AzTrials
AzTrials

Relapsed Acute Myeloid Leukemia (AML)Phase 1NCT03787498

A Study of PLX2853 in Relapsed or Refractory Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

A Phase 1 study testing PLX2853 in people with Relapsed Acute Myeloid Leukemia (AML) and Refractory Acute Myeloid Leukemia (AML), run by Opna Bio LLC at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this research study is to evaluate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of the investigational drug PLX2853 in subjects with Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
PLX2853
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
6 sitesUnited States
Study dates
19 Mar 2019to 30 Jun 2021
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Confirmed diagnosis of one of the following myeloid malignancies, based on the 2016 revision of the World Health Organization classification:
  • Relapsed or refractory AML.
  • Subjects must have received no more than 3 prior induction therapies and have no standard therapeutic option that is expected to result in a clinical benefit.
  • Relapsed or refractory MDS.
  • Subjects must have high-risk disease (intermediate or greater disease according to the revised International Prognostic Scoring System [IPSS-R]).
  • Subjects must have received no more than 3 prior therapies, 1 of which must have included a hypomethylating agent such as azacytidine or decitabine.
Show all 20Show fewer
  • Subjects must have no standard therapeutic option that is expected to result in a clinical benefit.
  • Age ≥18 years.
  • Eastern Cooperative Oncology Group (ECOG) performance status of ≤2.
  • Life expectancy of ≥3 months in the judgment of the investigator.
  • Adequate renal, hepatic, and coagulation parameters:
  • Measured or calculated (Cockcroft-Gault formula) creatinine clearance (CrCl) ≥60 mL/min.
  • Total bilirubin ≤1.5 × ULN unless due to Gilbert's syndrome.
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 × ULN.
  • Prothrombin time or international normalized ratio ≤1.5 × ULN.
  • Activated partial thromboplastin time ≤1.5 × ULN.
  • Women of child-bearing potential must have a negative pregnancy test at Screening and must agree to use an effective form of contraception from the time of the negative pregnancy test to 90 days after the last dose of study drug. Effective forms of contraception include abstinence, hormonal contraception in conjunction with a barrier method, or a double barrier method. Women of non-child-bearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥ 1 year.
  • Fertile men must agree to use an effective method of birth control during the study and for 90 days after the last dose of study drug.
  • Resolution (to ≤Grade 1 or baseline) of all significant toxicity associated with prior cancer therapy prior to study drug initiation. (Grade 2 alopecia or residual Grade 2 peripheral neuropathy is allowed.)
  • Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.

Who cannot join

  • Prior treatment with a bromodomain inhibitor.
  • Any one of the following therapies:
  • Stem cell transplantation within 90 days of study drug initiation;
  • Active immunosuppressive therapy for graft-versus-host disease (GVHD);
  • GVHD prophylaxis within 2 weeks of study drug initiation.
  • Known uncontrolled fungal, bacterial, and/or viral infection ≥Grade 2.
Show all 24Show fewer
  • Uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia.
  • Active symptomatic central nervous system involvement of AML. (Individuals who have had leptomeningeal disease that was effectively treated are eligible.)
  • A diagnosis of acute promyelocytic leukemia or chronic myeloid leukemia in blast crisis.
  • Known or suspected allergy to the study drug or any agent given in association with this trial.
  • Women who are either pregnant or breast feeding.
  • Clinically significant cardiac disease.
  • Inability to take oral medication or significant nausea and vomiting, malabsorption, or significant small bowel resection that, in the opinion of the Investigator, would preclude adequate absorption.
  • Individuals who are known to be infected with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) or are known carriers of HBV or HCV. Individuals who are positive for HCV antibody must be negative for HCV RNA by polymerase chain reaction (PCR) to be eligible. Individuals with occult or prior HBV infection (defined as being seropositive for total hepatitis B core antibody and seronegative for hepatitis B surface antigen) may be included if HBV DNA is undetectable. These individuals must be willing to undergo additional testing per local standard of care.
  • Active second malignancy with the exception of any of the following:
  • Adequately treated basal cell carcinoma, squamous cell carcinoma of the skin, or in situ cervical cancer;
  • Adequately treated Stage I cancer from which the subject is currently in remission and has been in remission for ≥2 years;
  • Low-risk prostate cancer with Gleason score <7 and prostate-specific antigen <10 ng/mL;
  • Any other cancer from which the patient has been disease-free for ≥3 years.
  • Major surgery or significant injury within the 14-day period prior to study drug initiation.
  • Anti-cancer therapy in the period immediately preceding study drug initiation.
  • Any other medical, psychological, familial, sociologic, or geographic condition that, in the judgement of the investigator, would potentially hamper compliance with the study protocol or interfere with the study endpoints or the subject's ability to participate in the study.
  • Participation in any other therapeutic clinical study. (Participation in observational or registry trials is allowed.)
  • Individuals who are on active anticoagulation therapy (e.g., warfarin, factor Xa inhibitors, thrombin inhibitors, heparin).

