OsteosarcomaPhase 1/2NCT05588648
Study to Assess Safety and Efficacy of Vactosertib in Adolescents and Adults With Recurrent, Refractory or Progressive Osteosarcoma
A Phase 1/2 study testing Vactosertib in people with Osteosarcoma, run by MedPacto, Inc. at 7 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Osteosarcoma
- What is being tested?
- Vactosertib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 1 May 2023to Feb 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects must meet all the following inclusion criteria to be eligible for enrollment:
- Informed Consent/Assent Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Age ≥12 years at the time of screening
- Type of Subject and Disease Characteristics
- Subjects may be male or female, with adolescents aged 12 to 17 years and adults aged 18 years or older, as defined by applicable country-specific legal age classifications. No large studies have evaluated the use of vactosertib in younger pediatric subjects, for this reason, children younger than 12 years of age are excluded from this study.
- Subjects must have clinical, radiologic, and histologic confirmation of Osteosarcoma (OSa), which must be documented.
Show all 16Show fewer
- Subjects must have measurable disease according to RECIST 1.1, diagnosed as recurrent, refractory or progressive osteosarcoma, and must have no available standard therapy.
- Subjects must have recovered from the acute toxic effects with ≤ Grade 1 as defined by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 of all prior chemotherapy and immunotherapy with the exception of alopecia, anorexia, bone pain, and tumor pain prior to entering this study.
- Myelosuppressive chemotherapy: Must have adequate recovery of counts from previous treatment prior to entry onto this study.
- Inclusion criteria include adequate renal function, ECOG performance status 0-2, and either Lansky performance status of 50-100% (<16 years old), or Karnofsky performance status 50-100% (≥16 years old). Subjects who are unable to walk because of paralysis, but who are up in a wheelchair will be considered ambulatory for the purpose of assessing the performance score.
- Subjects must have normal organ and marrow function as defined below:
- Adequate bone marrow function defined as: i. Peripheral absolute neutrophil count (ANC) ≥ 750/mcL ii. Platelet count ≥ 75,000/mcL (transfusion independent) iii. Hemoglobin ≥ 8.0 g/dL (may receive packed red blood cell transfusions) b. Adequate liver function defined as: i. Total bilirubin ≤ 1.5 times the upper limit of normal for age ii. AST (SGOT) and ALT (SGPT) 2.5 X institutional upper limit of normal iii. Albumin (serum or plasma) > 2 g/dL c. Adequate cardiac function defined as: i. Ejection fraction of ≥ 50% by echocardiogram or MUGA
- Subjects must have the ability to understand and the willingness to sign a written informed consent document if ≥ 18 years of age and an assent document if < 18 years of age (per country).
- Renal laboratory inclusion:
- Relapsed osteosarcoma (first, second, third or any subsequent relapse, subject who have recovered from chemotherapy and any other investigational product/agent treatment, radiotherapy or surgical procedure), with histological confirmed diagnosis of osteosarcoma at original presentation, and progressive disease documented by imaging within 3 months of entry into the trial.
- Subjects with a life expectancy of at least 3 months.
Who cannot join
- The subject must be excluded from participating in the trial if:
- Subjects who have moderate or severe cardiovascular disease
- Subjects who have uncontrolled intercurrent illness, including but not limited to, ongoing or active infection requiring systemic therapy, symptomatic congestive heart failure (New York Heart Association Class III/IV), uncontrolled hypertension (≥150/90mmHg), unstable angina pectoris or myocardial infarction (≤ 6 months prior to screening), uncontrolled cardiac arrhythmia, clinically significant cardiac valvulopathy requiring treatment, serious chronic gastrointestinal conditions associated with diarrhea, or psychiatric illness/social situations that would limit compliance with study requirement, substantially increase risk of incurring AEs or compromise the ability of the subject to give written informed consent
- Subjects who have major abnormalities at the Investigator's discretion based on electrocardiogram (ECG)and Doppler ECHO and MUGA results at screening or within 14 days before screening. QT interval corrected for heart rate using Fridericia's formula (QTcF) ≥470 ms in both male and female calculated from 12-lead ECGs.
- Subjects who have increase in brain natriuretic peptide (BNP) or increase in troponin (over 99th percentile upper reference limit) at Screening (based on the normal range of relevant study center)
- Subjects who have risk factors for ascending aortic aneurysm such as genetic disorder and trauma and risk factors for aortic stenosis
Show all 27Show fewer
- Subjects who have a history of heart or aorta surgery
- Subjects who have clinically significant gastrointestinal bleeding within 4 weeks before screening
- Subjects who have a known history or suspected hypersensitivity to any excipients of the investigational product.
