Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation

Conditions

• Corticosteroid Refractory Acute Graft vs Host Disease

Eligibility Criteria

Sex

ALL

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Ruxolitinib (RUX) — DRUG
  Ruxolitinib was provided as 5 mg tablets for oral use.
• Best Available Therapy (BAT) — DRUG
  BAT was based on the investigator's best judgment, taking into account the manufacturer's instructions, labeling, patient's medical condition, and institutional guidelines for any dose adjustment. The BAT in this study was identified by the investigator prior to patient randomization among the following treatments currently used in this setting: anti-thymocyte globulin (ATG), extracorporeal photopheresis (ECP), mesenchymal stromal cells (MSC), low-dose methotrexate (MTX), mycophenolate mofetil (MMF), mTOR inhibitors (everolimus or sirolimus), etanercept, or infliximab. No other types or combinations of BAT were permitted.

Study Details

Assess the efficacy and safety of ruxolitinib compared to Best Available Therapy (BAT) in patients with corticosteroid-refractory acute graft vs. host disease (aGvHD) after allogeneic stem cell transplantation.

Key Dates

Start date

Mar 10, 2017

Status verified

May 2022

Primary completion

Jun 24, 2019

Completion

Apr 23, 2021

Study Design

Enrollment

310 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Ruxolitinib
  These patients were administered Ruxolitinib orally twice per day (b.i.d) at a dose of 10 mg bid, as two 5-mg tablets. Ruxolitinib was taken without regards to food.
• Active Comparator: Best Available Therapy (BAT)
  These patients were administered BAT per the Investigator's best judgement based on a specific list of BAT.

Primary Outcome Measure

Overall Response Rate (ORR) at Day 28 [ Time Frame: Day 28 ]