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SOLAR: Efficacy and Safety of Cobomarsen (MRG-106) vs. Active Comparator in Subjects With Mycosis Fungoides

Phase 2
Terminated
Conditions
Cutaneous T-Cell Lymphoma/Mycosis Fungoides
Interventions
Registration Number
NCT03713320
Lead Sponsor
miRagen Therapeutics, Inc.
Brief Summary

The main objective of this clinical trial is to study the efficacy and safety of cobomarsen (also known as MRG-106) for the treatment of cutaneous T-cell lymphoma (CTCL), mycosis fungoides (MF) subtype. Cobomarsen is designed to inhibit the activity of a molecule called miR-155 that may be important to the growth and survival of MF cancer cells. The study will compare the effects of cobomarsen to vorinostat, a drug that has been approved for the treatment of CTCL in the United States and several other countries.

Participants in the clinical trial will be randomly assigned to receive either weekly doses of cobomarsen by injection into a vein or daily oral doses of vorinostat. Participants will continue on their assigned treatment as long as there is no evidence of progression of their cancer. The effects of treatment will be measured based on changes in skin lesion severity, as well as the length of time that the subject's disease remains stable or improved, without evidence of disease progression. The safety and tolerability of cobomarsen will be assessed based on the frequency and severity of observed side effects.

Participants assigned to receive vorinostat who experience progression of their disease during their participation in this study may have the option to be treated with cobomarsen in an open-label, crossover arm of the same study if they meet the entry criteria for that part of the study.

Detailed Description

Study Design:

Subjects will be randomly assigned in a 1:1 ratio to receive either cobomarsen or vorinostat. Approximately 126 subjects (63 per arm) are expected to be enrolled. Cobomarsen will be administered in the clinic by 2-hr intravenous infusion on Days 1, 3, 5 and 8, and weekly thereafter. Vorinostat will be dispensed to study subjects and taken as a daily oral dose according to the manufacturer's labeled dosing instructions. Treatment will continue until the subject becomes intolerant, develops clinically significant side effects, progresses, or the trial is terminated. An interim analysis will be conducted after approximately 40 subjects have been followed for a minimum of approximately 6 months. Enrollment will be suspended until the completion of the interim analysis.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
37
Inclusion Criteria
  • Biopsy-proven CTCL, MF subtype
  • Clinical stage IB, II, or III, with staging based on screening assessments
  • Minimum mSWAT score of 10 at screening
  • Receipt of at least one prior therapy for CTCL

Key

Exclusion Criteria
  • Previous enrollment in a cobomarsen study
  • Prior therapy with vorinostat or other HDAC inhibitors, or contraindication to an HDAC inhibitor
  • Sézary syndrome or mycosis fungoides with B2 involvement, defined as documented history of B2 and/or B2 staging at screening
  • Evidence of large cell transformation
  • Lymph node involvement at screening, unless radiologically or histologically confirmed to be nonmalignant
  • Visceral involvement related to MF at screening

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
CobomarsenCobomarsenCobomarsen will be administered by intravenous 2-hour infusion at a dose of 282 mg on Days 1, 3, 5, 8, and weekly thereafter
VorinostatVorinostatVorinostat will be administered orally at a dose of 400 mg (four 100-mg capsules) once daily with food, at approximately the same time each day.
Primary Outcome Measures
NameTimeMethod
Percentage of Subjects Achieving an Objective Skin Response of at Least 4 Months Duration (ORR4)Date of first dose through the earlier of last study visit or interim analysis data cut-off date of 12-Oct-2020, up to 16 months

ORR4 is the percentage of subjects with a complete response (CR) or partial response (PR) in the skin for 4 consecutive months confirmed by repeat assessments no less than 28 days (± 3 days) later. The modified Severity Weighted Assessment Tool (mSWAT) is used to measure skin disease severity based on the percentage of body surface area (BSA) with patches, plaques, or tumors. Total scores are calculated by multiplying the BSA percentage for each category of lesion (patch, plaque, or tumor) by a weighting factor and adding the three sub-scores. Lower scores indicate a lower degree of skin disease severity. CR corresponds to 100% clearance of skin lesions present at baseline (mSWAT score of 0). PR corresponds to 50-99% clearance of skin disease present at baseline (at least 50% reduction in mSWAT score), without new tumors.

