Primary Liver CancerPhase 1NCT01829971
A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection
A Phase 1 study testing MRX34 in people with Primary Liver Cancer and SCLC, run by Mirna Therapeutics, Inc. at 10 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Liver Cancer, SCLC, Lymphoma
- What is being tested?
- MRX34
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 155 participants
- Where
- 10 sitesin 2 countries
- Study dates
- Apr 2013to May 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged ≥ 18 years
- Patients with histologically confirmed viral related hepatocellular, SCLC, non-cutaneous/ non-uveal melanoma, ovarian, TNBC, Sarcoma, Bladder and RCC.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
- 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.
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- 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 normal
- 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 apply.
- 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
- 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 Class.
Who cannot join
- Myocardial infarction within the past 6 months, unstable and/or symptomatic arrhythmia, or evidence of ischemia on ECG.
- Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy.
- Pregnant or nursing women.
- Known infection with human immunodeficiency virus (HIV).
- 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.
- Patients with recent history of hemorrhage and patients predisposed to hemorrhage due to coagulopathies or structural anomalies.
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- Patients who require treatment with therapeutic doses of coumadin-type anticoagulants (maximum daily dose of 1mg allowed for port line patency permitted).
- Patients with cirrhosis classed as Child-Pugh B or C.
- Patients with central nervous system (CNS) metastasis. Intrathecal chemotherapy is allowed for patients who require CNS prophylaxis or therapy.
- Patients for whom dexamethasone is contraindicated.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States6 sites
Texas Oncology Dallas
Dallas, Texas, 75246
UT Southwestern Medical Center
Dallas, Texas, 75390
MD Anderson Cancer Center
Houston, Texas, 77030
Uthscsa/Ctrc
San Antonio, Texas, 78229
Virginia G. Piper Cancer Center
Scottsdale, Arizona, 85258
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
South Korea4 sites
Severance Hospital, Yonsie University Health System
Seoul, Seodaemun-Gu, 03722
Seoul National University Hospital
Seoul, 110-744
Samsung Medical Center
Seoul, 135-710
Asan Medical Center
Seoul, 138-736
About this study
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.
Read the detailed description
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 period.
- Conditions
- Primary Liver Cancer, SCLC, Lymphoma, Melanoma, Multiple Myeloma, Renal Cell Carcinoma
- Being tested
- MRX34
- Keywords
- microRNA, Advanced cancer
- Medical subject headings
- Carcinoma, Hepatocellular, Lymphoma, Melanoma, Multiple Myeloma, Carcinoma, Renal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRX34Drug
micro RNA therapy
| Study group | Role | What they receive |
|---|---|---|
| MRX34 | Experimental | Single agent MRX34 Receives: Drug: MRX34 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The maximum tolerated dose (MTD) for MRX34 and the recommended phase 2 dose (RPh2D)
Dose-limiting toxicity (DLT) in 3-6 patients at the end of one treatment cycle
Measured: 18 months
Secondary outcomes (2)
Peak blood concentration and Area Under the Curve (AUC) of MRX34 after IV dosing
Measured: 18 months
Number of patients with evidence of clinical activity of MRX34
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2013 (estimated)
- Study startApr 2013
- Primary completionMar 2017 (estimated)
- Study completionMay 2017 (estimated)
Why it stopped: Five immune related serious adverse events
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Liver CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 155 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirna Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cancer Prevention Research Institute of Texas
- Responsible party
- Sponsor
- Investigators
- O'Neill VIncent, MD, Study director, Mirna Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01829971
- Sponsor's protocol ID
- MRX34-101
- Status last verified
- Sep 2016
- First submitted
- 8 Apr 2013
- Last updated
- 27 Sep 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Lucibello G, Mograbi B, Milano G, Hofman P, Brest P. PD-L1 regulation revisited: impact on immunotherapeutic strategies. Trends Mol Med. 2021 Sep;27(9):868-881. doi: 10.1016/j.molmed.2021.06.005. Epub 2021 Jun 26.