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Northside Hospital

    Atlanta, Georgia, 30342

  • Sidney Kimmel Comprehensive Cancer At Johns Hopkins

    Baltimore, Maryland, 27287

  • Ohio State University Medical Center

    Columbus, Ohio, 43210

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • NewYork-Presbyterian / Weill Cornell Medical Center

    New York, New York, 10065

  • Oregon Health and Sciences University

    Portland, Oregon, 97239

Other trials in United States

About this study

Official title: A Phase 1b Dose-escalation Study to Assess the Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of PLX2853 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome

The purpose of this research study is to evaluate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of the investigational drug PLX2853 in subjects with Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome

Being tested
PLX2853
Keywords
PLX2853, Acute Myeloid Leukemia, AML, Myelodysplastic Syndrome, MDS, Hematologic Malignancy, Blood Cancer
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Hematologic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PLX2853Drug

    Tablets

Study groups in NCT03787498
Study groupRoleWhat they receive
PLX2853ExperimentalApproximately 30 subjects will be enrolled as part of dose escalation to identify the MTD/RP2D of PLX2853. Up to 6 additional subjects may be enrolled at the MTD/RP2D to further characterize the PK and PDy of PLX2853.
Receives: Drug: PLX2853

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

    Measured: First dose of study drug through at least 30 days after end of treatment

  2. Area under the concentration-time curve (AUC) of PLX2853

    Measured: From first dose of PLX2853 up to 30 days after end of treatment

  3. Maximum observed concentration (Cmax) of PLX2853

    Measured: From first dose of PLX2853 up to 30 days after end of treatment

  4. Time to peak concentration (Tmax) of PLX2853

    Measured: From first dose of PLX2853 up to 30 days after end of treatment

  5. Half life (t1/2) of PLX2853

    Measured: From first dose of PLX2853 up to 30 days after end of treatment

  6. Terminal elimination rate constant (Kel)

    Measured: From first dose of PLX2853 up to 30 days after end of treatment

  7. Number of participants who experience dose limiting toxicity as defined in the protocol

    Dose escalation will be guided by a modified continuous reassessment method (mCRM) using a Bayesian logistic regression model that follows the escalation with overdose control (EWOC) principle. In this method, a decision to escalate to the next dose level is based on a review of all subjects who have completed the DLT observation period.

    Measured: up to 18 months

Secondary outcomes (6)

  1. Overall complete remission (OCR) rate

    AML - Complete Remission (CR) + CR with incomplete hematological recovery (CRi); MDS - CR

    Measured: From the first dose of study drug until the date of documented best response to treatment, assessed up to 18 months

  2. Overall response rate (ORR)

    AML - Complete Remission (CR) + CR with incomplete hematologic recovery (CRi) + Partial Remission (PR); MDS - CR + PR

    Measured: From the first dose of study drug until the date of documented response to treatment, assessed up to 18 months

  3. Duration of response (DOR)

    Measured: DOR defined as the time from the initial objective response to disease progression or death, whichever occurs first, assessed up to 18 months

  4. Event-free survival (EFS)

    Measured: EFS time is defined as the time from the first dose of PLX2853 to treatment failure, relapse after initial response or death from any cause, assessed up to 18 months.

  5. Progression-free survival (PFS)

    Measured: PFS time is defined as the time from the first dose of PLX2853 to disease progression or death, whichever occurs first, assessed up to 18 months.

  6. Overall survival (OS)

    Measured: From the first dose of study drug until the date of death from any cause, assessed up to 18 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Dec 2018
  2. Study start19 Mar 2019
  3. Primary completion30 Jun 2021
  4. Study completion30 Jun 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03787498
Sponsor's protocol ID
PLX124-02
Status last verified
Apr 2022
First submitted
23 Dec 2018
Last updated
12 Apr 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing PLX2853 in people with Relapsed Acute Myeloid Leukemia (AML) and Refractory Acute Myeloid Leukemia (AML), run by Opna Bio LLC at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 (time frame: First dose of study drug through at least 30 days after end of treatment). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03787498.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in