On the use of non-concurrent controls in platform trials: a scoping review
ConclusionsStatistical methods for incorporating non-concurrent controls are available in the literature, either by means of methods originally proposed for the incorporation of external controls or non-concurrent controls in platform trials. Methods mainly differ with respect to how the concurrent and non-concurrent data are combined and temporary changes handled. Regulatory guidance for non-concurrent controls in platform trials are currently still limited. (Source: Trials)
Source: Trials - June 15, 2023 Category: Research Source Type: clinical trials
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 24, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today provided the following update on the Biologics License Application (BLA) for SRP-9001... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - May 24, 2023 Category: Pharmaceuticals Source Type: clinical trials
Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 12, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) Cellular, Tissue and Gene... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - May 12, 2023 Category: Pharmaceuticals Source Type: clinical trials
Soligenix Provides Regulatory Update on HyBryte
PRINCETON, N.J., April 14, 2023. Soligenix, Inc. (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - April 14, 2023 Category: Pharmaceuticals Source Type: clinical trials
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Condition: Sickle Cell Disease Intervention: Other: Interview Sponsor: St. Jude Children's Research Hospital Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 12, 2023 Category: Research Source Type: clinical trials
