The purpose of the study is to assess the efficacy and safety of ruxolitinib in Chinese adult and pediatric participants aged 12 years or older with corticosteroid-refractory chronic graft vs. host disease (SR-cGvHD).
This is a single arm, multi-center, open label study which will enroll approximately 50 participants and investigate the efficacy and safety of ruxolitinib administered in adult and adolescent (≥12 years old) Chinese participants with SR-cGvHD. The total duration on study for an individual participant will be up to 164 weeks (approximately 3 years). The study consists of following periods, with each cycle comprised of 4 weeks (28 days): * Screening Period (Day -28 to Day -1) * Treatment period (Day 1 to Cycle 39/EOT) * Safety follow-up (Last dose +30 days) * Long-term survival follow-up period (EOT to 156 weeks on study).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Ruxolitinib is taken orally daily at 10 mg BID, given as two 5 mg tablets.
Novartis Investigative Site
Hefei, Anhui, China
Overall Response Rate (ORR)
ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response, according to National Institute of Health (NIH) Consensus Criteria.
Time frame: Cycle 7 Day 1; each Cycle =28 days
Failure-free Survival (FFS)
FFS is defined as the time from the date of start of study treatment to the earliest of: i) relapse or recurrence of underlying disease or death due to underlying disease, ii) nonrelapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD.
Time frame: up to 3 years
Best Overall Response (BOR)
Percentage of participants who achieved overall response (CR+PR) at any time point (up Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD).
Time frame: at any point up to cycle 7 day 1 (each cycle is 28 days) or the start of additional systemic therapy for cGvHD, approx. 6 months
ORR at end of cycle 3
Percentage of participants who achieved overall response (CR+PR) at Cycle 4 Day 1.
Time frame: end of cycle 3; each cycle = 28 days
Duration of Response (DOR)
DOR is assessed for responders only and is defined as the time from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD.
Time frame: from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD, approx.36 months
Overall Response (OS)
OS is defined as the time from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause.
Novartis Pharmaceuticals
CONTACT
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Novartis Investigative Site
Guangzhou, Guangdong, China
RECRUITINGNovartis Investigative Site
Guangzhou, Guangdong, China
RECRUITINGNovartis Investigative Site
Nanning, Guangxi, China
RECRUITINGNovartis Investigative Site
Guiyang, Guizhou, China
RECRUITINGNovartis Investigative Site
Shijiazhuang, Hebei, China
RECRUITINGNovartis Investigative Site
Zhengzhou, Henan, China
RECRUITINGNovartis Investigative Site
Wuhan, Hubei, China
RECRUITINGNovartis Investigative Site
Nanjing, Jiangsu, China
RECRUITINGNovartis Investigative Site
Xuzhou, Jiangsu, China
RECRUITING...and 12 more locations
Time frame: from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause, approx. 36 months
Non-Relapse Mortality (NRM)
NRM is defined as the time from date of study treatment (ruxolitinib) initiation to date of death not preceded by underlying disease relapse/recurrence.
Time frame: from date of study treatment (ruxolitinib) initiation to date of death not preceded by underlying disease relapse/recurrence, approx. 36 months
Malignancy Relapse/Recurrence (MR)
Malignancy relapse/recurrence is defined as the time from date of study treatment to hematologic malignancy relapse/recurrence.
Time frame: from date of study treatment to hematologic malignancy relapse/recurrence, approx. 36 months
Reduction of daily corticosteroids dose at cycle 7 day 1
Systemic corticosteroid use is the percentage of participants with \>=50% reduction from baseline in daily corticosteroid dose, the proportion of subjects with reduction from baseline to ≤ 0.2 mg/kg/day methylprednisolone (or equivalent dose of ≤ 0.25 g/kg/day prednisone or prednisolone), and subjects successfully tapered off all systemic corticosteroids at Cycle 7 Day 1, by time intervals and overall.
Time frame: Cycle 7 Day 1; each cycle = 28 days