drugset / Trial / NCT02806947
A Study to Evaluate Steroid-free Treatment for Standard-Risk aGVHD (BMT CTN 1501)
Phase 2
Completed
127 enrolled
Medical College of Wisconsin
Blood and Marrow Transplant Clinical Trials Network · collabNational Cancer Institute (NCI) · collabNational Heart, Lung, and Blood Institute (NHLBI) · collabNational Marrow Donor Program · collab
RandomizedParallel-groupOpen-labelTreatment
Summary
The study is a Phase II randomized, open label, multicenter trial designed to identify whether sirolimus is a potential alternative to prednisone as an up-front treatment for patients with standard-risk acute GVHD defined according to clinical and biomarker-based risk stratification. This trial incorporates both a novel up front GVHD therapy (sirolimus) as well as a novel BMT CTN developed acute GVHD biomarker test.
Timeline
- Start
- 2016-10
- Primary completion
- 2018-08-17
- Completion
- 2019-02-19
Outcome
Missed primary endpoint
registry analysis (other test); Sirolimus vs Prednisone; Risk Difference (RD) -0.082 (90% CI -0.223 to 0.059) NCT02806947 ↗
registry analysis (other test); Sirolimus vs Prednisone; Risk Difference (RD) -0.152 (95% CI -0.315 to 0.011) NCT02806947 ↗
Publications
- Background Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, Martin P, Chien J, Przepiorka D, Couriel D, Cowen EW, Dinndorf P, Farrell A, Hartzman R, Henslee-Downey J, Jacobsohn D, McDonald G, Mittleman B, Rizzo JD, Robinson M, Schubert M, Schultz K, Shulman H, Turner M, Vogelsang G, Flowers ME. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005 Dec;11(12):945-56. doi: 10.1016/j.bbmt.2005.09.004.
- Giaccone L, Faraci DG, Butera S, Lia G, Di Vito C, Gabrielli G, Cerrano M, Mariotti J, Dellacasa C, Felicetti F, Brignardello E, Mavilio D, Bruno B. Biomarkers for acute and chronic graft versus host disease: state of the art. Expert Rev Hematol. 2021 Jan;14(1):79-96. doi: 10.1080/17474086.2021.1860001. Epub 2020 Dec 24.
- Pidala J, Hamadani M, Dawson P, Martens M, Alousi AM, Jagasia M, Efebera YA, Chhabra S, Pusic I, Holtan SG, Ferrara JLM, Levine JE, Mielcarek M, Anasetti C, Antin JH, Bolanos-Meade J, Howard A, Logan BR, Leifer ES, Pritchard TS, Horowitz MM, MacMillan ML. Randomized multicenter trial of sirolimus vs prednisone as initial therapy for standard-risk acute GVHD: the BMT CTN 1501 trial. Blood. 2020 Jan 9;135(2):97-107. doi: 10.1182/blood.2019003125.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Prednisone | Other / unclassified | 2 mg/kg | — |
| Subject | Sirolimus | Small molecule | 5 mg/m2 | — |
| Subject | Sirolimus | Small molecule | 6 mg | — |