A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection

Study ID

Cancer Related

Healthy Volunteers

Study Sites

  • Clements University Hospital
  • UT Southwestern Ambulatory Services
  • Zale Lipshy University Hospital

Joyce Bolluyt

Principal Investigator
Muhammad Beg, M.D.

Official Title

A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection

Brief Overview

This is a study to evaluate the safety of MRX34 in patients with primary liver cancer or
other selected solid tumors or hematologic malignancies. The drug is given intravenously,
for 5 days in a row and then two weeks off.


This is a Phase I, open-label, multicenter, dose-escalation study to investigate the safety,
Pharmacokinetics and Pharmacodynamics of the micro ribonucleic acid (microRNA) MRX34, in
patients with unresectable primary liver cancer or advanced or metastatic cancer with or
without liver involvement or hematologic malignancies. MRX34 will be administered daily x 5
with 2 weeks off (total of 21 days) for 3 cycles followed by a no-treatment observation

Participant Eligibility

Inclusion Criteria:

1. Aged ≥ 18 years

2. Patients with histologically confirmed viral related hepatocellular, SCLC,
non-cutaneous/ non-uveal melanoma, ovarian, TNBC, Sarcoma, Bladder and RCC.

3. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

4. Acceptable liver function:

- Total bilirubin ≤ 1.5 times the upper limit of normal (ULN); for patients with
hepatocellular carcinoma only, total bilirubin ≤ 3 mg/dL (i.e. Child-Pugh Score
for bilirubin is no greater than 2).

- Aspartate aminotransferase (AST), alanine aminotransferase (ALT) and alkaline
phosphatase (ALP) ≤ 5 x ULN.

5. Acceptable renal function:

• Serum creatinine ≤ 1.5 times the ULN, or calculated creatinine clearance ≥ 60
mL/min/1.73 m2 for patients with creatinine levels above 1.5 times the institutional

6. Acceptable hematological status:

- Absolute Neutrophil Count (ANC) ≥ 1500 cells/mm3

- Platelet count ≥ 100,000 plts/mm3 (without transfusion); ≥ 75,000 plts/mm3 for
patients with hepatocellular carcinoma only. For hematologic malignancy patients
blood counts cited above do not apply

- Hemoglobin ≥ 9 g/dL

- For the hematologic malignancy patients, blood count values cited above do not

7. Prothrombin time (PT) or International Normalized Ratio (INR) ≤ 1.25 x ULN; for
patients with hepatocellular carcinoma only, INR <1.7 or prothrombin time (PT) or < 4
seconds above ULN (i.e. Child-Pugh Score is no greater than 1 for the coagulation
parameter); for patients with hepatocellular carcinoma only, serum albumin > 2.8 g/dL
(i.e. Child-Pugh Score for albumin is no greater than 2). For the hematologic
malignancy patients, the coagulation and albumin status cited above do not apply

8. For patients with hepatocellular carcinoma only, Child-Pugh Class A (score 5-6)
disease. Score for hepatic encephalopathy must be 1; the score for ascites must be no
greater than 2 and clinically irrelevant; for the determination of the Child-Pugh

Exclusion Criteria:

1. Myocardial infarction within the past 6 months, unstable and/or symptomatic
arrhythmia, or evidence of ischemia on ECG.

2. Active, uncontrolled bacterial, viral, or fungal infections requiring systemic

3. Pregnant or nursing women.

4. Known infection with human immunodeficiency virus (HIV).

5. Serious nonmalignant disease (e.g., hydronephrosis, liver failure, heart failure, or
other conditions) that could compromise protocol objectives in the opinion of the
investigator and/or the sponsor.

6. Patients with recent history of hemorrhage and patients predisposed to hemorrhage due
to coagulopathies or structural anomalies.

7. Patients who require treatment with therapeutic doses of coumadin-type anticoagulants
(maximum daily dose of 1mg allowed for port line patency permitted).

8. Patients with cirrhosis classed as Child-Pugh B or C.

9. Patients with central nervous system (CNS) metastasis. Intrathecal chemotherapy is
allowed for patients who require CNS prophylaxis or therapy.

10. Patients for whom dexamethasone is contraindicated.