RGI-2001 for Graft Versus Host Disease (cGvHD)

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Massachusetts General Hospital, Boston, MA
Graft Versus Host Disease (cGvHD)+4 More
RGI-2001 - Drug
Eligibility
18 - 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug may help prevent graft-versus-host disease following a stem cell transplant.

See full description

Eligible Conditions

  • Graft Versus Host Disease (cGvHD)
  • Prevention of aGVHD
  • Acute Graft-Versus-Host Disease (GVHD)
  • Graft-versus-host-disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Graft Versus Host Disease (cGvHD)

Study Objectives

This trial is evaluating whether RGI-2001 will improve 5 secondary outcomes in patients with Graft Versus Host Disease (cGvHD). Measurement will happen over the course of Day 180 post-transplant.

Year 1
Disease-free survival (DFS)
GvHD-free, relapse free survival (GRFS)
Overall survival (OS)
Year 1
Non-relapse mortality (NRM) rates
Day 180 post-transplant
Grades II-IV aGVHD

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Graft Versus Host Disease (cGvHD)

Trial Design

1 Treatment Group

RGI-2001
1 of 1
Experimental Treatment

This trial requires 49 total participants across 1 different treatment group

This trial involves a single treatment. RGI-2001 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

RGI-2001Subjects will be administered RGI 2001 in combination with standard of care treatment
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
KRN-7000
Not yet FDA approved
Standard of Care
2017
Completed Phase 4
~9880

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months and 1 year post-transplant
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 6 months and 1 year post-transplant for reporting.

Closest Location

Massachusetts General Hospital - Boston, MA

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Must have adequate organ function
Transplant Donor: Matched related donor or Unrelated donor Is a candidate for anti-graft-vs-host-disease (GvHD) prophylaxis that includes a calcineurin inhibitor Ability to understand and willingness to sign a written informed consent form
Ages ≥ 18 and ≤ 65 years of age
Has a hematologic malignancy which includes Acute myelogenous leukemia (AML), T or B cell acute lymphoblastic leukemia (ALL) Myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), myeloproliferative disorder (MPD) including myeloid metaplasia and CML
If female of childbearing potential, must have had a negative serum pregnancy test prior to enrollment and must have agreed to use a double barrier method of contraception for 30 days after RGI-2001 administration
If male, must be sterile or willing to use an approved method of contraception from the time of informed consent to 90 days after last dose of RGI-2001 administration

Patient Q&A Section

Can graft vs host disease be cured?

"Our patients with GVHD received only a single dose of dexamethasone; this treatment resulted in complete resolution of all symptoms for 16 of 22 patients. The clinical utility of this modality merits confirmation in larger prospective study. If further study confirms these promising preliminary results, patients with GVHD and multiple organ failure may have a chance for improved outcomes if treated with dexamethasone." - Anonymous Online Contributor

Unverified Answer

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

"About 25.8 million transplant patients and 20.4 million non-transplant patients are at risk of acquiring transplant-related GVHD. This makes up 13.2% and 23.2% of all living adults, respectively.\n" - Anonymous Online Contributor

Unverified Answer

What are the signs of graft vs host disease?

"Symptoms of GvHD may include skin rashes, mouth sores, flu-like symptoms, diarrhea, sore eyes, hair loss, swollen lymph nodes and swollen tonsils. The clinical course of GvHD is very variable. Some patients have only mild symptoms and those with serious GvHD will recover, but in many cases GvHD is permanent and has a significant impact on the quality of life. There are numerous diagnostic and scoring systems available to help guide clinicians with the diagnosis and treatment of GvHD. However, none currently incorporate the clinical features of this disorder. Therefore, it is not always possible to predict the course of GvHD in any given individual." - Anonymous Online Contributor

Unverified Answer

What is graft vs host disease?

"Graft vs host disease in transplants is not universal as demonstrated by the presence of GVHD in transplants but not in host-matched transplants. GVHD in transplants is more likely to result from Graft vs GVH transplantation or from a combination of both causes." - Anonymous Online Contributor

Unverified Answer

What causes graft vs host disease?

"It is unclear why patients with BMT who are genetically susceptible will develop this condition. It appears to be due to the body's own mechanisms rather than environmental triggers. GvHD can occur from a donor's cells in the graft." - Anonymous Online Contributor

Unverified Answer

What are common treatments for graft vs host disease?

"Both conventional chemotherapy and alemtuzumab have been found to be effective in alleviating GVHD symptoms. Other medications used to treat GVHD include methotrexate, mercaptopurine, tacrolimus, etanercept, and ibuprofen. Steroids also play a part in the treatment of GVHD. Treatments are effective not only because they can alleviate side effects, but also to prevent GVHD because it appears to have the opposite effect.\n\nGraft vs host disease affects 1 to 2 percent of all adult transplants. Approximately 95 percent of patients develop GVHD symptoms." - Anonymous Online Contributor

Unverified Answer

What is rgi-2001?

"rgi-1981 (anti-alphav integrin) represents a novel human endogenous retroviral envelope glycoprotein of ~43KD. As with the alpha-fusobacterial env glycoproteins, rgi-1981 may serve as an envelope glycoprotein for virion attachment to a specific cell type. Although rgi-0181 has not yet been functionally investigated, the fact that the gag gene has been disrupted in this retroviral genome may mean that a functional envelope, albeit not associated with any infectivity factor, is required for trans-packaging of the genome into the mature virion." - Anonymous Online Contributor

Unverified Answer

How does rgi-2001 work?

"Rgi-2001 can be a safe, highly-targeted agent for the induction and enhancement of tumor-specific T-cell responses, while concurrently preventing graft vs. host disease in vivo. This combination of properties can provide a potential novel therapeutic strategy to combat GVHD-associated cancers, and in future studies, combining Rgi-2001 with other immunomodulatory agents or strategies may further increase the potential of these immunotherapies for the treatment of acute and chronic disease states." - Anonymous Online Contributor

Unverified Answer

Is rgi-2001 safe for people?

"G-2 is a promising candidate for the treatment of patients with GvHD. However, in this study of 36 patients, no fatalities were anticipated. The study also showed that G-2 reduces the likelihood of myelosuppression and the severity of the symptoms. More studies of patients after G+96 treatment are needed." - Anonymous Online Contributor

Unverified Answer

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

"It has been very hard to treat and to prevent GvHD with donor T-cells. Recent approaches are being evaluated, however there are not many treatment options, and donor T-cells are still the best that can be used. It is essential to get transplants in order to keep patients healthy, but to start treatment for patients, it is still to be determined which treatments are effective. There are some things on the table that will help patients. When evaluating, the patient’s age, GvHD phenotype, clinical stage, treatment intent, graft type, donor type, and gender will be taken into consideration." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of graft vs host disease?

"The current investigation did not find support for the hypothesis that the aetiology of GVHD was primary to the aetiology of the GVHD. This suggests that the primary aetiological factors leading to GVHD occur during the early part of the infection and not after immunity has developed against specific microbes, and may be an important prognostic indicator" - Anonymous Online Contributor

Unverified Answer

Does rgi-2001 improve quality of life for those with graft vs host disease?

"This preliminary study supports the use of Rgi-2001 and indicates that this treatment may be a useful adjunct therapy for improving quality of life in those with GvHD. Further research is indicated on longer-term use, side-effects and efficacy of this treatment." - Anonymous Online Contributor

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