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RTX-240 Monotherapy

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ClinicalTrials.gov Identifier: NCT04372706
Recruitment Status : Recruiting
First Posted : May 4, 2020
Last Update Posted : May 4, 2021
Sponsor:
Information provided by (Responsible Party):
Rubius Therapeutics

Brief Summary:
Open label, multicenter, multidose, first-in-human Phase 1/2 study of RTX-240 for the treatment of patients with (1) relapsed/refractoryR/R or locally advanced solid tumors (Phase 1/2) or (2) R/R AML (Phase 1 only).

Condition or disease Intervention/treatment Phase
Solid Tumor, AML Adult Drug: RTX-240 Phase 1 Phase 2

Detailed Description:
This is a Phase 1/2, open label, multicenter, multidose, first-in-human (FIH) dose escalation and expansion to determine the safety and tolerability, recommended phase 2 dose and optimal dosing interval, pharmacology, and antitumor activity of RTX-240 in adult patients with relapsed/refractory or locally advanced solid tumors (Phase 1/2) or relapsed/refractory acute myeloid leukemia (Phase 1 only). RTX-240 is a cellular therapy that co-expresses 4-1BBL and IL-15TP, a fusion of IL-15 and IL-15 receptor alpha, with the goal of harnessing the innate and adaptive immune systems for the treatment of cancer. The study will include a monotherapy dose escalation phase followed by an expansion phase in specified tumor types.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 216 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1/2 Study of RTX-240 Monotherapy
Actual Study Start Date : May 6, 2020
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : June 2023

Arm Intervention/treatment
Experimental: Part 1: RTX-240 Dose Escalation
Phase 1: RTX-240 monotherapy dose escalation in Solid Tumors.
Drug: RTX-240
Engineered red cells co-expressing 4-1BBL and IL-15TP

Experimental: Part 2: RTX-240 Solid Tumor Expansion
Phase 2: RTX-240 administered intravenously on Day 1 of each cycle.
Drug: RTX-240
Engineered red cells co-expressing 4-1BBL and IL-15TP

Experimental: Part 3: RTX-240 Dose Escalation
Phase 1: RTX-240 monotherapy dose escalation in AML
Drug: RTX-240
Engineered red cells co-expressing 4-1BBL and IL-15TP




Primary Outcome Measures :
  1. Safety Assessment: Measured by incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 38 months ]
  2. Dose limiting toxicities (DLTs) of RTX-240 as determined by incidence and severity of adverse events (AEs) [ Time Frame: Up to 38 months ]
  3. Pharmacodynamics (PD) of RTX-240 will be evaluated through changes in NK cell number relative to baseline [ Time Frame: Up to 38 months ]
  4. Pharmacodynamics (PD) of RTX-240 will be evaluated through changes in T-cell number relative to baseline [ Time Frame: Up to 38 months ]

Secondary Outcome Measures :
  1. PK of RTX-240 as measured by detection of the number of RTX cells positive for both 4-1BBL and IL-15 using flow cytometry. [ Time Frame: Assessed From the 1st dose of RTX-240 until 30 days after last of study treatment ]
  2. Determination of the Immunogenicity of RTX-240 Measured by the incidence of antibodies to RTX-240 [ Time Frame: Assessed From the 1st dose of RTX-240 until 30 days after last of study treatment ]
  3. Anti-tumor activity of RTX-240 measured by clinical benefit rate (CBR) (% of patients who achieve CR, PR or SD) [ Time Frame: Up to 38 months ]
  4. Anti-tumor activity of RTX-240 measured by duration of response (DoR) [ Time Frame: Up to 38 months ]
  5. Anti-tumor activity of RTX-240 measured by progression free survival (PFS) [ Time Frame: Up to 38 months ]
  6. Anti-tumor activity of RTX-240 measured by overall survival (OS) [ Time Frame: Up to 38 months ]
  7. Anti-tumor activity of RTX-240 measured by time to response (TTR). [ Time Frame: Up to 38 months ]
  8. Anti-tumor activity of RTX-240 measured by time to progression (TTP) [ Time Frame: Up to 38 months ]
  9. Anti-Tumor activity of RTX-240 Measured by Overall Response Rate (ORR) in Dose Escalation [ Time Frame: Up to 38 months ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed written informed consent obtained prior to study procedures
  • Patients ≥18 years with an ECOG 0 or 1 (Parts 1 and 2) or 0-2 (Part 3).
  • Relapsed/Refractory (R/R) or locally advanced, unresectable solid tumor for which no standard therapy exists (Parts 1 and 2), or for which the patient is ineligible or has declined standard therapyR/R, cytologically confirmed AML (Part 3).
  • Disease must be measurable per Response Evaluation Criteria
  • The shorter of 28 days or 5 half-lives must have elapsed since the completion of prior therapy, before initiation of study treatment.
  • Adequate Organ Function as Defined by the protocol:

