A study to evaluate the safety and efficacy of Vactosertib and Imatinib in patients with advanced desmoid tumor (aggressive fibromatosis)

Not Applicable

Completed

• Conditions: Neoplasms

• Registration Number: KCT0003449
• Lead Sponsor: Yonsei University Health System, Severance Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Completion: 2024-03-26
• Last Update Posted: 27 May 2024

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 29

Inclusion Criteria

1. Histologically confirmed desmoid tumor (aggressive fibromatosis) not available for local treatment (surgical resection or radiation therapy)
2. Eastern Cooperative Oncology Group performance status of 0-1
3. Measurable lesion (RECIST-Response evaluation criteria criteria in solid tumors 1.1.)
4. Adequate laboratory findings
-Hb = 9.0 g/dL
-Absolute neutrophil count (ANC) = 1000 /µL
-Platelet = 75,000/ µL
-Total Bilirubin: = 1.5 × UNL (upper normal limit) (= 2 × UNL in patients with liver metastasis)
-Serum Creatinine
1) =1.5 X ULN OR
2) for > 1.5 X ULN : measured creatinine clearance (CL) >50 mL/min (by 24h urine test or Calculated creatinine clearance [CL] )
-AST(SGOT)/ALT(SGPT): = 3.0 × UNL or = 5.0 × UNL (in patients with liver metastasis)
-Alkaline Phosphatase (ALP): = 3.0 × UNL or = 5.0 × UNL (in patients with liver or bone metastasis)
5. All patients must be able to provide a newly acquired tumor biopsy during screening (preferred) or provide an available tumor sample taken =3 years prior to screening.
6. Subjects must have ejection fraction = 50% and no clinically significant valvular dysfunction

Exclusion Criteria

1. Previous TGF-ß inhibitor and/or imatinib exposed patient
2. Patient who has had chemotherapy, radiotherapy, or biological therapy within 2 weeks
3. Any unresolved chronic toxicity greater than CTC grade 2 from previous anticancer therapy.
4. Has an active infection requiring systemic therapy
5. Uncontrolled intercurrent illness, including symptomatic congestive heart failure (NYHA Class III/IV), uncontrolled hypertension (=150/90mmHg), unstable angina pectoris or myocardial infarction (= 6 months prior to screening), uncontrolled cardiac arrhythmia, cardiac valulopathy
6. Uncontrolled or active CNS metastasis and/or carcinomatous meningitis
7. Child-Pugh B or C liver cirrhosis
8. History of another primary malignancy.
9. Female patients who are pregnant or breastfeeding or male or female patients of reproductive potential who are not willing to employ effective birth control from screening to 90 days after the last dose of IP
10. Major surgical procedure (as defined by the Investigator) within 28 days prior to the first dose of IP.
11. Current or prior use of immunosuppressive medication within 14 days before the first IP.

Study & Design

• Study Type: Interventional Study
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Phase 1: To evaluate the safety and tolerability of vactosertib in combination with imatinib including estimation of the maximum tolerated dose (MTD) and/or characterization of DLTs.;Phase 2 : Clinical benefic rate

Secondary Outcome Measures

Name	Time	Method
Adverse event