On April 5, 2022 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, reported that it has expanded its partnership with Medidata, a Dassault Systèmes company (Press release, Cytori Therapeutics, APR 5, 2022, View Source [SID1234611460]).
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The goal of the expanded partnership is to speed enrollment, improve patient access to an innovative therapy and reduce clinical trial costs in Plus Therapeutics’ planned forthcoming Phase 2 registrational trial of Rhenium-186 NanoLiposome (186RNL) in recurrent glioblastoma (GBM). The partnership will utilize Medidata’s Synthetic Control Arm (SCA) platform that facilitates the use of historical clinical trial (HCT) data in a manner that historically has been favorably received by the U.S. Food and Drug Administration. The expanded partnership follows a successful preliminary assessment stage intended to determine project feasibility and probability of success.
"Synthetic control arms reduce the time and cost associated with complex clinical trials in rare diseases such as glioblastoma," said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. "Plus has been quite impressed with Medidata’s team, capabilities and platform in the recently completed feasibility phase. Furthermore, the benefit is passed down to patients and their families, allowing for fewer patients to be exposed to placebos or existing standard-of-care treatments that might not be effective for them, offering them greater access to potentially life-extending therapies."
Medidata will provide the Company with a SCA based on a historical pool of anonymized HCT data to incorporate into Plus Therapeutics’ planned Phase 2 trial of 186RNL in recurrent GBM. SCAs are especially advantageous in indications such as recurrent GBM where the standard-of-care control treatment is considered undesirable by many patients and physicians.
About the Synthetic Control Arm
Medidata’s Synthetic Control Arm (SCA) – a type of external control – is formed by carefully selecting patients from Medidata’s extensive repository of historical clinical trials to match the baseline demographic and disease characteristics of the patients treated with the new investigational product. Case studies have shown that SCAs can effectively mimic a classic randomized control and, therefore, can be used to accurately interpret the treatment effects of an investigational product.
SCAs can help enhance the scientific validity of single-arm trials and, in certain indications, enhance randomized clinical trials to expose fewer prospective patients to control and/or ineffective or existing standard-of-care treatments that might not provide a benefit to the patient. This is done while still providing highly valid scientific evidence. These factors can influence a patient’s willingness to participate in a trial where there is a very poor prognosis and perceived inadequate standard of care.