Ignite Creation Date:
2025-12-24 @ 10:57 PM
Ignite Modification Date:
2025-12-25 @ 8:25 PM
Study NCT ID:
NCT03761069
Status:
TERMINATED
Last Update Posted:
2022-02-02
First Post:
2018-11-29
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
Sponsor:
PTC Therapeutics
Organization:
Study Overview
Official Title:
Phase 1B Study of PTC299 in Relapsed/Refractory Acute Leukemias
Status:
TERMINATED
Status Verified Date:
2022-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Sponsor terminated the study.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: