TABLE 2:

Clinical trial adaptive design for study of Prochymal/Remestemcel-L for GvHD

Study	NCT00366145	NCT02336230
Product/ Sponsor	Prochymal™/ Osiris Therapeutics	Remestemcel-L™/ Mesoblast
Cell dosing	2 million cells/kg twice a week for 4 weeks	2 million cells/kg twice a week for 4 weeks
Primary outcome	Complete GvHD Response of greater than or equal to 28 days duration	Overall GvHD Response Rate at Day 28 as to include both complete response and partial response: • Complete response (CR) - Resolution of GVHD in all involved organs. • Partial response (PR) - Organ improvement by at least one stage without worsening of any other organ.
Target Enrollment	240	60
Ages eligible	6 Months to 70 Years	2 months and 17 years
GvHD grade at enrollment	Any grade B-D (IBMTR grading) of acute GvHD	• Grade C or D GvHD involving the skin, liver, and/or gastrointestinal (GI) tract • Grade B GvHD involving the liver and/or GI tract, with or without concomitant skin disease • Exclusion: Grade B GvHD with skin-only involvement.
Steroid refractory definition	No improvement after 3 days and a duration of no greater than 2 weeks	Progression within 3 days or no improvement within 7 consecutive days
Study sites	70	23
Completed	May 2009	February 2018

Meaningful study design adaptations in NCT02336230 are in bold characters.