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Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias | Clinical Trials | Clareo Health

TERMINATEDPHASE1

Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias

Phase 1B Study of PTC299 in Relapsed/Refractory Acute Leukemias

NCT03761069•PTC Therapeutics

Summary

This is an open-label, non-randomized, Phase 1b study to evaluate the safety, pharmacokinetics (PK) profiles, and preliminary evidence of antitumor activity of PTC299 and the metabolite, O-desmethyl PTC299, in participants with relapsed/refractory acute myeloid leukemia (AML) who have exhausted standard available therapies known to provide clinical benefit. The study is designed as a series of cohort-based dose escalations. For each cohort, a minimum of 3 evaluable participants with PK and safety data will be assessed. Additional participants will be recruited if additional PK data are needed to assess mean exposure based on the observed variability.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Participant must have relapsed/refractory AML and exhausted standard available therapies known to provide clinical benefit.
•Subjects must be greater than or equal to 18 years of age.
•Subjects must have Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to (≤) 2
•Women of childbearing potential must be willing to practice a highly-effective method of birth control for up to 50 days after the last dose of study drug.
•A man who is sexually active with a woman of childbearing potential and has not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug.
•Subjects must be willing to participate to the study, have the ability to understand and adhere to study visit schedule and other protocol procedures, and be able and willing to sign a written informed consent form.

Exclusion Criteria

•Medical history:
•Women who are or plan to become pregnant, or who are currently breastfeeding.
•Persistence of any clinically relevant (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 2 or above) toxicities from previous therapy.
•Active alcohol or drug abuse.
•Previous drug-induced liver injury.
•Cardiac assessments:
•Uncontrolled congestive heart failure, unstable angina pectoris.
•History or current evidence of a myocardial infarction during the last 6 months.
•QTc prolongation greater than (\>) 500 milliseconds (msec) (Fridericia formula).
•Congenitally long QT syndrome or has received any marketed or experimental compound in the last 4 weeks or 5 half-lives (whichever is shorter) prior to entering the study with possible or known effects of QT prolongation. (If equivalent medication is not available, QTc will be closely monitored.)
+20 more criteria

Locations (17)

Rocky Mountain Cancer Center

Aurora, Colorado, United States

Yale University

New Haven, Connecticut, United States

Henry Ford Health System

Detroit, Michigan, United States

Rutgers, Cancer Institute of NJ

New Brunswick, New Jersey, United States

Columbia

New York, New York, United States

University of Rochester MC

Rochester, New York, United States

Duke Cancer Center

Durham, North Carolina, United States

Gabrail Cancer Center

Canton, Ohio, United States

Oncology Hematology Care, Inc.

Cincinnati, Ohio, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Key Dates

Start Date

October 29, 2018

Primary Completion Date

December 28, 2021

Completion Date

December 28, 2021

Last Updated

February 2, 2022

Enrollment

ACTUAL participants

Conditions

Leukemia, Myeloid, AcuteAML

Interventions

PTC299

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 2, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias

Inclusion Criteria

Exclusion Criteria

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

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