Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease

Phase 4

Recruiting

• Conditions: Steroid-refractory Acute Graft Versus Host Disease
• Interventions: Drug: Ruxolitinib

• Registration Number: NCT06462469
• Lead Sponsor: Novartis Pharmaceuticals

Brief 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).

Detailed Description

Participants will start with a screening period to assess the eligibility; only participants who meet all the inclusion and none of the exclusion criteria will start study treatment from Day 1 to Week 24 or end of treatment. Following safety follow up visits, participants will receive the long-term follow-up until Month 12.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2024-06-05
• Study First Submitted QC: 2024-06-14
• Study First Posted: 2024-06-17
• Study Start: 2024-07-04
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-21
• Last Update Posted: 2024-11-25
• Primary Completion: 2025-09-22
• Study Completion: 2026-11-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ruxolitinib	Ruxolitinib	Participants will receive ruxolitinib orally of 10 mg BID daily (given as two 5-mg tablets, approximately 12 hours apart).

Primary Outcome Measures

Name	Time	Method
Overall Response Rate (ORR) at Day 28 per Investigators	Day 28	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.

Secondary Outcome Measures

Name	Time	Method
Non-relapse mortality (NRM)	From date of start of study treatment to date of death, up to approx. 12 months	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.
Best overall response (BOR)	From week 1 to Day 28	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.
Event-free survival (EFS)	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	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.
Reduction of daily corticosteroids dose	Up to Day 56	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.
Cumulative incidence of chronic GvHD	From Week 1 to long term follow up of month 12	Cumulative incidence of chronic GvHD (cGvHD) includes mild, moderate and severe occurrences.
Overall survival (OS)	From the date of start of study treatment to date of death, up to approx. 12 months	Overall survival (OS) is defined as the time from the date of start of study treatment to date of death due to any cause.
Malignancy Relapse/Progression (MR)	From date of start of study treatment to hematologic malignancy relapse/progression, up to approx. 12 months	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.
Durable Overall response rate (ORR) at Day 56	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.
Duration of Response (DOR)	From Week 1 to long term follow up Month 12	DOR is defined as the time from first response until aGvHD progression or the date of additional systemic therapies for aGvHD.
Failure-free survival (FFS)	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	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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇨🇳 Tianjin, China