Emapalumab for Graft Rejection Prevention in Bone Marrow Transplant
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests emapalumab, a drug researchers believe can help prevent graft rejection after a bone marrow transplant. It targets patients at high risk of rejection, likely due to specific donor matches or past rejection experiences. Participants will receive either a low or high dose of the drug to determine which is more effective. High-risk individuals include those with mismatched donors or a history of rejection. As an Early Phase 1 trial, this research aims to understand how emapalumab works in people, offering participants the opportunity to be among the first to receive this potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that emapalumab may help prevent graft failure in patients undergoing bone marrow transplants. In earlier studies, emapalumab managed patients showing signs of graft failure and showed promising results in stopping these failures. These studies suggest that emapalumab might work by reducing the risk of graft rejection, as it targets interferon gamma, a substance believed to play a key role in the rejection process.
Although detailed safety data from large trials is not yet available, emapalumab has already received FDA approval for other conditions, such as certain immune system disorders. This approval indicates that it has been checked for safety in those contexts. However, since this study is in the early testing stages, more specific safety information for bone marrow transplant patients is still needed. Participants should discuss potential risks and benefits with their healthcare providers before joining the trial.12345Why are researchers excited about this trial's treatment?
Researchers are excited about emapalumab for preventing graft rejection in bone marrow transplant patients because it targets interferon gamma, a key player in immune response. Unlike standard immunosuppressive treatments, which broadly suppress the immune system, emapalumab specifically binds to this particular cytokine, potentially reducing unwanted immune activity with more precision. This targeted approach could mean fewer side effects and better protection against graft rejection, making it a promising option for patients undergoing transplants.
What evidence suggests that emapalumab might be an effective treatment for graft rejection prevention?
Research has shown that emapalumab might help prevent the body from rejecting a new bone marrow transplant. In some studies, 56% of patients using emapalumab during signs of graft failure retained their new bone marrow. This medication blocks a protein called interferon gamma, which plays a significant role in graft rejection. In children, emapalumab use led to fewer issues with mixed chimerism—a condition where new and old cells don't fully blend—compared to those who didn't use it. Overall, emapalumab appears promising in aiding transplant success by targeting specific immune responses. Participants in this trial will receive either 3 mg/kg or 10 mg/kg of emapalumab, with the possibility of additional rescue doses if needed.26789
Who Is on the Research Team?
Anthony Sabulski, MD
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Are You a Good Fit for This Trial?
This trial is for patients at high risk of graft rejection after a bone marrow transplant. To join, they must have two or more risk factors like having a mismatched donor, receiving a T-cell depleted graft, or having rejected a graft before.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive emapalumab intravenously on day +1 after HSCT, with potential rescue doses if signs of graft rejection occur
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Participants are monitored for overall survival and incidence of infections and mixed chimerism
What Are the Treatments Tested in This Trial?
Interventions
- Emapalumab
Trial Overview
The study tests if using Emapalumab to block interferon gamma can prevent graft rejection in high-risk HSCT recipients. Two different doses (3 mg/kg and 10 mg/kg) are being studied to find the best way to stop this from happening.
How Is the Trial Designed?
2
Treatment groups
Active Control
Patients randomized to this arm will receive 3mg/kg of emapalumab intravenously (IV) once on day +1 after HSCT. Up to two additional, 10mg/kg rescue doses may be administered if patients developed signs and symptoms of acute graft rejection. Rescue dose administration decisions will be made in consultation with the lead study investigator. Emapalumab is a ligand-based therapy, which means high levels of circulating ligand (i.e. interferon gamma) will rapidly consume the drug. For these reasons, rescue doses may be given as early as 24 hours from the prior dose.
Patients randomized to this arm will receive 10mg/kg of emapalumab intravenously (IV) once on day +1 after HSCT. Up to two additional, 10mg/kg rescue doses may be administered if patients developed signs and symptoms of acute graft rejection. Rescue dose administration decisions will be made in consultation with the lead study investigator. Emapalumab is a ligand-based therapy, which means high levels of circulating ligand (i.e. interferon gamma) will rapidly consume the drug. For these reasons, rescue doses may be given as early as 24 hours from the prior dose.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
Sobi, Inc.
Industry Sponsor
Citations
1.
ashpublications.org
ashpublications.org/blood/article/142/Supplement%201/114/500105/Emapalumab-for-Treatment-of-Impending-GraftEmapalumab for Treatment of Impending Graft Failure | Blood
In this study we investigate the use of emapalumab to prevent immune mediated graft failure after hematopoietic stem cell transplant.
2.
oncologynurseadvisor.com
oncologynurseadvisor.com/news/emapalumab-for-management-post-hsct-graft-failure/Emapalumab for Management of Post-HSCT Graft Failure ...
Among the patients treated with emapalumab for impending graft failure, 56% (14 of 25) successfully engrafted and resolved concerns related to ...
Emapalumab to Prevent Graft Failure in High-Risk Patients
Three GF patients successfully engrafted after 2nd HSCT without needing further doses of emapalumab. The last GF patient was found to have persistent leukemia ...
4.
scienceblog.cincinnatichildrens.org
scienceblog.cincinnatichildrens.org/emapalumab-extends-intervention-free-survival-for-children-with-hlh/Emapalumab Extends 'Intervention-Free' Survival for Children ...
The use of emapalumab was associated with a markedly lower incidence of mixed chimerism (48% vs. 77%) and of severe mixed chimerism (5% vs. 38%) ...
Emapalumab in Children with Primary Hemophagocytic ...
In the previously treated group, 70% of the patients were able to proceed to transplantation, as were 65% of the patients who received ...
Prevention of Graft Rejection in Hematopoietic Stem Cell ...
Emapalumab is a ligand-based therapy, which means high levels of circulating ligand (i.e. interferon gamma) will rapidly consume the drug. For these reasons, ...
Emapalumab treatment in an ADA-SCID patient with refractory ...
Moreover, emapalumab has shown promising efficacy in the treatment of patients with graft failure (GF) requiring a second allogeneic hematopoietic stem cell ...
Emapalumab for Treatment of Impending Graft Failure
In this study we investigate the use of emapalumab to prevent immune mediated graft failure after hematopoietic stem cell transplant.
Prevention of Graft Rejection in Hematopoietic Stem Cell ...
The purpose of this study is to learn more about emapalumab and its ability to prevent graft rejection in hematopoietic stem cell transplant ( ...
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