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Compares treatment options for Chronic Graft-versus-host-disease
Official title A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
ClinicalTrials.gov ID: NCT04710576
What this study is testing
What is Axatilimab?
Axatilimab is an investigational medicine, being studied as a potential treatment for chronic graft-versus-host-disease.
Also referred to as Niktimvo, SNDX-6352.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 2 years
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- Participants must be 2 years of age or older, at the time of signing the informed consent.
- Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression...
- Participants with refractory or recurrent active cGVHD despite at least 2 lines of systemic therapy.
- Refractory disease defined as meeting any of the following criteria:
- The development of 1 or more new sites of disease while being treated for cGVHD.
You likely can't join if
- Participants are excluded from the study if any of the following criteria apply:
- Has acute GVHD without manifestations of cGVHD.
- Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative...
- History of acute or chronic pancreatitis.
- History of myositis.
- History or other evidence of severe illness, uncontrolled infection or any other conditions that would make the participant, in the opinion of the...
See the full eligibility criteria
- Participants must be 2 years of age or older, at the time of signing the informed consent.
- Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression. Active cGVHD is defined as the presence of signs and symptoms of cGVHD...
- Participants with refractory or recurrent active cGVHD despite at least 2 lines of systemic therapy.
- Refractory disease defined as meeting any of the following criteria:
- The development of 1 or more new sites of disease while being treated for cGVHD.
- Progression of existing sites of disease despite at least 1 month of standard or investigation therapy for cGVHD.
- Participants who have not achieved a response within 3 months on their prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.
- Recurrent cGVHD is active, symptomatic disease (after an initial response to prior therapy) as defined, based on the NIH 2014 consensus criteria, by organ-specific or global assessment or for which the physician...
- Participants may have persistent, active acute and cGVHD manifestations (overlap syndrome), as defined by 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD.
- Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged \<16 years)
- Adequate organ and bone marrow functions evaluated during the 14 days prior to randomization.
- Creatinine clearance (CrCl) ≥30 milliliter/minute based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Concomitant use a of systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking corticosteroids at study randomization, they must be on a...
- Concomitant use of CNI or mammalian target of repamycin (mTOR) inhibitors (sirolimus or everolimus) is allowed but not required.
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. A parent/guardian should provide consent for pediatric...
- Participants are excluded from the study if any of the following criteria apply:
- Has acute GVHD without manifestations of cGVHD.
- Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
- History of acute or chronic pancreatitis.
- History of myositis.
- History or other evidence of severe illness, uncontrolled infection or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study.
- Participants with acquired immune deficiency syndrome (AIDS).
- Hepatitis B (defined as hepatitis B virus [HBV] surface antigen positive and HBV core antibody positive, with positive HBV deoxyribonucleic acid [DNA], or HBV positive core antibody alone with positive HBV DNA...
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of randomization, unless previously treated with curative intent and approved by Sponsor's Medical Monitor (for...
- Female participant who is pregnant or breastfeeding.
- Previous exposure to CSF1-R targeted therapies.
- Taking agents for treatment of cGVHD other than corticosteroids or either a CNI or mTOR inhibitor is prohibited.
- For approved or commonly used agents, other than corticosteroids, CNI and mTOR inhibitor, a washout of 2 weeks or 5 half-lives, whichever is shorter, is required at study enrollment.
- Receiving another investigational treatment within 28 days of randomization.
- Participants should not be participating in any other treatment study. Pediatric participants are encouraged to also participate in the ongoing developmental studies of the Pediatric cGVHD Symptom Scale (PCSS).
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Duarte, California, United States
- Los Angeles, California, United States
- Stanford, California, United States
- Washington D.C., District of Columbia, United States
- Gainesville, Florida, United States
- Jacksonville, Florida, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- Tampa, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- New Orleans, Louisiana, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Worcester, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Detroit, Michigan, United States
- Minneapolis, Minnesota, United States
+ 78 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Duarte, California, United States; Los Angeles, California, United States; Stanford, California, United States; Washington D.C., District of Columbia, United States; Gainesville, Florida, United States and 92 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.