In the ten months since the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold it placed on U.S.-based clinical trials studying the drug tazemetostat, enrollment has reopened at sites across the country. In particular, clinical studies examining the EZH2 inhibitor tazemetostat in patients with INI1-negative tumors, including chordoma, are open and continuing to accrue patient-participants. Two of these trials are currently accepting chordoma patients – a Phase 2 trial for adults and a Phase 1 trial for pediatric patients.
The partial clinical hold followed the submission of a safety report by the tazemetostat manufacturer, Epizyme, to the FDA about a pediatric patient with A histological subtype of chordoma that is more aggressive and usually grows faster than the conventional type. It is more common in children and young adults, and is often characterized by loss of the INI1 protein. chordoma enrolled in the company’s Phase 1 pediatric study who developed secondary T-cell lymphoblastic lymphoma (T-LBL).
In the following months, Epizyme convened a panel of scientific and medical experts and conducted a comprehensive review to:
- Determine which secondary malignancies might be associated with tazemetostat treatment
- Assess the potential risk factors for patients involved in the tazemetostat trials
- Implement a risk mitigation and monitoring plan in an effort to prevent future incidents
Epizyme’s formal response to the FDA included a thorough assessment of clinical benefit and safety data across all of its trials in hematological malignancies and solid tumors – both adult and pediatric – including the two trials enrolling chordoma patients.
With the partial clinical hold lifted, Epizyme has reopened enrollment in all of its company-sponsored trials in the U.S. If you or a loved one is interested in learning more about this Research studies involving human subjects that are done to test whether a treatment is safe, and how well it will work to treat a specific disease., please speak with your healthcare team.
Questions? Contact a Chordoma Foundation Patient Navigator for more information.
Learn more about the tazemetostat trials: