Study to Assess Safety and Efficacy of Vactosertib in Adolescents and Adults With Recurrent, Refractory or Progressive Osteosarcoma
- Registration Number
- NCT05588648
- Lead Sponsor
- MedPacto, Inc.
- Brief Summary
MP-VAC-209 is a Phase I/II, open label, single arm, multi-center study to assess safety, tolerability, and antitumor activity of vactosertib as a single agent in adolescents and adults with recurrent, refractory, or progressive osteosarcoma. Vactosertib is given orally, twice a day, to people 12 years of age and older who meet the criteria for study enrollment.
- Detailed Description
This is a Phase I/II, open label, single arm, proof of concept, multi-center (global) study to assess safety, tolerability, pharmacokinetics and antitumor activity of vactosertib as a Single Agent in Adolescents and Adults with Recurrent, Refractory or Progressive Osteosarcoma or bone lesions that have progressed, relapsed or are refractory to standard systemic therapy. Adolescents are defined as subjects aged 12 to \<18-year-old.
During the Phase I part of the trial, dose limiting toxicity will be used to decide dose escalation according to the standard 3+3 rules to determine the maximum tolerated dose (MTD) and the recommended phase II dose (RP2D) in adults and adolescents.
Dose escalation will be conducted sequentially and independently in adults and adolescents, respectively. Dose escalation in adolescents will not start before dose escalation in adults is completed. Data from the Phase I part of the trail in adults will be reviewed by the DSMB before opening the Phase II part. Data from the Phase I in adolescents will be reviewed by the DSMB before each dose (de-)escalation. As soon as the RP2D in adolescents is established, adolescents will be enrolled in Phase II.
During Phase II, subjects will be enrolled at the RP2D determined independently for adults and adolescents. Data of the subjects enrolled in the Phase II will be pooled with data of subjects enrolled at the same dose level during Phase I to evaluate the efficacy in a total of 42 adult and adolescent subjects.
In all parts of the study, the DSMB will convene upon occurrence of a grade 5 or any two sequentially occurring grade 4 drug related adverse events. In such case, accrual may not resume unless the DSMB has conducted a review and offered a recommendation to continue the study.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 48
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single Arm Vactosertib Single arm, open-label, no blinding or randomization procedure will be involved.
- Primary Outcome Measures
Name Time Method Dose finding associated with the overall nature and severity of AEs associated with treatment. Assessment of adverse events and laboratory abnormalities. through study completion, an average of 1 year Maximum tolerated dose (MTD) and recommended dose (RD)
- Secondary Outcome Measures
Name Time Method Overall survival. Acquiring the measures associated with phase I/phase II primary endpoints. Overall survival (1 year) calculated from date of enrollment to the dates of documented evidence of progression or death up to 12 months.
Time to response(TTR) Post two cycle of treatment, evaluation. through study completion, an average of 1 year. To evaluate the time to response(TTR) from Vactosertib treatment to show PR or CR. To characterize the pharmacokinetics(PK) of Vactosertib to 6 patients assigned to dose- escalation phase and the firsts 12 patients assigned to the dose expansion phase.
Trial Locations
- Locations (3)
UH Rainbow Babies & Children's Hospital
πΊπΈCleveland, Ohio, United States
National Cancer Center
π°π·Gyeonggi-do, Korea, Republic of
Korea Institute of Radiological & Medical Sciences
π°π·Seoul, Korea, Republic of