    • GFR ≥ 50 mL/min/1.73,
    • AST and ALT ≤ 3 × the ULN and total bilirubin ≤ 1.5 × ULN,
    • In the absence of cancer within the liver, or AST and ALT ≤ 5 × ULN and total bilirubin ≤ 3 × ULN, in the setting of primary or metastatic liver tumors.
    • ANC ≥ 10 × 103/μL and platelet count ≥ 100 × 103/μL without myeloid growth factor support or transfusion, respectively, for at least one week.
    • Hemoglobin should be ≥ 9 g/dL without red blood cell transfusion for at least two weeks.
    • Patients must have LVEF ≥ 45%
  • Patients enrolling into Part 2 of the study must be diagnosed with a solid tumor that has been selected for an expansion cohort

Exclusion Criteria:

  • Primary central nervous system (CNS) malignancy or CNS involvement, unless asymptomatic, previously treated, and stable without steroids (Parts 1 and 2) or known CNS leukemia (Part 3).
  • Known hypersensitivity to any component of study treatment or excipients.
  • Positive antibody screen using institution's standard type and screen test.
  • Clinically significant, active and uncontrolled infection, including human immunodeficiency virus (HIV), Hepatitis B virus (HBV), or Hepatitis C virus (HCV).
  • Clinically significant coagulopathy, uncontrolled hypertension or autoimmune hemolytic anemia
  • Leukemic blast count ≥25 x 103/µL (Part 3)
  • Concomitant conditions requiring active immunosuppression
  • Grade 3 immune related Adverse Event (irAE)
  • Prior malignancy within the past 3 years, with protocol specified exceptions

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04372706


Contacts
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Contact: Christina Coughlin, M.D., Ph.D. 617-679-9600 chris.coughlin@rubiustx.com

Locations
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United States, California
University of California San Diego Recruiting
La Jolla, California, United States, 92093
Contact: Sandip Patel, MD    585-822-6477    patel@ucsd.edu   
The Angeles Clinic & Research Institute Recruiting
Los Angeles, California, United States, 90025
Contact: Saba Mukarram    310-231-2181    SMukarram@theangelesclinic.org   
United States, Colorado
Sarah Cannon Research Institute/ Colorado Blood Cancer Institute Recruiting
Denver, Colorado, United States, 80218
Contact: Sarah Cannon Research Institute    844-482-4812    asksarah@sarahcannon.com   
United States, Florida
Sylvester Comprehensive Cancer Center/UMHC Recruiting
Miami, Florida, United States, 33136
Contact: Jaime Merchan, MD    305-243-1287    Merchan2@med.miami.edu   
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Ryan Sullivan, MD    617-724-5197    rsullivan7@mgh.harvard.edu   
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Research Nurse Navigator, RN    212-342-5162    cancerclinicaltrials@cumc.columbia.edu   
United States, Oregon
Oregon Health & Sciences University - Knight Cancer Institute Recruiting
Portland, Oregon, United States, 97239
Contact: Knight Cancer Institute Clinical Trials       trials@ohsu.edu   
United States, Pennsylvania
Thomas Jefferson University Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Usama Gergis, MD    215-955-4367    Usama.Gergis@jefferson.edu   
UPMC Hillman Cancer Center Recruiting
Pittsburgh, Pennsylvania, United States, 15232
Contact: Sarah Brodeur    412-623-2944    brodeurs@upmc.edu   
United States, Tennessee
Sarah Cannon Research Institute Recruiting
Nashville, Tennessee, United States, 37203
Contact: Sarah Cannon Research Site    615-329-7478    CANN.ResearchReferrals@scresearch.net   
United States, Virginia
Virginia Cancer Specialists Recruiting
Fairfax, Virginia, United States, 22031
Contact: Alexander Spira, MD, PhD,    703-280-5390    alexander.spira@usoncology.com   
Sponsors and Collaborators
Rubius Therapeutics
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Responsible Party: Rubius Therapeutics
ClinicalTrials.gov Identifier: NCT04372706    
Other Study ID Numbers: RTX-240-01
First Posted: May 4, 2020    Key Record Dates
Last Update Posted: May 4, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No