Trials / Completed
CompletedNCT01154452
Vismodegib and Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced or Metastatic Sarcoma
A Phase 1B/II Study of GDC-0449 (NSC 747691) in Combination With RO4929097, a Gamma-Secretase Inhibitor (GSI) in Advanced/Metastatic Sarcomas
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 78 (actual)
- Sponsor
- National Cancer Institute (NCI) · NIH
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This randomized phase I/II clinical trial is studying the side effects and best dose of gamma-secretase/notch signalling pathway inhibitor RO4929097 when given together with vismodegib and to see how well they work in treating patients with advanced or metastatic sarcoma. Vismodegib may slow the growth of tumor cells. Gamma-secretase/notch signalling pathway inhibitor RO4929097 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving vismodegib together with gamma-secretase/notch signalling pathway inhibitor RO4929097 may be an effective treatment for sarcoma.
Detailed description
PRIMARY OBJECTIVES: I. To determine the maximum-tolerated dose (MTD) of gamma-secretase inhibitor RO4929097 (RO4929097) when given in combination with fixed-dose Hedgehog antagonist GDC-0449 (GDC-0449) which will become the recommended dose for the phase II portion of this study. (Phase Ib) II. To assess the progression-free survival (PFS) of the combination of RO4929097 with and without GDC-0449 in two arms of patients with advanced sarcoma. (Phase II) SECONDARY OBJECTIVES: I. To describe the tolerability and adverse event profile of daily GDC-0449 administered orally in combination with daily RO4929097 administered orally for 21 consecutive days. (Phase Ib) II. To describe the pharmacokinetics of the combination of the combination of GDC-0449 and RO4929097. (Phase Ib) III. To assess Response Evaluation Criteria In Solid Tumors (RECIST) 1.1 overall response rates (complete and partial response \[CR+PR\]) for combination therapy. (Phase Ib and II) IV. To conduct pharmacodynamic studies in tissue biopsies (pre- and post-study) for explorative and hypothesis-generating studies. (Phase Ib and II) V. To assess overall survival. (Phase II) VI. To further describe the pharmacokinetics and pharmacodynamics of the combination of GDC-0449 and RO4929097 at the phase II dose at the continuous schedule. (Phase II) V. To conduct pharmacodynamic studies in tissue biopsies (pre- and post- study drug\[s\]) for explorative and hypothesis generating studies. (Phase II) OUTLINE: This is a phase I, dose-escalation study of gamma-secretase/notch signalling pathway inhibitor RO4929097 followed by a phase II study. PHASE IB: PART A: Patients receive vismodegib orally (PO) once daily (QD) on days 1-21. PART B: Beginning within 7 days of finishing part A, patients receive vismodegib PO and gamma-secretase/notch signalling pathway inhibitor RO4929097 PO QD on days 1-21. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. PHASE II: Patients are randomized to 1 of 2 treatment arms. ARM I: Patients receive gamma-secretase/notch signalling pathway inhibitor RO4929097 PO QD on days 1-21. ARM II: Patients receive vismodegib PO and gamma-secretase/notch signalling pathway inhibitor RO4929097 PO QD on days 1-21. In both arms, courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 4 weeks.
Conditions
- Adult Alveolar Soft Part Sarcoma
- Adult Angiosarcoma
- Adult Desmoplastic Small Round Cell Tumor
- Adult Epithelioid Hemangioendothelioma
- Adult Epithelioid Sarcoma
- Adult Extraskeletal Myxoid Chondrosarcoma
- Adult Extraskeletal Osteosarcoma
- Adult Fibrosarcoma
- Adult Leiomyosarcoma
- Adult Liposarcoma
- Adult Malignant Mesenchymoma
- Adult Malignant Peripheral Nerve Sheath Tumor
- Adult Rhabdomyosarcoma
- Adult Synovial Sarcoma
- Adult Unclassified Pleomorphic Sarcoma
- Chondrosarcoma
- Clear Cell Sarcoma of the Kidney
- Conjunctival Kaposi Sarcoma
- Dermatofibrosarcoma Protuberans
- Gastrointestinal Stromal Tumor
- Metastatic Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Metastatic Osteosarcoma
- Ovarian Sarcoma
- Recurrent Adult Soft Tissue Sarcoma
- Recurrent Adult Unclassified Pleomorphic Sarcoma of Bone
- Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Recurrent Kaposi Sarcoma
- Recurrent Osteosarcoma
- Recurrent Uterine Corpus Sarcoma
- Small Intestine Leiomyosarcoma
- Stage III Adult Soft Tissue Sarcoma
- Stage III Uterine Sarcoma
- Stage IV Adult Soft Tissue Sarcoma
- Stage IV Uterine Sarcoma
- Unclassified Pleomorphic Sarcoma of Bone
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Gamma-Secretase Inhibitor RO4929097 | Given PO |
| OTHER | Laboratory Biomarker Analysis | Correlative studies |
| OTHER | Pharmacological Study | Correlative studies |
| DRUG | Vismodegib | Given PO |
Timeline
- Start date
- 2010-06-01
- Primary completion
- 2015-02-01
- Completion
- 2015-02-01
- First posted
- 2010-06-30
- Last updated
- 2018-08-15
- Results posted
- 2016-07-22
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT01154452. Inclusion in this directory is not an endorsement.