CLINICAL TRIAL

Efavaleukin Alfa for Graft vs Host Disease

Waitlist Available · 18+ · All Sexes · Chuo-ku, Japan

Safety and Efficacy of Efavaleukin Alfa in Subjects With Steroid Refractory Chronic Graft Versus Host Disease

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About the trial for Graft vs Host Disease

Eligible Conditions
Chronic Graft Versus Host Disease cGVHD · Graft vs Host Disease

Treatment Groups

This trial involves 2 different treatments. Efavaleukin Alfa is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Experimental Group 1
Efavaleukin Alfa
DRUG
Experimental Group 2
Efavaleukin Alfa
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Efavaleukin alfa
Not yet FDA approved

Eligibility

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The subject is a recipient of an allogeneic HSCT, which is a complex and intensive treatment for cancer and other serious diseases show original
Moderate to severe cGVHD was present at the screening visit in accordance with the 2014 cGVHD NIH Consensus Criteria, with involvement of at least one of the following organs: skin, mouth, eyes, GI tract, liver, lungs, and joint and fascia. show original
The patient has only received three treatments for cGVHD in the past, excluding topical treatments. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 52 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 52 weeks.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Efavaleukin Alfa will improve 3 primary outcomes and 4 secondary outcomes in patients with Graft vs Host Disease. Measurement will happen over the course of 4 weeks following first dose per subject.

Phase 1b: Incidence of dose limiting toxicities
4 WEEKS FOLLOWING FIRST DOSE PER SUBJECT
Calculate incidence of dose limiting toxicities (DLT's) for all subjects who complete the 4 weeks following their first dose of efavaleukin alfa within each cohort. To be evaluable for a DLT subjects must have received at least 2 doses of efavaleukin alfa or have experienced a DLT within the DLT evaluation period.
Phase 2: Changes in quality of life
52 WEEKS
Measure changes in quality of life as measured by Karnofsky performance status.
Phase 2: Changes in symptom burden over time
52 WEEKS
Evaluate changes in symptom burden as measured by the Lee Symptom Scale. A change of 6 to 7 points on the Lee Symptom Scale will be considered clinically significant and relates to improvement in quality of life.
Phase 2: Failure free survival rate
52 WEEKS
Evaluate failure free survival, defined as absence of relapse, death, or need for additional systemic immunosuppressant cGVHD therapy
Phase 2: Best overall response rate defined as the proportion of subjects achieving a Complete Response (CR) or Partial Response (PR) during the study according to the 2014 cGVHD NIH Consensus Criteria
52 WEEKS
Evaluate efficacy as measured by the best overall response rate during the study according to the 2014 cGVHD National Institutes of Health Consensus Criteria
Phase 1b: Incidence of all treatment-related and treatment-emergent adverse events and serious adverse events
52 WEEKS
Evaluate safety of efavaleukin alfa
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of graft vs host disease?

In all cases of GVHD there are at least two criteria that must be met:\n1. The condition must be newly acquired and\n2. The manifestations must be new and unprolonged.\nSigns may appear in the three months following graft: skin rashes, gastrointestinal problems, and fever. Other signs and symptoms may appear following many months and may take months to resolve, including a chronic feeling of being unwell. The cause may be from the graft which manifests as a skin rash or GI problems. Sometimes the GVHD is so severe it affects vital organs or can't be controlled.

Anonymous Patient Answer

What are common treatments for graft vs host disease?

A large majority of GvsHD treatment protocols include prednisone, an antimetabolite/cytarabine, and methotrexate, but many GvsHD protocols use two or even three therapies. Most of the common treatments for GvsHD are supportive, such as maintenance steroids and antimetabolites/cytarabines. However, it is vital that all patients remain on immunosuppressants as they are likely to be needed to prevent GvsHD if they relapse. Other common treatment for GvsHD include donor lymphocyte infusion and the aforementioned immunosuppressants.

Anonymous Patient Answer

How many people get graft vs host disease a year in the United States?

The incidence of autoimmune diseases was similar to the reported rates in other populations. Results from a recent clinical trial were also comparable with published rates in other countries. This suggests that some of the risk for autoimmune diseases are genetically determined and are at least likely to be modified by environmental factors.

Anonymous Patient Answer

What causes graft vs host disease?

A major factor in the pathogenesis of GvHD is a direct response of T cells to the donor antigen prior to their engraftment. The role of T-helper-1 cells and the cytokine IL-2 in GvHD pathogenesis was also discussed.

Anonymous Patient Answer

What is graft vs host disease?

Graft vs host disease is a common complication after hematopoietic stem cell transplantation and is characterized by graft-versus-host cell-mediated tissue damage and abnormal host function. The main causes of GvHD are the donor-type recipient mismatch and the T-cell deficiency in the immunosuppressive therapy. It is also associated with a wide array of conditions involving donor-type specific immune response, resulting in different types of GvHD.

Anonymous Patient Answer

Can graft vs host disease be cured?

It has been suggested that the presence of a GVHD score of 3 or greater is a significant negative prognosticator of GVHD occurrence. We found no evidence of a statistically significant negative association between GVHD and survival after a median follow-up of more than a decade. As with other medical conditions often associated with GVHD, GVHD does not prove to be a significant clinical predictor of survival. However, these associations should be confirmed in a pooled prospective study of the entire population.

Anonymous Patient Answer

Does efavaleukin alfa improve quality of life for those with graft vs host disease?

Treatment with either EFA or EFA-IV improves most aspects of HRQOL compared with the placebo in patients with GVHD, and the magnitude of improvement in specific HRQOL questions is similar. These data provide the impetus for larger and longer-term studies of EFA as a potential treatment for GVHD.

Anonymous Patient Answer

What is the average age someone gets graft vs host disease?

The average age of people who get GVHD can be ascertained by obtaining the report card of a hospital that treats a patient with GVHD. The [Age] of GVHD in the report card is used to estimate the average age of people who get GVHD. You can look at the [Age] estimates of [different types of GVHD] on the [Graft vs Host Disease] website.

Anonymous Patient Answer

Is efavaleukin alfa typically used in combination with any other treatments?

EFA is often used in combinatorial approaches with other treatments. The most common combination is with prednisone, as EFA appears to provide a high likelihood of achieving a complete remission with that combination. EFA has also been reported to be used as a first-line immunosuppressive treatment.

Anonymous Patient Answer

What is the primary cause of graft vs host disease?

In a recent study, findings suggests that GVHD is in part due to a graft vs host response to the haemopoietic stem cell transplants and that the host response would be more of a significant risk factor for the development of the disease. Patients who are GVHD-positive have significantly less prolonged remission and more relapses when compared to GVHD negative patients.

Anonymous Patient Answer

Have there been any new discoveries for treating graft vs host disease?

We [have] had many new discoveries in the last 5 years. [New treatments have] all [been found through] systematic research on GvHD in [all] kinds of cancers [and] solid and hematological malignancies; there [is] a real understanding of how the immune system behaves in cancer and how its behavior can be modified. [We have also] made significant advances in the understanding of how the different T cells and antibodies can help [by targeting different cells] and what the signals can be to help the immune system attack the target.

Anonymous Patient Answer

Who should consider clinical trials for graft vs host disease?

It is important that all patients undergoing HT for chronic lymphocytic leukemia are discussed by their treating physician with regard for trial eligibility. Many patients, especially younger patients, have the opportunity to enroll in trial protocols but choose not to complete such trials.

Anonymous Patient Answer
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