- Subjects who have received prior treatment targeting the signaling pathway of TGF-β
- Subjects who are currently using or planning to use prohibited medications with vactosertib. For these medications, a washout period equivalent to at least 5 half-lives prior to the first dose of the investigational product is recommended.
- Drugs that are exclusively or primarily eliminated by cytochrome P-450 isozyme (CYP) including CYP1A2, CYP2D6, CYP2B6, or CYP3A4 (Concurrent use of drugs that are known potent CYP3A4 inducers including but not limited to Phenytoin, Rifampin, and St. John's wort. Concurrent use of foods that are known strong CYP3A4 inhibitors including but not limited to grapefruit juice, Itraconazole, Ketoconazole, Lopinavir/ritonavir, Mibefradil, and Voriconazole. The topical use of these medications (if applicable), such as 2% ketoconazole cream, may be allowed.)
- Drugs that are exclusively or primarily eliminated by UDP glucuronyltransferase (UGT) 1A1 (UGT1A1)
- Drugs that are substrates for the drug transporter multidrug resistance protein 1 (MDR1) have a narrow therapeutic window or are strong inhibitors of drug transporter MDR1
- Subjects who are unable to swallow tablets
- Subjects who have a history of or are suspected of drug abuse
- Female subjects of child-bearing potential who have a positive result on a pregnancy test at screening or are unable to agree to use an effective barrier method of birth control to avoid pregnancy during the study period (e.g., sterilization, intrauterine contraceptive device, combination of oral contraception and barrier contraception, combination of other hormone delivery systems and barrier contraception, contraceptive cream, combination of cream, jelly, or form and diaphragm or condom). Male subjects of reproductive potential must agree to use an adequate method of contraception starting with the first dose of study therapy through 90 days after the last dose of study therapy.
- Subjects, in the opinion of the Investigator, who are unsuitable to participate in the study
- Subjects who have received an investigational product or used an investigational medical device within 28 days prior to screening, or within 5 half-lives of the investigational product (whichever is longer), or who are currently participating in another clinical trial.
- Note: Subjects in follow-up phase of a previous clinical trial may be enrolled in this study if more than 4 weeks have passed since the last administration.
- Subjects who have not completed an adequate washout period prior to the first dosing of the investigational product will be excluded (< 3 weeks for chemotherapy, < 6 weeks for curative radiotherapy, or < 2 weeks for palliative radiotherapy)
- Subjects who are severely underweight and, in the opinion of the investigator, unsuitable for study treatment, according to country-specific criteria.
- Subjects with a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
- HIV-positive subjects including those receiving antiretroviral therapy, are ineligible because of the risk for developing a lethal infection when treated with immunosuppressive therapy.
- Subject with active or acute infection confirmed at screening.
- Non-metastatic osteosarcoma (OSa) for whom standard therapy is available at the time of the study may be excluded.
- Subject with chronic use of corticosteroids or other immunosuppressive agents. Note: High dose is defined as a prescription of >5 mg oral prednisolone and long term as duration of treatment >1 month (based on NICE. Corticosteroids - oral (accessed Apr 2017).
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 2 countries
South Korea6 sites, 6 recruiting
- Recruiting
National Cancer Center
Gyeonggi-do
Lee Jun-ah
- Recruiting
Seoul National University Bundang Hospital
Gyeonggi-do
Hyoung Soo Choi
- Recruiting
Korea Institute of Radiological & Medical Sciences
Seoul
Wanhyung Cho
- Recruiting
Samsung Medical Center
Seoul
Jiwon Lee
- Recruiting
Seoul Asan Medical Center
Seoul
Kyung-Nam Koh
- Recruiting
Seoul National University Hospital
Seoul
Hyoung Jin Kang
United States1 site, 1 recruiting
- Recruiting
UH Rainbow Babies & Children's Hospital
Cleveland, Ohio, 44106
Kristen VanHeyst, DO, (216) 844-3345, Kristen.VanHeyst@UHhospitals.org
Kristen VanHeyst, DO
About this study
Official title: A Phase I/II, Open-label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of Vactosertib as a Single Agent in Adolescents and Adults With Recurrent, Refractory or Progressive Osteosarcoma
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.
Read the 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.
- Conditions
- Osteosarcoma
- Being tested
- Vactosertib
- Medical subject headings
- Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VactosertibDrug
Also called TEW-7197
Vactosertib is given twice a day, five days on and two days off in four-week cycles. Vactosertib is a transforming growth factor-beta (TGF-β) type 1 receptor inhibitor.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Single arm, open-label, no blinding or randomization procedure will be involved. Receives: Drug: Vactosertib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose finding associated with the overall nature and severity of AEs associated with treatment.
