TOURMALINE-AL1 trial, a phase III, randomized clinical trial evaluating the effect of Ninlaro (ixazomib) in combination with dexamethasone, did not meet the first of the two primary endpoints of significant improvement in overall hematologic response in patients with relapsed or refractory systemic light-chain amyloidosis.
The study is sponsored by Takeda Pharmaceutical Co. The results were presented during an oral session at the 61st American Society of Hematology annual meeting.
Hematologic responses were seen in 53% versus 51% of patients receiving Ninlaro plus dexamethasone versus physician’s choice (odds ratio 1.10 [95% CI 0.60-2.01], p=0.762) as assessed by an adjudication committee. The second primary endpoint of two-year vital organ deterioration or death was not mature at the time of analysis. Other endpoints studied including vital organ progression free survival, hematologic PFS, time to treatment failure and time to subsequent therapy were numerically higher in the Ninlaro plus dexamethasone arm compared to the physician’s choice arm.
Takeda said the company is committed to making data available to researchers to continue investigation of this disease. Ninlaro is not approved as a treatment for AL amyloidosis.
“AL amyloidosis is a rare condition, for which prognosis and patient outcomes are poor. Current treatments are often retrofitted from therapies used for multiple myeloma,” said Angela Dispenzieri, Mayo Clinic, and the trial’s principal investigator and lead author. “For a phase III study that did not meet its primary endpoint, this trial provides interesting information for this community and for future studies. Ongoing research and development to investigate potential treatment options for this underserved patient population is critical.”
