antigen for \>1 year prior to enrollment
  • Other malignancy treated with curative intent with no known active disease for \> 2 years prior to enrollment

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Mid Florida Hematology and Oncology Center

Orange City, Florida, 32763, United States

Location

AdventHealth Bone and Marrow Transplant Center

Orlando, Florida, 32804, United States

Location

American Oncology Partners of Maryland, PA

Bethesda, Maryland, 20817, United States

Location

New Jersey Center for Cancer Research

Brick, New Jersey, 08724, United States

Location

Memorial Sloan Kettering Cancer Center - Main Campus

New York, New York, 10065, United States

Location

North Shore Hematology Oncology Associates. DBA New York Cancer and Blood Specialists

Port Jefferson Station, New York, 11776, United States

Location

Gabrail Cancer Center Research

Canton, Ohio, 44718, United States

Location

Thomas Jefferson University, Sidney Kimmel Cancer Center

Philadelphia, Pennsylvania, 19107, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteLymphoma, B-CellLeukemia, Lymphocytic, Chronic, B-CellLeukemia, Myelomonocytic, ChronicLymphoma, FollicularLymphoma, Mantle-CellLymphoma, B-Cell, Marginal ZoneMyelodysplastic SyndromesMyeloproliferative DisordersHematologic NeoplasmsRecurrence

Interventions

Azacitidinevenetoclax

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoma, Non-HodgkinLymphomaLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, B-CellLeukemia, LymphoidChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsMyelodysplastic-Myeloproliferative DiseasesBone Marrow DiseasesNeoplasms by Site

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 25, 2021

First Posted

November 4, 2021

Study Start

March 22, 2022

Primary Completion

January 19, 2024

Study Completion

January 19, 2024

Last Updated

January 31, 2024

Record last verified: 2024-01

Locations