Secondary Outcome Measures
NameTimeMethod
Time to Maximal Effect in mSWATMonthly from first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Time to greatest improvement in mSWAT score

Objective Response Rate in the Skin of at Least 28-days Duration (ORR1)Monthly from first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Percentage of participants achieving ≥ 50% improvement in mSWAT of at least 28-days duration

Time to ProgressionDate of first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Time from date of randomization until the earliest date of confirmed progression

Time to ≥ 50% Improvement in mSWATMonthly from first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Time from date of randomization until ≥ 50% improvement in mSWAT score

Pruritus Medication UtilizationDate of first dose through end of treatment or interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Change from baseline in number of pruritus medications taken per subject

Progression-free Survival (PFS)Date of first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Time from date of randomization until the date of earliest documented progression or death from any cause. The duration of PFS was censored at the date of the last mSWAT assessment if the subject was alive and had no documented progression. Disease progression in the skin is defined as ≥ 25% increase in mSWAT score from baseline or, in participants with complete or partial response, increase in mSWAT score of greater than the sum of the nadir plus 50% baseline score.

Complete Response RateDate of first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Percentage of subjects with a complete response in the skin based on mSWAT

Percentage of Subjects Achieving ≥ 50% Improvement in mSWAT at 28 Days28 days after first dose

Percentage of subjects achieving ≥ 50% improvement from baseline in mSWAT at 28 days after first dose

Percentage of Subjects Achieving ≥ 50% Improvement in mSWAT at 4 Months4 months after first dose

Percentage of subjects achieving ≥ 50% improvement from baseline in mSWAT at 4 months after first dose

Duration of Response in SkinMonthly from first dose through interim analysis data cut-off date of 12-Oct-2020, up to 16 months

Duration of response in skin (no progression after achieving ≥ 50% improvement in mSWAT)

Trial Locations

Locations (41)

The Ohio State University Comprehensive Cancer Center

🇺🇸

Columbus, Ohio, United States

Inova Schar Cancer Institute

🇺🇸

Fairfax, Virginia, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

University of Washington

🇺🇸

Seattle, Washington, United States

Hôpital Robert Dubré, CHU de Reims

🇫🇷

Reims, France

University Clinic UZ Leuven

🇧🇪

Leuven, Belgium

City of Hope

🇺🇸

Duarte, California, United States

Hospital Universitario 12 de Octubre

🇪🇸

Madrid, Spain

Hospital Universitario de Salamanca

🇪🇸

Salamanca, Spain

UCLA

🇺🇸

Los Angeles, California, United States

Chao Family Comprehensive Cancer Center at University of California, Irvine

🇺🇸

Orange, California, United States

Dartmouth Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Rochester Skin Lymphoma Medical Group

🇺🇸

Fairport, New York, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

Linear Clinical Research

🇦🇺

Nedlands, Australia

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

Westmead Hospital

🇦🇺

Westmead, New South Wales, Australia

Cross Cancer Institute

🇨🇦

Edmonton, Alberta, Canada

Hôpital Saint-Louis

🇫🇷

Paris, France

Jewish General Hospital

🇨🇦

Montréal, Quebec, Canada

Centre Hospitalier Lyon-Sud

🇫🇷

Pierre-Bénite, France

Centre Hospitalier Universitaire de Rouen

🇫🇷

Rouen, France

Policlinico S. Orsola-Malpighi

🇮🇹

Bologna, Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

🇮🇹

Milano, Italy

AOU Citta dell Salute e della Scienza di Torino

🇮🇹

Torino, Italy

Vall d'Hebron Institute of Oncology

🇪🇸

Barcelona, Spain

Fundación Jiménez Diaz

🇪🇸

Madrid, Spain

Consorcio Hospital General Universitario Valencia

🇪🇸

Valencia, Spain

University Hospitals of Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

🇬🇧

Birmingham, United Kingdom

Beatson West of Scotland Cancer Centre

🇬🇧

Glasgow, United Kingdom

Guy's and St. Thomas' NHS Foundation Trust, Cancer Center

🇬🇧

London, United Kingdom

The Christie NHS Foundation Trust

🇬🇧

Manchester, United Kingdom

Hôpital Saint André, CHU de Bordeaux

🇫🇷

Bordeaux, France

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

The University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Smilow Cancer Hospital at Yale-New Haven

🇺🇸

New Haven, Connecticut, United States

H. Lee Moffitt Cancer Center and Research Institute

🇺🇸

Tampa, Florida, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

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