Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03761069 · ClinicalTrials.gov registry record · Phase 1
Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
A Phase 1 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 1
- Development phase
- 33
- Enrollment target
NCT03761069: Clinical Trial Phase 1 study, sponsored by PTC Therapeutics.
NCT03761069 is a Phase 1 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 33 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (45% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03761069, a Phase 1 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 33 participants
- Enrollment target
Study 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.
Interventions
- DRUG PTC299
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 33 participants |
| Start Date | 2018-10-29 |
| Est. Completion | 2021-12-28 |
| Phase | Phase 1 |
Why NCT03761069 stopped before completion
NCT03761069 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 33 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (45% lower).
The record links to 0 conditions, and to 1 intervention - of which PTC299 is the first listed.
NCT03761069 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03761069 about?
NCT03761069 is a clinical study titled "Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias". 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 stan...
What is the current status of trial NCT03761069?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 33 participants. The study started on 2018-10-29. Estimated completion is 2021-12-28.
What interventions are being tested in trial NCT03761069?
The interventions under investigation include: PTC299 (DRUG).
Who is sponsoring clinical trial NCT03761069?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03761069's enrollment target sits among peer trials
33 1280th of 2000 higher than 705 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02861898 · 33 participants · Phase 1
Super-selective Intra-arterial Repeated Infusion of Cetuximab for the Treatment of Newly Diagnosed Glioblastoma
- NCT04871607 · 33 participants · Phase 2
Yttrium-90 Labeled Anti-CD25 Monoclonal Antibody Combined With BEAM Chemotherapy Conditioning for the Treatment of Primary Refractory or Relapsed Hodgkin Lymphoma
- NCT05071937 · 33 participants · Phase 2
ZEN003694 Combined With Talazoparib in Patients With Recurrent Ovarian Cancer
- NCT05168618 · 33 participants · Phase 2
Cabozantinib and Atezolizumab for the Treatment of Metastatic Castration-Resistant Prostate Cancer, The AtezoCab Trial
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI