This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric 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
60
Axatilimab at the protocol-defined dose.
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
Pharmacokinetics Parameter (PK): Cmax of axatilimab
Defined as maximum observed plasma concentration of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: Tmax of axatilimab
Defined as the time to reach the maximum plasma concentration of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: Cmin of axatilimab
Defined as the minimum observed plasma concentration of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: AUC(0-t) of axatilimab
Defined as the area under the concentration-time curve up to the last measurable concentration of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: AUC 0-∞ of axatilimab
Defined as the area under the concentration-time curve from 0 to infinity of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: CL of axatilimab
Defined as the apparent oral dose clearance of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: Vz of axatilimab
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
City of Hope Medical Center
Duarte, California, United States
RECRUITINGLucile Packard Child Hospital End
Palo Alto, California, United States
NOT_YET_RECRUITINGChildrens National Medical Center
Washington D.C., District of Columbia, United States
NOT_YET_RECRUITINGAdventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant
Orlando, Florida, United States
NOT_YET_RECRUITINGChildrens Healthcare of Atlanta
Atlanta, Georgia, United States
NOT_YET_RECRUITINGCongenital Heart Center At Mott Children'S Hospital
Ann Arbor, Michigan, United States
WITHDRAWNMemorial Sloan Kettering Cancer Center
New York, New York, United States
RECRUITINGLevine Cancer Institute
Charlotte, North Carolina, United States
NOT_YET_RECRUITINGChildrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
NOT_YET_RECRUITINGFred Hutchinson Cancer Research Center
Seattle, Washington, United States
NOT_YET_RECRUITING...and 32 more locations
Defined as the apparent oral dose volume of distribution of axatilimab.
Time frame: Up to 5 years
Pharmacokinetics Parameter: t1/2 of axatilimab
Defined as the apparent terminal phase disposition half-life of axatilimab.
Time frame: Up to 5 years
Best Overall Response (BOR)
Defined as 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 systemic therapy for cGVHD.
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
Duration of Response (DOR) (in responders only)
Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD, initiation 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
Organ-specific response
Organ-specific response as defined in the protocol.
Time frame: Up to 5 years
Percent reduction in daily corticosteroid dose at 6 months
Defined as Percent reduction in daily corticosteroid dose at Cycle 7 Day 1, and participants successfully tapered off all corticosteroids at Cycle 7 Day 1.
Time frame: 6 months
Changes in parameters collected using the pediatric stem cell quality of life (Q0L) questionnaire (PedsQL Stem Cell Transplant Module)
Time frame: Up to 5 years
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