23.0705:  Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS or ULTOMIRIS in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Significant Extravascular Hemolysis (csEVH)

Principal Investigator: Vivek Sharma, MD, FACP


Description:  This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.

More Info:  https://clinicaltrials.gov/study/NCT05982938?term=NCT05982938&rank=1


23.0606:  A Long-Term Follow-Up Study in Subjects with Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial 

Principal Investigator: Vivek Sharma, MD, FACP 

Phase:  Choose an item. 

Description:  The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy. 

ClinicalTrials.gov:  https://clinicaltrials.gov/study/NCT05768386?term=NCT05768386&rank=1

22.0978:  A Phase 1, Open-label, Multicenter Study of the Safety, Pharmacokinetics, and Pharmacodynamics of MK-4464 as Monotherapy and in Combination with Pembrolizumab in Participants with Advanced/Metastatic Solid Tumors

Principal Investigator: Rebecca Redman, M.D.

Phase:  I

Description: The purpose of this study is to assess the safety, pharmacokinetics, and preliminary efficacy of MK-4464 as monotherapy and in combination with pembrolizumab in participants with advanced/metastatic solid tumors.

More Info:  https://clinicaltrials.gov/ct2/show/NCT05514444?term=NCT05514444&draw=2&rank=1

08.0388 - Protocol for the Cancer Database and Specimen Repository (CDSR) at the Brown Cancer Center (Previous Lung Repository).

Principal Investigator: Rebecca Redman, M.D.

Description: The mission of the Cancer Database and Specimen Repository (CDSR) is to collect clinical information and samples of human specimens from cancer patients and from control patients for research, and to distribute specimens to researchers for the advancement of science.