CURE's Clinical Trial Corner: October 2019

As the cancer treatment landscape continues to grow, patients and their caregivers should be aware of the various clinical trials currently being conducted — and ones they can possibly join.

Here is a list of the recent trial initiations that occurred in October.

Glioblastoma

The first patient with recurrent glioblastoma multiforme was dosed with MB-108 (oncolytic virus C134), an attenuated herpes simplex virus type 1, according to Mustang Bio, Inc.

In the phase 1 trial, researchers are evaluating the safety and efficacy of MB-108 and will enroll up to 24 patients. They will receive escalating doses of MB-108, for which safety will be assessed at each dose level before proceeding to the next.

Mustang Bio and Nationwide Children’s Hospital, where the virus was developed, plan to conduct a subsequent clinical trial that will investigate the combination treatment of MB-101 (IL13Rα2-specific CAR T) and MB-108 to potentially enhance efficacy in treating glioblastoma.

Myelodysplastic Syndromes

The first patient was dosed in the phase 3 IMerge trial, designed to evaluate imetelstat, a first-in-class telomerase inhibitor, in those who are transfusion dependent with a lower risk for myelodysplastic syndromes (MDS) and are relapsed after or refractory to erythropoiesis-stimulating agents.

The randomized, double-blind, placebo-controlled clinical trial is planned to enroll approximately 170 patients to determine if imetelstat improves the rate of red blood cell transfusion independence.

“Patients with lower risk MDS become dependent on serial transfusions which leads to iron overload, heart and kidney complications, decreases in quality of life and shorter overall survival. Reducing transfusion burden and achieving transfusion independence remain significant medical needs for this disease,” Dr. Aleksandra Rizo, chief medical officer of Geron, the agent’s manufacturer, said in a press release. “Dosing of the first patient in the IMerge phase 3 clinical trial is an important step in developing imetelstat as a potential alternative for patients with lower risk MDS who have limited treatment options available today.”

The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia.

Gastric or Gastroesophageal Junction Cancer

The first patient was dosed in the phase 2/3 MAHOGANY clinical trial, designed to evaluate margetuximab in combination with a checkpoint inhibitor (MGA012), with or without chemotherapy, as a potential first-line treatment for patients with HER2-positive gastric cancer or gastroesophageal junction (GEJ) cancer.

The clinical trial of margetuximab, an investigational, Fc-optimized monoclonal antibody targeting HER2, will be conducted globally. In module A of the study, the primary endpoint is objective response rate. In module B, researchers will evaluate margetuximab plus a checkpoint inhibitor in combination with chemotherapy compared to standard of care therapy of Herceptin (trastuzumab) with chemotherapy in patients with HER2-positive tumors irrespective of PD-L1 expression. The primary endpoint will be overall survival.

Prostate Cancer

Sophiris Bio Inc. and the Food and Drug Administration (FDA) agreed upon the design of a single phase 3 clinical trial to evaluate topsalysin, a targeted focal therapy, in approximately 700 patients with intermediate risk for localized prostate cancer.

Patients will be randomized to receive a single administration of either topsalysin or placebo to determine the proportion of patients at 12 months who have failed treatment. In order to receive FDA approval, the company will have to evaluate all patients that progress to alternative treatments for an additional 12 months.

Ewing Sarcoma

Memorial Sloan Kettering Cancer Center (MSKCC) and Nationwide Children’s Hospital have been added as sites for the phase 1/2 trial of seclidemstat for the treatment of patients with relapsed or refractory Ewing sarcoma.

The open-label dose escalation and dose expansion study will determine the maximum tolerated dose and establish the initial safety profile of seclidemstat.

In addition to MSKCC and Nationwide Children’s, active clinical trial site locations include, Johns Hopkins All Children’s Hospital in St. Petersburg, Fla.; Children’s Hospital of Los Angeles; Moffitt Cancer Center in Tampa, Fla.; Dana-Farber Cancer Institute in Boston; the University of Texas MD Anderson Cancer Center in Houston; and the Sarcoma Oncology Center in Santa Monica, Calif.

Thrombocytopenia

Patient enrollment has begun in the pivotal phase 3 PACIFICA trial, designed to compare the efficacy and safety of pacritinib with physician’s choice therapy in 180 adult patients with myelofibrosis and severe thrombocytopenia.

"An estimated one-third of patients with myelofibrosis are severely thrombocytopenic — a population with limited therapeutic options and poor survival, thereby making this disease setting a very important area of unmet medical need. Moving forward, successful trial execution is our primary focus, and with patient enrollment now underway, we expect to report topline results in mid-2021,” Dr. Adam R. Craig, president and chief executive officer of CTI BioPharma, said in a press release.

Liver Cancer

The open-label, multi-center phase 1b/2 clinical trial evaluating Fotivda (tivozanib) in combination with Imfinzi (durvalumab) has started to enroll patients with hepatocellular carcinoma (HCC) who have not received prior systemic therapy. The study is expected to enroll approximately 50 patients.

The phase 1b portion will evaluate the safety, tolerability, dose limiting toxicity, maximum tolerated dose and preliminary anti-tumor activity and, assuming satisfactory completion of the phase 1b portion of the study, a phase 2 expansion cohort will enroll at the dose schedule designated in phase 1b.