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Terminated Phase 1 Interventional

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

ClinicalTrials.gov ID: NCT03761069

Public ClinicalTrials.gov record NCT03761069. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 5, 2026, 2:43 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03761069
Recruitment status
Terminated
Study type
Interventional
Phase
Phase 1
Lead sponsor
PTC Therapeutics
Industry
Enrollment
33 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
18 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Oct 28, 2018
Primary completion
Dec 27, 2021
Completion
Dec 27, 2021
Last update posted
Feb 1, 2022

2018 – 2021

United States locations

U.S. sites
17
U.S. states
11
U.S. cities
17
Facility City State ZIP Site status
Rocky Mountain Cancer Center Aurora Colorado 80012
Yale University New Haven Connecticut 06510
Henry Ford Health System Detroit Michigan 48202
Rutgers, Cancer Institute of NJ New Brunswick New Jersey 08903
Columbia New York New York 10032
University of Rochester MC Rochester New York 14642
Duke Cancer Center Durham North Carolina 27710
Gabrail Cancer Center Canton Ohio 44718
Oncology Hematology Care, Inc. Cincinnati Ohio 45236
Cleveland Clinic Foundation Cleveland Ohio 44195
Rhode Island, Miriam Hospital Providence Rhode Island 02903
SCRI Tennessee Oncology Nashville Tennessee 37203
Texas Oncology, P.A. Austin Texas 78705
Texas Oncology, P.A. Fort Worth Texas 76104
MD Anderson Cancer Center Houston Texas 77030
Texas Oncology, P.A. - San Antonio Medical Center San Antonio Texas 78240
Swedish Cancer Institute Seattle Washington 91804

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03761069, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Feb 1, 2022 · Synced Sep 5, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03761069 live on ClinicalTrials.gov.

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