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RGI-2001 for Preventing GVHD in Blood Cancer

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

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

Diagnosis of hematological malignancy:

Must not have

Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.

Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, recent myocardial infarction or stroke, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 100 days

Awards & highlights

Approved for 20 Other Conditions

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any active infections needing treatment.

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I do not have any severe illnesses that would stop me from following the study's requirements.

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I do not have HIV, Hepatitis B, or Hepatitis C.

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I am scheduled to receive preventive DLI therapy.

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I am not pregnant or breastfeeding.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of patients achieving successful donor engraftment

Secondary study objectives

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

Side effects data

From 2023 Phase 2 trial • 49 Patients • NCT04014790

22%

Nausea

20%

Rash

18%

Stomatitis

18%

Diarrhea

18%

Vomiting

18%

Decreased Appetite

14%

Abdominal Pain

14%

Fatigue

14%

Platelet Count Decreased

12%

Muscosal Inflammation

12%

Anaemia

12%

Dyspnoea

12%

Alanine Aminotransferase increased

12%

Neutrophil Count Decreased

12%

Hypomagnesaemia

12%

White blood cell count decreased

10%

Dysgeusia

Hyponatremia

Headache

Hyperglycemia

Dry Skin

Dizziness

Pruritis

Constipation

Hyperphosphataemia

Aspartate Aminotransferase increased

Blood Alkaline Phosphatase Increased

Blood Lactate Dehyrogenase increased

Hypophosphataemia

Blood Creatinine increased

Tremor

Hypertension

Pulmonary veno-occlusive disease

Myocarditis

Septic Shock

Pelvic Pain

Deep Vein Thrombosis

Upper respiratory infection

100%

80%

60%

40%

20%

Study treatment Arm

RGI-2001

Awards & Highlights

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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