Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Participants With Previously Treated Solid Tumors

Completed · Phase 1/Phase 2

Conditions studied: Solid Tumors

In brief

This open-label, dose-escalation study is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of cobimetinib in pediatric and young adult participants with solid tumors with known or potential kinase pathway activation for which standard therapy has proven to be ineffective or intolerable or for which no curative standard-of-care treatment options exist. The study will be conducted in two stages: a dose-escalation stage and an expansion stage at the recommended dose.

Key facts

Study ID: NCT02639546
Run by: Hoffmann-La Roche
People needed: 56
Starts: 2016-05-20
Expected to finish: 2021-07-21
Last updated by the study team: 2022-09-16

Who can join

Age: 1 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

For dose-escalation stage (tablets): age at study entry >= 6 years to < 18 years
For dose-escalation stage (suspension): age at study entry >= 6 months to < 18 years. Participants <1 year of age will not be enrolled until >= 6 participants >= 1 year to < 18 years of age have received at least one cycle of therapy with suspension and until safety and pharmacokinetic assessment of these participants have been conducted.
For expansion stage: age at study entry to be >= 6 months (>=6 years if suspension is not available) to < 30 years. Participants >= 6 months to < 1 year of age may not be enrolled until >= 6 participants >= 1 year to < 18 years of age have received at least one cycle of therapy with suspension in the dose-escalation phase and until safety and pharmacokinetic assessment of these participants have been conducted.
Tumor for which prior treatment has proven to be ineffective or intolerable or for which no standard therapy exists
Tumor with known or expected RAS/RAF/MEK/ERK pathway involvement. Diagnosis must be one of the following tumor types:
Central nervous system gliomas, including high- and low-grade gliomas, and diffuse intrinsic pontine glioma (DIPG) Embryonal rhabdomyosarcoma and other non-rhabdomyosarcoma soft tissue sarcomas Neuroblastoma Melanoma Malignant peripheral nerve sheath tumor Rhabdoid tumors, including atypical teratoid/rhabdoid tumor (ATRT) NF1-associated tumor (including plexiform neurofibroma), schwannoma, or RASopathy-associated tumor that in the judgment of the investigator is life threatening, results in severe symptoms (including severe pain), or is in close proximity to vital structures
Measurable disease as defined by mINRC, RANO criteria for HGG, RANO criteria for LGG, RECIST v1.1, or evaluable by nuclear medicine techniques, immunocytochemistry, tumor markers, or other reliable measures
Availability of tumor tissue at study enrollment
Lansky performance status or Karnofsky performance status of >= 50 percent
Life expectancy >= 3 months
Adequate hematologic, cardiac, and end-organ function
Body weight must be >= 20 kilograms (kg) if suspension is not available

You may not qualify if…

Pregnant or lactating women
Close proximity in time to treatment with high-dose chemotherapy, stem-cell rescue, differentiation therapy, immunotherapy, thoracic or mediastinal radiotherapy, hormonal therapy, biologic therapy, herbal cancer therapy, hematopoietic growth factor, investigational therapy, or St. John's wort according to protocol-defined criteria prior to initiation of study drug
Inability to swallow oral medications
Impaired gastrointestinal absorption
History or evidence of retinal pathology according to protocol-defined criteria, including serous retinopathy
History of Grade >= 2 central nervous system (CNS) hemorrhage
History of CNS hemorrhage within 28 days of study entry. This criterion may be waived at the investigator's request if the CNS hemorrhage was asymptomatic, with approval of the Medical Monitor
Known active infection (excluding fungal infection of the nail beds) within 28 days prior to initiation of study drug that has not completely resolved
Major surgical procedure or significant traumatic injury within 4 weeks prior to initiation of study drug, or anticipation of need for major surgical procedure during the course of the study
Prior allogenic bone marrow transplantation or prior solid organ transplantation

Where it is running

Arkansas Children'S Hospital — Little Rock, Arkansas, United States
Arnold Palmer Hosp-Children — Orlando, Florida, United States
Dana Farber Cancer Institute — Boston, Massachusetts, United States
Memorial Sloan Kettering Cancer Center — New York, New York, United States
University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States
Hôpital de la Timone, Oncologie Pédiatrique — Marseille, France
Institut Curie, Oncologie Pédiatrique — Paris, France
Institut Gustave Roussy; Service Pediatrique — Villejuif, France
Universitaetsklinikum Muenster — Münster, Germany
Schneider Children's Medical Center — Petah Tikva, Israel
Fondazione IRCCS Istituto Nazionale dei Tumori; Struttura Complessa di Pediatria Oncologica — Milan, Lombardy, Italy
Hospital Universitari Vall d'Hebron — Barcelona, Spain
Hospital Infantil Universitario Nino Jesus — Madrid, Spain
Hospital Universitari i Politecnic La Fe de Valencia — Valencia, Spain
Alderhey Childrens Trust — Liverpool, United Kingdom
Great Ormond Street Hospital; Dept. Of Pediatric Oncology — London, United Kingdom
The Royal Victoria Infirmary; Paediatric and Adolescent Oncology Unit — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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