Multiple Myeloma Research Foundation Bets on Adaptive Platform Clinical Trial Design

A new clinical trial involving patients with multiple myeloma is making use of a platform study design in order to test multiple different therapies for the hematologic malignancy. The MyDRUG platform trial will be run by the Multiple Myeloma Research Foundation (MMRF) to simultaneously investigate multiple targeted treatments.

Platform trials make use of an adaptive study design which offers distinct advantages over conventional clinical trial frameworks; while most clinical trials will test the safety and efficacy of a single drug in the target patient population, platform trials allow researchers to assess multiple compounds and rapidly eliminate those that don’t appear to be effective.

The MMRF will be using findings from the CoMMpass study – which found that myeloma can be broken down into 12 subtypes based on its genomic signature – to test the efficacy of therapeutic agents targeting different aspects of the cancer. AbbVie, Celgene, Eli Lilly, Genentech, Janssen Pharmaceuticals, and Takeda Oncology are all pharmaceutical companies whose compounds will be tested in the trial.

All but one of the drugs to be tested in the adaptive platform trial – Janssen’s erdafitinib – have been approved for other indications in oncology. Darzalex (daratumab) is another Janssen-owned compound to be included in the study which has already been approved to treat patients with multiple myeloma when used in combination with other therapeutics.

AbbVie’s chronic lymphocytic leukemia drug Venclexta (venetoclax), Genentech’s melanoma treatment Cotellic (cobimetinib), Celgene’s acute myeloid leukemia drug Idhifa (enasidenib) and Eli Lilly’s Verzenio (abemaciclib) will also be assessed for efficacy in patients with high risk myeloma. Takeda’s Ninlaro (ixazomib) and Celgene’s Pomalyst (pomalidomide) will be tested in a treatment arm involving patients without any of the mutations identified in those participating in other arms of the study.*

“MyDRUG is a major milestone for the MMRF in terms of putting precision medicine to work for patients through our unique end-to-end model, which aims to deliver the right treatment to every patient living with myeloma,” said Paul Giusti, President and Chief Executive Officer of the MMRF. “This would not be possible without the groundwork of CoMMpass. Now, with the initiation of MyDRUG, we have the potential to accelerate the development of treatments for patients based on their specific genomic characteristics.”

Myeloma patients who have not received a stem cell transplant and have relapsed within 18 months of receiving treatment will be eligible to participate in the MyDRUG platform trial. Those who have relapsed within 36 months despite receiving a transplant will also be able to be enrolled.

Interestingly, the MyDRUG platform trial will have no end date, nor will the study investigators place any limitations on the number of drugs studied or the number of patients enrolled and dosed in the trial.

“The MMRF MyDRUG Study is an incredible milestone for the myeloma community and I am proud to be part of this truly collaborative effort that will accelerate the delivery of much needed new options to high-risk patients,” said Dr. Shaji Kumar of the Mayo Clinic, and Principal Investigator for MyDRUG. “This trial would not be possible without the MMRF, which has the leadership to spearhead a study this complex, with so many pharmaceutical and academic partners working together for the benefit of patients.”

While studies making use of the adaptive platform trials framework are not the most common in the pharma industry, the National Institutes of Health (NIH) clinical trials database at ClinicalTrials.gov does list a few active trials. Among these are a platform trial run by GSK testing new treatments for non-small cell lung cancer (NSCLC) and a study investigating multiple anti-PD-1 combination drugs in patients with various advanced or metastatic solid tumors.

As the second most common form of blood cancer, myeloma affects 30,000 new adult patients each year with over 12,500 patients dying as a result of the disease. Currently, treatments for myeloma aren’t always effective for patients with the high-risk form of the disease, representing a significant unmet medical need for this population.

*Editor’s Note: A previous version of this article did not specify which compound developed by Takeda would be included in the trial.