Enrollment has begun in the second part of a clinical trial testing safusidenib, an experimental oral treatment, as a maintenance therapy for high-grade IDH1-mutant astrocytoma — an aggressive form of glioma.
The study (NCT05303519) — sponsored by safusidenib’s developer, Nuvation Bio — aims to enroll 300 adults at sites in the U.S., Australia, and China. Participants must have already undergone standard-of-care treatment (chemotherapy, radiation and/or surgery).
The trial has two parts. The first tested the efficacy, safety, and pharmacokinetics (how the compound moves throughout the body) of safusidenib in people with recurrent or progressive IDH1-mutant glioma. The second part will now assess the efficacy of maintenance safusidenib treatment versus placebo following completion of standard-of-care treatment.
Study focuses on delaying tumor growth
Participants will be randomly assigned to receive safusidenib or a placebo twice a day. The main goal is to find out whether the treatment can help people live longer without their tumor growing, a measure called progression-free survival.
The trial was originally conceived as a Phase 2 study, but after discussions with the U.S. Food and Drug Administration (FDA), Nuvation is working to expand it to a Phase 3 trial. If the results are positive, this Phase 3 version could be used to seek FDA approval for the therapy.
“With our first patient now enrolled in the U.S., we look forward to expanding the study into additional sites to support potential registration, as we continue our commitment to bringing therapies with meaningful clinical benefits to more patients with cancers that have severe unmet treatment needs,” David Hung, MD, founder, president and CEO of Nuvation, said in a company press release.
Astrocytoma is a type of brain cancer that occurs due to the uncontrolled growth of astrocytes — star-shaped brain cells that normally help support nerve function. In high-grade astrocytoma, the cancer is aggressive and tends to spread quickly within the brain. Standard treatments such as chemotherapy and surgery can help control the tumor, but the disease often returns after treatment.
“No targeted therapies have been approved to delay recurrence or progression in these patients, who are dealing with an aggressive disease that inevitably returns,” Hung said.
How safusidenib is designed to work
High-grade astrocytomas sometimes carry a mutated form of the IDH1 protein, which contributes to cancer growth. Safusidenib is designed to inhibit the mutant protein, thus limiting cancer growth. An early Phase 1 study (NCT05577416) tested safusidenib in people with low-grade glioma undergoing surgery, with results indicating the therapy was generally well-tolerated and showed signs of anticancer activity.
Katherine Peters, MD, PhD, an investigator on the trial at Duke Cancer Institute, said the therapy also “has shown promising activity in a Phase 1 study of patients with recurrent or progressive high-grade IDH1-mutant gliomas, with higher response rates than other IDH inhibitors have demonstrated in this setting.”
“Following surgery and standard-of-care treatment, these patients and their healthcare providers are left to watch and wait for progression or recurrence. Patients with this form of glioma need effective, well-tolerated options that can further delay this eventuality,” Peters said. “We look forward to further studying [safusidenib’s] potential in this trial.”
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