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RGI-2001 for Preventing GVHD in Blood Cancer

Q: Could you please share the outcomes of prior studies that utilized RGI-2001?

Currently, 1092 tests are evaluating the efficacy of RGI-2001 with 196 trials in Phase 3. Notably, several research sites are situated within Philadelphia and 3098 total locations throughout the world have initiated clinical trials on this drug.

Q: Does the trial permit participants of advanced age to participate?

According to the indicated qualifications, only individuals aged between 18 and 80 may participate in this medical trial.

Q: In what ways does RGI-2001 typically assist patients?

RGI-2001 is commonly used to facilitate organ transplantation and can be administered for a range of other diseases, including but not limited to <a href="https://www.withpower.com/clinical-trials/multiple-sclerosis">multiple sclerosis</a>, <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, myelocytic acute rejection, kidney transplants.

Q: Is recruitment currently available for this research study?

Affirmative. Documents made available on clinicaltrials.gov demonstrate that this <a href="https://www.withpower.com/clinical-trials/experimental">experimental</a> intervention, which was first posted in August 2020, is presently recruiting candidates. The study plans to include 20 test subjects from a single location.

Q: Are there any opportunities to participate in this clinical experiment?

This clinical trial is seeking 20 persons aged 18 to 80 years old with a <a href="https://www.withpower.com/clinical-trials/myelodysplastic-syndrome">myelodysplastic syndrome</a>. The key requirements for participants additionally include: passing renal and cardiac function assessments, having an Eastern Cooperative <a href="https://www.withpower.com/clinical-trials/oncology">Oncology</a> Group (ECOG) performance status of two or lower, bilirubin levels below 2.5 mg/dL, normal values for alanine aminotransferase (ALT), aspartate aminotransaminase (AST), and alkaline phosphatase tests; plus undergoing haploidentical allogeneic hematopoietic cell transplantation from either a 1st

Q: How many participants is this clinical research recruiting?

Affirmative. According to clinicaltrials.gov, this clinical trial posted on August 14th 2020 is currently in need of participants. The last update was published on September 30 2021 and the study requires 20 individuals across 1 site.

Q: What sort of risks do patients face when using RGI-2001?

The risk associated with RGI-2001 has been evaluated and assigned a score of 1. This is due to the limited data that exists in regards to its safety profile and efficacy outcomes from Phase 1 trials.

Phase 1

Waitlist Available

Led By Zachariah DeFilipp, MD

Research Sponsored by Zachariah Michael DeFilipp

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with adequate physical function as measured by: Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction >25% Hepatic: Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or hemolysis ALT, AST, and Alkaline Phosphatase < 5 x ULN Renal: Serum creatinine within normal range, or if serum creatinine is outside normal range, then renal function (measured or estimated creatinine clearance or GFR) ≥ 40mL/min/1.73m2 Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted Ability to understand and the willingness to sign a written informed consent document

- Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 100 days

Awards & highlights

Study Summary

This trial is studying RGI-2001 to see how well it works in preventing GVHD in people with blood cancer who will have a blood stem cell transplant.

Who is the study for?

Adults aged 18-80 with certain blood cancers (like AML, ALL, MDS, MPN, CMML) in remission or with low disease activity and those with chemosensitive Hodgkin's or Non-Hodgkin's lymphoma. Participants must be undergoing a specific type of stem cell transplant from a half-matched relative and have good heart, liver, kidney, and lung function.Check my eligibility

What is being tested?

The trial is testing RGI-2001 to prevent Graft-vs-Host Disease (GVHD), which can occur after stem cell transplants when donor cells attack the recipient's body. It involves patients who are receiving transplants due to various hematological conditions.See study design

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions related to immune system activation such as fever or fatigue; organ inflammation; increased risk of infections; and complications from the underlying condition being treated.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My lung function tests are at least half of what is expected for someone my age and size.

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I have been diagnosed with a blood cancer.

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I am getting a stem cell transplant from a family member who is a partial match.

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My heart pumps well enough, meeting the minimum required efficiency.

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My lymphoma responds well to chemotherapy.

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My kidney function is normal or meets the minimum required level.

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I can take care of myself but might not be able to do heavy physical work.

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I am between 18 and 80 years old.

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My leukemia is currently in complete remission.

