Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT03761069
Study type Interventional
Source PTC Therapeutics
Contact
Status Terminated
Phase Phase 1
Start date October 29, 2018
Completion date December 28, 2021

See also
  Status Clinical Trial Phase
Recruiting NCT05319587 - Study of Liposomal Annamycin in Combination With Cytarabine for the Treatment of Subjects With Acute Myeloid Leukemia (AML) Phase 1/Phase 2
Active, not recruiting NCT04090736 - Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy Phase 3
Completed NCT01617226 - Randomised Study of Azacitidine Versus Azacitidine With Vorinostat in Patients With AML or High Risk MDS Phase 2
Terminated NCT00916045 - Pilot Study of Unrelated Cord Blood Transplantation Phase 2
Terminated NCT00957580 - Trial of Pimasertib in Hematological Malignancies Phase 2
Completed NCT00640796 - Pilot Study of Expanded, Donor Natural Killer Cell Infusions for Refractory Non-B Lineage Hematologic Malignancies and Solid Tumors Phase 1
Completed NCT00458250 - Feasibility of Haploidentical Hematopoietic Stem Cell Transplantation Using CAMPATH-1H Phase 1
Active, not recruiting NCT05424380 - A Phase 1, Open Label Study of Intravenous GSK3745417 to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Determine RP2D & Schedule in Participants With Relapsed or Refractory Myeloid Malignancies Including AML and HR MDS Phase 1
Completed NCT01690624 - BI 836858 Dose Escalation in Patients With Refractory or Relapsed Acute Myeloid Leukemia and in Patients in Complete Remission With High Risk to Relapse Phase 1
Recruiting NCT05471700 - Single Arm Study of Azacitidine and Venetoclax for Treatment of Newly Diagnosed Fit Acute Myeloid Leukemia Patients Phase 1/Phase 2
Not yet recruiting NCT05016063 - Dual CD33-CLL1-CAR-T Cells in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia Early Phase 1
Not yet recruiting NCT04450784 - ObServatory Children Acute RElated Therapy Leukemia
Recruiting NCT04265963 - CD123-Targeted CAR-T Cell Therapy for Relapsed/Refractory Acute Myeloid Leukemia Phase 1/Phase 2
Terminated NCT04968860 - Oral Health Condition and Quality of Life in Children With Leukemia
Recruiting NCT03793517 - Decitabine Plus mBU/CY for High Risk Acute Leukemia With MRD Pre-HSCT Phase 2/Phase 3
Terminated NCT02841540 - A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes Phase 1
Recruiting NCT05453903 - A Study of JNJ-75276617 in Combination With Acute Myeloid Leukemia (AML) Directed Therapies Phase 1
Completed NCT03720366 - A Study of Perpetrator Drug Interactions of Enasidenib in AML Patients Phase 1
Withdrawn NCT04230564 - Acute Myeloid Leukemia Real World Treatment Patterns
Completed NCT01835587 - Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS). Phase 1/Phase 2