Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease
NCT Number: NCT06462469
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE4
Conditions: Steroid-refractory Acute Graft Versus Host Disease
Interventions: DRUG: Ruxolitinib
Sponsor: Novartis Pharmaceuticals
Sex: ALL
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 54
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-04
Completion Date: 2026-11-05
Locations: Novartis Investigative Site, Guangzhou, Guangdong, 510515, China|Novartis Investigative Site, Wuhan, Hubei, 430030, China|Novartis Investigative Site, Chang Chun, Jilin, 130021, China|Novartis Investigative Site, Hangzhou, Zhejiang, 310003, China|Novartis Investigative Site, Beijing, 100034, China|Novartis Investigative Site, Beijing, 100039, China|Novartis Investigative Site, Dalian, 116000, China|Novartis Investigative Site, Fuzhou, 350001, China|Novartis Investigative Site, Guangzhou, 510180, China|Novartis Investigative Site, Shanghai, 200025, China|Novartis Investigative Site, Tianjin, 300020, China

Summary:
The purpose of this study is to assess the efficacy and safety of ruxolitinib therapy in Chinese adults and adolescents (≥ 12 years old) with Grade II-IV steroid-refractory acute graft versus host disease (SR-aGvHD).

Primary Outcome Measures: Overall Response Rate (ORR) at Day 28 per Investigators, The ORR at Day 28 defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or nonresponse, according to standard criteria and assessed by investigators., Day 28
Secondary Outcome Measures: Durable Overall response rate (ORR) at Day 56, Durable ORR at Day 56 is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR) at Day 28 and maintain a CR or PR at Day 56., Day 56|Duration of Response (DOR), DOR is defined as the time from first response until aGvHD progression or the date of additional systemic therapies for aGvHD., From Week 1 to long term follow up Month 12|Best overall response (BOR), Percentage of participants who achieved overall response (complete response (CR) + Partial response (PR) at any time point up to and including Day 28 and before the start of additional systemic therapy for aGvHD., From week 1 to Day 28|Overall survival (OS), Overall survival (OS) is defined as the time from the date of start of study treatment to date of death due to any cause., From the date of start of study treatment to date of death, up to approx. 12 months|Non-relapse mortality (NRM), Non-relapse mortality (NRM) is defined as the time from date of start of study treatment to date of death not preceded by hematologic disease relapse/progression., From date of start of study treatment to date of death, up to approx. 12 months|Event-free survival (EFS), Event-free survival (EFS) is defined as the time from the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death due to any cause., From the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death, up to approx. 12 months|Failure-free survival (FFS), Failure-free survival (FFS) is defined as the time from the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment., From the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment, up to approx. 12 months|Malignancy Relapse/Progression (MR), Malignancy Relapse/Progression (MR) is defined as the time from date of start of study treatment to hematologic malignancy relapse/progression. Calculated for participants with underlying hematologic malignant disease., From date of start of study treatment to hematologic malignancy relapse/progression, up to approx. 12 months|Reduction of daily corticosteroids dose, This includes the assessment of systemic corticosteroid use and daily dose, and the percentage of participants successfully tapered off all systemic corticosteroids until Day 56, by time intervals and overall., Up to Day 56|Cumulative incidence of chronic GvHD, Cumulative incidence of chronic GvHD (cGvHD) includes mild, moderate and severe occurrences., From Week 1 to long term follow up of month 12