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I have a blood cancer in remission or with low disease activity.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 100 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~100 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 100 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of patients achieving successful donor engraftment

Secondary outcome measures

100-day non-relapse mortality (NRM) rate.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Regimen 2: Fludarabine, Melphalan, and TBIExperimental Treatment7 Interventions

-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician Pre- stem cell transplant: Fludarabine predetermined dose, intravenously 3 times per cycle Melphalan, infusion, determined dosage, once per cycle Total body irradiation (TBI) once per cycle. Post stem cell transplant Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle RGI-2001: IV, predetermined dose, weekly to 6 total doses

Group II: Regimen 1: Fludarabine, Cyclophosphamide, and TBIExperimental Treatment6 Interventions

-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician Pre- stem cell transplant: Fludarabine predetermined dose, intravenously, 4 times per cycle Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion Total body irradiation (TBI) once during treatment cycle Post stem cell transplant: Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral. Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle RGI-2001: IV, predetermined dose, weekly to 6 total doses

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

KRN-7000

Not yet FDA approved

Cyclophosphamide

FDA approved

Melphalan

FDA approved

TBI

2014

Completed Phase 2

~1040

Sirolimus

FDA approved

Mycophenolate mofetil

FDA approved

0 / 11Eligibility criteria met

Find a Location

Who is running the clinical trial?

Zachariah Michael DeFilippLead Sponsor

1 Previous Clinical Trials

8 Total Patients Enrolled

Regimmune CorporationIndustry Sponsor

2 Previous Clinical Trials

117 Total Patients Enrolled

Zachariah DeFilipp, MDPrincipal InvestigatorMassachusetts General Hospital

5 Previous Clinical Trials

123 Total Patients Enrolled

Media Library

RGI-2001 Clinical Trial Eligibility Overview. Trial Name: NCT04473911 — Phase 1

Graft-versus-Host Disease Research Study Groups: Regimen 2: Fludarabine, Melphalan, and TBI, Regimen 1: Fludarabine, Cyclophosphamide, and TBI

Graft-versus-Host Disease Clinical Trial 2023: RGI-2001 Highlights & Side Effects. Trial Name: NCT04473911 — Phase 1

RGI-2001 2023 Treatment Timeline for Medical Study. Trial Name: NCT04473911 — Phase 1

Frequently Asked Questions

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

Could you please share the outcomes of prior studies that utilized RGI-2001?

"Currently, 1092 tests are evaluating the efficacy of RGI-2001 with 196 trials in Phase 3. Notably, several research sites are situated within Philadelphia and 3098 total locations throughout the world have initiated clinical trials on this drug."

Answered by AI

Does the trial permit participants of advanced age to participate?

"According to the indicated qualifications, only individuals aged between 18 and 80 may participate in this medical trial."

Answered by AI

In what ways does RGI-2001 typically assist patients?

"RGI-2001 is commonly used to facilitate organ transplantation and can be administered for a range of other diseases, including but not limited to multiple sclerosis, leukemia, myelocytic acute rejection, kidney transplants."

Answered by AI

Is recruitment currently available for this research study?

"Affirmative. Documents made available on clinicaltrials.gov demonstrate that this experimental intervention, which was first posted in August 2020, is presently recruiting candidates. The study plans to include 20 test subjects from a single location."

Answered by AI

Are there any opportunities to participate in this clinical experiment?

"This clinical trial is seeking 20 persons aged 18 to 80 years old with a myelodysplastic syndrome. The key requirements for participants additionally include: passing renal and cardiac function assessments, having an Eastern Cooperative Oncology Group (ECOG) performance status of two or lower, bilirubin levels below 2.5 mg/dL, normal values for alanine aminotransferase (ALT), aspartate aminotransaminase (AST), and alkaline phosphatase tests; plus undergoing haploidentical allogeneic hematopoietic cell transplantation from either a 1st"

Answered by AI

How many participants is this clinical research recruiting?

"Affirmative. According to clinicaltrials.gov, this clinical trial posted on August 14th 2020 is currently in need of participants. The last update was published on September 30 2021 and the study requires 20 individuals across 1 site."

Answered by AI

What sort of risks do patients face when using RGI-2001?

"The risk associated with RGI-2001 has been evaluated and assigned a score of 1. This is due to the limited data that exists in regards to its safety profile and efficacy outcomes from Phase 1 trials."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Haplo Peripheral Blood Sct In GVHD Prevention

Zachariah Michael DeFilipp 2020. "Haplo Peripheral Blood Sct In GVHD Prevention". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04473911.

All > Gvhd > Myelodysplastic Syndrome