A Phase 1 interventional study of Cobomarsen in Cutaneous T-cell Lymphoma (CTCL), Mycosis Fungoides (MF) and Chronic Lymphocytic Leukemia (CLL), sponsored by miRagen Therapeutics, Inc.. Completed at 19 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-11-23.
Sponsored by miRagen Therapeutics, Inc. · Phase 1, Interventional, and Treatment
Objectives of this clinical trial are to evaluate the safety, tolerability, pharmacokinetics and potential efficacy of the investigational drug, cobomarsen (MRG-106), in patients diagnosed with certain lymphomas and leukemias, including cutaneous T-cell lymphoma (CTCL) [mycosis fungoides (MF) subtype], chronic lymphocytic leukemia (CLL), diffuse large B-cell lymphoma (DLBCL) [activated B-cell (ABC) subtype], and adult T-cell leukemia/lymphoma (ATLL). Cobomarsen is an inhibitor of a molecule called miR-155 that is found at high levels in these types of cancers and may be important in promoting the growth and survival of the cancer cells. Participants in the clinical trial will receive weekly doses of cobomarsen administered by injection under the skin or into a vein, or by injection directly into cancerous lesions in the skin (for CTCL only). Blood samples will be collected to measure how cobomarsen is processed by the body, and other measurements will be performed to study how normal and cancerous cells of the immune system respond when exposed to cobomarsen.
Study Design:
Exclusion Criteria:
Intratumoral Injection of cobomarsen
Drug: Cobomarsen
Subcutaneous, intravenous or a combination of systemic and intratumoral administration of cobomarsen with or without stable background therapy
Drug: Cobomarsen
Subcutaneous or intravenous administration of cobomarsen as monotherapy
Drug: Cobomarsen
Subcutaneous or intravenous administration of cobomarsen as monotherapy
Drug: Cobomarsen
Subcutaneous or intravenous administration of cobomarsen as monotherapy
Drug: Cobomarsen
Subcutaneous or intravenous administration of cobomarsen as monotherapy
Drug: Cobomarsen
Also known as: MRG-106
Safety and tolerability of cobomarsen based on vital signs, physical examination, clinical laboratory tests, ECG, and incidence and severity of adverse events
Time frame: From start of treatment to end of study participation
Area under the plasma concentration vs. time curve (AUC) of cobomarsen following single and repeat doses administered intratumorally, subcutaneously or intravenously
Time frame: Up to 56 days
Peak plasma concentration (Cmax) of cobomarsen following single and repeat doses administered intratumorally, subcutaneously or intravenously
Time frame: Up to 56 days
Trough plasma concentration (Ctrough) of cobomarsen following each 4-week cycle of dosing
Time frame: Monthly from Week 5 up to end of study participation
Skin disease severity (index lesions) - MF only
Changes in MF skin lesion severity before and after treatment based on the Composite Assessment of Index Lesion Severity (CAILS) score
Time frame: Every 2 weeks from start of treatment until end of study participation
Skin disease severity (whole body) - MF only
Changes in MF skin lesion severity before and after treatment based on the modified Severity Weighted Assessment Tool (mSWAT) score
Time frame: Every 2 weeks from start of treatment until end of study participation
Overall Response Rate in the skin - MF
Proportion of subjects who achieve a partial response (PR) or complete response (CR) in the skin, based on SWAT score
Time frame: Approximately 1 year
Overall Response Rate - CLL
Proportion of subjects who achieve a PR or CR as defined by IWCLL criteria (Hallek et al., 2008) based on CT scans, bone marrow biopsies, and flow cytometry
Time frame: Approximately 1 year
Minimal Residual Disease (MRD) - CLL only
Proportion of subjects who achieve a CR with no evidence of MRD by flow cytometry
Time frame: Approximately 1 year
Overall Response Rate - DLBCL
Proportion of subjects who achieve a PR or CR as defined by the Lugano classification (Cheson et al., 2014) based on positron emission tomography-computed tomography (PET-CT) scans and bone marrow biopsy to confirm CR
Time frame: Approximately 1 year
Overall Response Rate - ATLL
Proportion of subjects who achieve a PR or CR as defined by international consensus criteria (Tsukasaki et al., 2009) based on CT scans and flow cytometry, and bone marrow biopsy to confirm CR
Time frame: Approximately 1 year
Duration of Response
Number of days from initial date of confirmed PR or CR until loss of response or relapse
Time frame: Up to approximately 2 years
Time to Progression
Number of days from first dose until objective disease progression
Time frame: Up to approximately 2 years
Progression Free Survival (PFS)
Number of days from first dose until objective disease progression or death from any cause
Time frame: Up to approximately 2 years
Overall Survival (OS)
Number of days from first dose until death from any cause
Time frame: Up to approximately 2 years
miR-155-5p expression in cutaneous lesions of subjects with MF
Exploratory assessment based on quantitative real time polymerase chain reaction (qRT-PCR) analysis of total RNA isolated from skin biopsies
Time frame: At baseline and between Week 16 and end of study participation
Proportion of neoplastic lymphoid cells in cutaneous lesions of subjects with MF
Exploratory histological assessment before and after treatment with cobomarsen
Time frame: At baseline and between Week 16 and end of study participation
Proportions of immune cell subsets
Exploratory assessment before and after treatment with cobomarsen by flow cytometry on whole blood
Time frame: At baseline and monthly or bimonthly, up to end of study participation
Dermatology-specific quality of life - MF only
Changes in skin-related quality of life based on the Skindex-29 assessment tool
Time frame: At baseline and monthly, up to approximately 2 years
Pruritus - MF only
Changes in intensity of skin itch based on the Pruritus Numerical Rating Scale
Time frame: At baseline and monthly, up to approximately 2 years
Plan to share: No
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miRagen Therapeutics, Inc.