Phase 2
N=208
A Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Cancers
Multiple Myeloma, Neoplasms
Bottom Line
View on ClinicalTrials.gov: NCT01524978 ↗Enrolled (actual)
208
Serious AEs
49.0%
Results posted
Nov 2017
Primary outcome: Primary: Confirmed Best Overall Response Rate (BORR) — 37.1; 50.0; 0; 7.4 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- cetuximab (Drug); vemurafenib (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Confirmed Best Overall Response Rate (BORR) |
37.1; 50.0; 0; 7.4; 22.2; 22.2 | — |
| SECONDARY Percentage of Participants With Confirmed Clinical Benefit |
48.4; 50.0; 0; 18.5; 44.4; 22.2 | — |
| SECONDARY Overall Response Rate (ORR) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Duration of Response (DOR) |
7.16; 8.16; NA; 6.54; 12.86; NA | — |
| SECONDARY Time to Response |
7.26; NA; NA; NA; NA; 5.75 | — |
| SECONDARY Time to Tumor Progression (TTP) |
7.33; 6.44; 3.88; 3.68; 3.02; 4.63 | — |
| SECONDARY Progression Free Survival (PFS) |
6.51; 6.44; 3.88; 3.68; 3.02; 4.63 | — |
| SECONDARY Overall Survival (OS) |
15.38; NA; 9.30; 7.16; 17.94; 24.54 | — |
| SECONDARY Maximum Tolerated Dose for Vemurafenib in Combination With Cetuximab |
960; 400 | — |
| SECONDARY Number of Dose-limiting Toxicities of Vemurafenib in Combination With Cetuximab |
1; 1 | — |
| SECONDARY Safety: Percentage of Participants With Adverse Event |
100; 100; 100; 100; 100; 100 | — |
Summary
This open-label, multi-center study will assess the efficacy and safety of vemurafenib in participants with BRAF V600 mutation-positive cancers (solid tumors and multiple myeloma, except melanoma and papillary thyroid cancer) and for whom vemurafenib is deemed the best treatment option in the opinion of the investigator. Participants will receive twice daily oral doses of 960 mg vemurafenib until disease progression, unacceptable toxicity, or withdrawal of consent. The safety and efficacy of vemurafenib in combination with cetuximab in a subset of participants with colorectal cancer will also be assessed.
Eligibility Criteria
Inclusion Criteria
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Must have recovered from all side effects of their most recent systemic or local treatment
- Adequate hematological, renal and liver function
For solid tumors only:
- Histologically confirmed cancers (excluding melanoma and papillary thyroid cancer) with a BRAF V600 mutation and that are resistant to standard therapy or for which standard or curative therapy does not exist
- Measurable disease according to Response Evaluation Criteria In Solid Tumors (RECIST)
For multiple myeloma only:
- Confirmed diagnosis of multiple myeloma with a BRAF V600 mutation
- Must have received at least one prior systemic therapy for the treatment of multiple myeloma
- Treated with local radiotherapy
- Must have relapsed and/or refractory multiple myeloma with measurable disease
Exclusion Criteria
- Melanoma, papillary thyroid cancer or hematological malignancies (with the exception of multiple myeloma)
- Uncontrolled concurrent malignancy
- Multiple myeloma: solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia
- Active or untreated central nervous system (CNS) metastases
- History of or known carcinomatous meningitis
- Concurrent administration of any anti-cancer therapies other than those administered in this study
- Other severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that would, in the investigator's opinion, contraindicate participation in this study
Data sourced from ClinicalTrials.gov (NCT01524978). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.