A Study to Evaluate the Safety and Efficacy of Vactosertib and Imatinib in Patients With Advanced Desmoid Tumor

NCT03802084 | Completed Phase 1

• 1 other identifier: 4-2018-0807
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase I/II, open-label, non-randomized, multicentre study to evaluate the clinical activity of vactosertib plus imatinib in desmoid tumor. Based on the background, TGF-β inhibition as a potential therapeutic target for desmoid tumor and convey significant implications for the clinical development. Therefore, investigator will conduct the phase II trial of vactosertib in combined with imatinib in desmoid tumor.

Conditions

Desmoid Tumor

Outcome Measures

Primary Outcomes (2)

• adverse event

  to evaluate the safety and tolerability

  4 weeks

• clinical benefit rate was determined using RECIST 1.1

  to evaluate antitumor activity by determining progression-free survival

  16weeks

Study Arms (1)

vactosertib/imatinib combination

EXPERIMENTAL

Drug: vactosertib/imatinib combination

Interventions

vactosertib/imatinib combination DRUG

Phase 1 : Imatinib 400mg QD P q28days Vactosertib 1 cohort 200mg bid (D1-5, 8-12, 15-19, 22-26) * 1 cohort 100mg bid Phase 2 : Imatinib 400mg QD PO q28days. Vactosertib RP2D, bid (D1-5, 8-12, 15-19, 22-26)

vactosertib/imatinib combination

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically confirmed desmoid tumor (aggressive fibromatosis) not available for local treatment (surgical resection or radiation therapy)
• Eastern Cooperative Oncology Group performance status of 0-1
• Measurable lesion (RECIST 1.1.)
• Patients with sufficient organ function according to laboratory findings
• Hemoglobin ≥ 9.0 g/dL
• Neutrophils ≥ 1000 /µL
• Platelets ≥ 75,000/µL
• Total Bilirubin ≤ 1.5 × UNL (upper normal limit): For patients with liver metastases, ≤2
• Serum creatinine ≤1.5 X ULN or \> 1.5 X Creatinine Clearance ≥50 mL/min for ULN patients (based on 24-hour urinalysis or Cockroft-Gault Formula calculations)
• AST(SGOT)/ALT(SGPT) ≤ 3.0 × UNL or ≤ 5.0 × UNL (for patients with liver or bone metastases)
• Alkaline Phosphatase (ALP): ≤ 3.0 × UNL or ≤ 5.0 × UNL (for patients with liver or bone metastases)
• 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.
• Subjects must have ejection fraction ≥ 50% and no clinically significant valvular dysfunction

You may not qualify if:

• Previous TGF-β inhibitor exposed patient
• Patient who has had chemotherapy, radiotherapy, or biological therapy within 2 weeks
• Any unresolved chronic toxicity greater than grade 2 from previous anticancer therapy.
• Has an active infection requiring systemic therapy
• 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
• Uncontrolled or active central nervous system metastasis and/or carcinomatous meningitis
• Child-Pugh B or C liver cirrhosis
• History of another primary malignancy.
• 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 investigational product(IP).
• Major surgical procedure (as defined by the Investigator) within 28 days prior to the first dose of investigational product(IP).
• Current or prior use of immunosuppressive medication within 14 days before the first investigational product(IP).

Sponsors & Collaborators

• Hyo Song Kim: lead

Study Sites (1)

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

MeSH Terms

Conditions

Desmoid Tumors

Interventions

vactosertib

Condition Hierarchy (Ancestors)

Fibroma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: professor

Study Record Dates

• First Submitted: January 7, 2019
• First Posted: January 14, 2019
• Study Start: April 15, 2019
• Primary Completion: March 26, 2024
• Study Completion: March 26, 2024
• Last Updated: May 13, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)