For phase 1, Maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) : incidence of DLT and adverse events, Changes from baseline in Physical Exam, Vital Signs, local laboratory and ECGs,
For phase 2, Antitumor activity of Vactosertib and PK analysis : ORR, PFS, DCR, DOR, TTP, TTR and OS, PK parameters
Measured: from baseline to study completion(up to 60 months)
Secondary outcomes (2)
Time to response(TTR)
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.
Measured: Post two cycle of treatment, evaluation. through study completion, an average of 1 year. (up to 60 months)
Overall survival.
Overall survival (1 year) calculated from date of enrollment to the dates of documented evidence of progression or death up to 12 months.
Measured: Acquiring the measures associated with phase I/phase II primary endpoints.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2022
- Study start1 May 2023
- Primary completionDec 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- MedPacto, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05588648
- Sponsor's protocol ID
- MP-VAC-209
- Status last verified
- Oct 2026
- First submitted
- 14 Sep 2022
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Open-label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of Vactosertib as a Single Agent in Adolescents and Adults With Recurrent, Refractory or Progressive Osteosarcoma
- ClinicalTrials.gov ID
- NCT05588648
- Acronym / sponsor ID
- MP-VAC-209
- Conditions
- Osteosarcoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- MedPacto, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 May 2023 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 20 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: VactosertibSingle arm, open-label, no blinding or randomization procedure will be involved. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Informed Consent/Assent Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Age ≥12 years at the time of screening
- Type of Subject and Disease Characteristics
- Subjects may be male or female, with adolescents aged 12 to 17 years and adults aged 18 years or older, as defined by applicable country-specific legal age classifications. No large studies have evaluated the use of vactosertib in younger pediatric subjects, for this reason, children younger than 12 years of age are excluded from this study.
- Subjects must have clinical, radiologic, and histologic confirmation of Osteosarcoma (OSa), which must be documented.
Key exclusion criteria (27 listed)
- Subjects who have moderate or severe cardiovascular disease
- Subjects who have clinically significant gastrointestinal bleeding within 4 weeks before screening
- Subjects who have a known history or suspected hypersensitivity to any excipients of the investigational product.
- Subjects who have received prior treatment targeting the signaling pathway of TGF-β
- Subjects who are currently using or planning to use prohibited medications with vactosertib. For these medications, a washout period equivalent to at least 5 half-lives prior to the first dose of the investigational product is recommended.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VactosertibAlso: TEW-7197Vactosertib is given twice a day, five days on and two days off in four-week cycles. Vactosertib is a transforming growth factor-beta (TGF-β) type 1 receptor inhibitor. | Drug | Single Arm |
Primary outcome
Dose finding associated with the overall nature and severity of AEs associated with treatment.
Time frame: from baseline to study completion(up to 60 months)
For phase 1, Maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) : incidence of DLT and adverse events, Changes from baseline in Physical Exam, Vital Signs, local laboratory and ECGs, For phase 2, Antitumor activity of Vactosertib and PK analysis : ORR, PFS, DCR, DOR, TTP, TTR and OS, PK parameters
2 secondary outcomes
Time to response(TTR)
Time frame: Post two cycle of treatment, evaluation. through study completion, an average of 1 year. (up to 60 months)
Overall survival.
Time frame: Acquiring the measures associated with phase I/phase II primary endpoints.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Osteosarcoma
RecruitingPhase 1/2
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma
RecruitingObservational study
Genetic Analysis of Pheochromocytomas, Paragangliomas and Associated Conditions
Pheochromocytoma, Paraganglioma
RecruitingPhase 2
Iscador® P (Mistletoe) Immunotherapy for Recurrent Resectable Osteogenic Sarcoma
Osteogenic Sarcoma Recurrent
RecruitingPhase 2
The CATSINDO Trial - Clinical and Translational Study in Newly Diagnosed Osteosarcoma
Osteosarcoma in Children, Osteosarcoma in Adolescents and Young Adults
RecruitingPhase 2
Evaluating Efficacy of B7-H3-CAR T Cells Administered at the End of Upfront Map Chemotherapy in Patients With Newly Diagnosed High-Risk Osteosarcoma
Pediatric Osteosarcoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vactosertib in people with Osteosarcoma, run by MedPacto, Inc. at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in South Korea, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kristen VanHeyst, DO ((216) 844-3345, Kristen.VanHeyst@UHhospitals.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose finding associated with the overall nature and severity of AEs associated with treatment. (time frame: from baseline to study completion(up to 60 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026.