FDA Recommends Umbrella Trials for Early Study of Multiple Cell/Gene Therapies
Multiple versions of a cell or gene therapy may be studied under a certain type of umbrella trial in which the products being studied are for a single disease, the FDA says in a new final guidance.
Umbrella trials can make the development of these products more flexible and efficient, the guidance says, by evaluating multiple versions at the same time and helping narrow the list of potential candidates.
“Comparisons can be facilitated by randomization between the study arms, if feasible,” the FDA advises. “Additionally, this trial design may facilitate sharing of the control group, potentially facilitating investigator participation and subject enrollment, and may simplify study management relative to conducting a separate clinical trial for each product version.”
But because the product versions being studied will significantly differ from each other, the agency advises that sponsors submit each candidate in separate investigational new drug applications (IND) that cross-reference each other.
The guidance provides details on how to submit information for these multiple INDs and file updates as the trial progresses, acknowledging that the structure and organization of multiple INDs for a single umbrella trial can be confusing. It explains how to add arms to the trial, submit protocol revisions and chemistry, manufacturing and controls (CMC) and pharmacology/toxicology information, and respond to clinical holds. It also covers IND safety reporting, and completion of the trial and its arms.
The guidance also includes an appendix containing multiple examples of different versions of cell and gene therapies that could be combined in umbrella trials and those that could not.
Read the full final guidance here: https://bit.ly/3EhNSEP.