PubMed 34187739Hong DS, Kang YK, Borad M, Sachdev J, Ejadi S, Lim HY, Brenner AJ, Park K, Lee JL, Kim TY, Shin S, Becerra CR, Falchook G, Stoudemire J, Martin D, Kelnar K, Peltier H, Bonato V, Bader AG, Smith S, Kim S, O'Neill V, Beg MS. Phase 1 study of MRX34, a liposomal miR-34a mimic, in patients with advanced solid tumours. Br J Cancer. 2020 May;122(11):1630-1637. doi: 10.1038/s41416-020-0802-1. Epub 2020 Apr 2.
PubMed 32238921
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection
- ClinicalTrials.gov ID
- NCT01829971
- Acronym / sponsor ID
- MRX34-101
- Conditions
- Primary Liver Cancer, SCLC, Lymphoma, Melanoma, Multiple Myeloma, Renal Cell Carcinoma
- Keywords
- microRNA, Advanced cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Five immune related serious adverse events
- Enrolment
- 155 participants (actual)
- Lead sponsor
- Mirna Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cancer Prevention Research Institute of Texas
- Study start
- Apr 2013
- Primary completion
- Mar 2017 (estimated)
- Study completion
- May 2017 (estimated)
- First posted
- 11 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MRX34 | Experimental | Drug: MRX34Single agent MRX34 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Aged ≥ 18 years
- Patients with histologically confirmed viral related hepatocellular, SCLC, non-cutaneous/ non-uveal melanoma, ovarian, TNBC, Sarcoma, Bladder and RCC.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
- Acceptable liver function:
- Acceptable renal function:
Key exclusion criteria (10 listed)
- Myocardial infarction within the past 6 months, unstable and/or symptomatic arrhythmia, or evidence of ischemia on ECG.
- Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy.
- Pregnant or nursing women.
- Known infection with human immunodeficiency virus (HIV).
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRX34micro RNA therapy | Drug | MRX34 |
Primary outcome
The maximum tolerated dose (MTD) for MRX34 and the recommended phase 2 dose (RPh2D)
Time frame: 18 months
Dose-limiting toxicity (DLT) in 3-6 patients at the end of one treatment cycle
2 secondary outcomes
Peak blood concentration and Area Under the Curve (AUC) of MRX34 after IV dosing
Time frame: 18 months
Number of patients with evidence of clinical activity of MRX34
Time frame: 18 months
Results and safety
The study was stopped early (reason given: Five immune related serious adverse events). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2016.
- Derived from this study: PubMed 34187739Lucibello G, Mograbi B, Milano G, Hofman P, Brest P. PD-L1 regulation revisited: impact on immunotherapeutic strategies. Trends Mol Med. 2021 Sep;27(9):868-881. doi: 10.1016/j.molmed.2021.06.005. Epub 2021 Jun 26.
- Derived from this study: PubMed 32238921Hong DS, Kang YK, Borad M, Sachdev J, Ejadi S, Lim HY, Brenner AJ, Park K, Lee JL, Kim TY, Shin S, Becerra CR, Falchook G, Stoudemire J, Martin D, Kelnar K, Peltier H, Bonato V, Bader AG, Smith S, Kim S, O'Neill V, Beg MS. Phase 1 study of MRX34, a liposomal miR-34a mimic, in patients with advanced solid tumours. Br J Cancer. 2020 May;122(11):1630-1637. doi: 10.1038/s41416-020-0802-1. Epub 2020 Apr 2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
O'Neill VIncent, MD study director
Mirna Therapeutics
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MRX34 in people with Primary Liver Cancer and SCLC, run by Mirna Therapeutics, Inc. at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 27 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Five immune related serious adverse events). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The maximum tolerated dose (MTD) for MRX34 and the recommended phase 2 dose (RPh2D) (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01829971, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Sep 2016.