On February 22, 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 a partnership with Medidata, a Dassault Systèmes company, to evaluate the use of its Synthetic Control Arm (SCA) solution in a planned Phase 2 trial of Rhenium-186 NanoLiposome (186RNL) in recurrent glioblastoma (GBM), a type of brain cancer (Press release, Cytori Therapeutics, FEB 22, 2022, View Source [SID1234608799]).
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SCAs are especially advantageous in indications such as recurrent GBM where the standard of care control treatment is considered undesirable by some patients and physicians. The SCA enables study designs that have a higher than usual probability of assignment of prospective patients to the investigational therapy. This enhances patient enrollment and retention and potentially reduces clinical trial costs while upholding the scientific integrity of the trial.
"Medidata has developed a pioneering capability and helped to validate the use of synthetic controls in clinical drug development," said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. "Although a recent advancement, the U.S. Food and Drug Administration has already agreed to recognize a Phase 3 clinical trial design incorporating a synthetic control arm in a registrational randomized control arm in recurrent GBM."
About the Synthetic Control Arm
Synthetic control arm – 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.