This study will be conducted to compare Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
IV infusion
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Objective Response (OR) at 6 months
Defined for each treatment group as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.
Time frame: 6 months
Failure-free survival (FFS)
Defined as the time from the date of randomization to the date of addition or initiation of another systemic therapy for cGVHD, relapse of underlying disease, or death due to any cause.
Time frame: Up to 5 years
Proportion of participants with a ≥ 7-point improvement in modified Lee Symptom Scale (mLSS) total score
Time frame: Up to 5 years
Overall Response at 12 months
Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD.
Time frame: 12 months
Best Overall Response (BOR)
Defined as the best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1), and at any timepoint up to the initiation of new therapy for cGVHD.
Time frame: Up to 5 years
DOR (in responders only)
Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD from baseline scoring, start of new systemic treatment for cGVHD, or death from any cause, whichever comes first. An additional measure of response durability will consider DOR as the time from the date of first response to the date of new systemic therapy for cGVHD or death from any cause, whichever occurs first.
Time frame: Up to 5 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Medical University of Graz
Graz, Austria
Medizinische Universitaet Innsbruck - Universitaetsklinik Fuer Innere Medizin Iii
Innsbruck, Austria
Krankenhaus Der Elisabethinen Linz Gmbh
Linz, Austria
Medizinische Universitat Wien, Universitatsklinik Fur Innere Medizin I
Vienna, Austria
St. Anna Childrens Hospital
Vienna, Austria
Az Sint-Jan Brugge Av
Bruges, Belgium
Chu Ucl Namur Site Godinne
Godinne-mont, Belgium
Universitair Ziekenhuis Leuven
Leuven, Belgium
Chu Liege -Centre Hospitalier Universitaire Sart Tilman
Liège, Belgium
Algemeen Ziekenhuis Delta
Roeselare, Belgium
...and 120 more locations
Organ-specific response
Organ-specific response as defined in the protocol.
Time frame: Up to 5 years
Overall Survival (OS)
Defined as the time from the date of randomization to the date of death due to any cause.
Time frame: Up to 5 years
Nonrelapse mortality (NRM)
Defined as the time from the date of randomization to the date of death not preceded by relapse of primary hematologic disease.
Time frame: Up to 5 years
Time to primary hematologic disease relapse
Defined as the time from the date of randomization to the date of relapse.
Time frame: Up to 5 years
Percent reduction in daily corticosteroid dose at 6 months
Time frame: 6 months
Proportion of participants who tapered off all corticosteroids at 6 months
Time frame: 6 months
Number of participants with Treatment-emergent Adverse Events (TEAEs)
Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.
Time frame: Up to 5 years and 30 days