AzTrials
AzTrials

Solid Tumor, AML AdultPhase 1/2NCT04372706

RTX-240 Monotherapy and in Combination With Pembrolizumab

A Phase 1/2 study testing RTX-240 and Pembrolizumab in people with Solid Tumor, AML Adult, run by Rubius Therapeutics at 11 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

Open label, multicenter, multidose, first-in-human Phase 1/2 study of RTX-240 monotherapy or in combination of pembrolizumab for the treatment of patients with (1) relapsed/refractory R/R or locally advanced solid tumors (Phase 1/2) or (2) R/R Acute Myeloid Leukemia (AML) (Phase 1 only). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Solid Tumor, AML Adult
What is being tested?
RTX-240 and Pembrolizumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
69 participants
Where
11 sitesUnited States
Study dates
6 May 2020to 30 Nov 2022
Phase
Phase 1/2
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

  • Signed written informed consent obtained prior to study procedures
  • Patients ≥18 years with an ECOG 0 or 1 (Parts 1, 2 and 4) or 0-2 (Part 3).
  • Relapsed/Refractory (R/R) or locally advanced, unresectable solid tumor for which no standard therapy exists (Parts 1, 2 and 4), or for which the patient is ineligible or has declined standard therapy or R/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 and Blood Cell Counts (Parts 1, 2, and 4) as defined by the protocol:
Show all 16Show fewer
  • 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 ≥ 1 × 10^3/μL without myeloid growth factor support for at least one week prior to enrollment
  • Platelet count ≥ 75 × 10^3/μL
  • Hemoglobin should be ≥ 9 g/dL without red blood cell transfusion for at least one week
  • Patients must have LVEF ≥ 45%
  • Patients enrolling into Part 2 of the study must be diagnosed with NSCLC, RCC, or anal cancers
  • Patients enrolling into Part 4 must be diagnosed with NSCLC or RCC
  • Patients enrolling into either Part 2 or 4 must have 2 or fewer prior treatment regimens. If patient received a prior PD-1/PD-L1-containing regimen, a prior response is required.

Who cannot join

  • Primary central nervous system (CNS) malignancy or CNS involvement, unless asymptomatic, previously treated, and stable without steroids (Parts 1, 2 and 4) 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
  • Class III or IV cardiomyopathy per the New York Heart Association criteria
Show all 12Show fewer
  • Leukemic blast count ≥ 25 x 10^3/µL (Part 3)
  • Concomitant conditions requiring active immunosuppression
  • History of clinically significant Grade 3 or higher immune related Adverse Event (irAE)
  • Prior malignancy within the past 3 years, with protocol specified exceptions
  • History of severe hypersensitivity to a PD-1/PD-L1 blocking Ab unless previously rechallenged successfully (Part 4)
  • Current noninfectious pneumonitis or a history of radiation pneumonitis or pneumonitis that required steroids, or Grade 2 or greater immune related pneumonitis, hepatitis, hypophysitis, or other endocrinopathy (Part 4)

Trial locations and contacts

11 sites in 1 country.

11 sites in 1 country

United States11 sites

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Sarah Cannon Research Institute/ Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • Virginia Cancer Specialists

    Fairfax, Virginia, 22031

  • University of California San Diego

    La Jolla, California, 92093

  • The Angeles Clinic & Research Institute

    Los Angeles, California, 90025

  • Sylvester Comprehensive Cancer Center/UMHC

    Miami, Florida, 33136

  • Sarah Cannon Research Institute

    Nashville, Tennessee, 37203

  • Columbia University Medical Center

    New York, New York, 10032

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Sciences University - Knight Cancer Institute

    Portland, Oregon, 97239

Other trials in United States

About this study

Official title: Phase 1/2 Study of RTX-240 Monotherapy and in Combination With Pembrolizumab

Open label, multicenter, multidose, first-in-human Phase 1/2 study of RTX-240 monotherapy or in combination of pembrolizumab for the treatment of patients with (1) relapsed/refractory R/R or locally advanced solid tumors (Phase 1/2) or (2) R/R Acute Myeloid Leukemia (AML) (Phase 1 only).

Read the detailed description

This is a Phase 1/2, open label, multicenter, multidose, first-in-human (FIH) dose escalation and expansion study 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 (R/R) or locally advanced solid tumors (Phase 1/2) or R/R acute myeloid leukemia (Phase 1 only), and RTX-240 in combination with pembrolizumab in adult patients with R/R or locally advanced solid tumors (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 stimulating the innate and adaptive immune systems for the treatment of cancer. The study includes a monotherapy dose escalation phase (Phase 1) followed by an expansion phase (Phase 2) in specified tumor types.

Being tested
RTX-240, Pembrolizumab

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RTX-240Drug

    Engineered red cells co-expressing 4-1BBL and IL-15TP

  • PembrolizumabDrug

    Humanized immunoglobulin G4 programmed death receptor-1 blocking antibody

Study groups in NCT04372706
Study groupRoleWhat they receive
Part 1: RTX-240 Dose EscalationExperimentalPhase 1: RTX-240 monotherapy dose escalation in Solid Tumors
Receives: Drug: RTX-240
Part 2: RTX-240 Solid Tumor ExpansionExperimentalPhase 2: RTX-240 monotherapy dose expansion in Non-small Cell Lung Cancer (NSCLC), Renal Cell Carcinoma (RCC), and anal cancers
Receives: Drug: RTX-240
Part 3: RTX-240 Dose EscalationExperimentalPhase 1: RTX-240 monotherapy dose escalation in AML
Receives: Drug: RTX-240
Part 4: RTX-240 Plus Pembrolizumab Dose EscalationExperimentalPhase 1: RTX-240 dose escalation in combination with Pembrolizumab in Solid Tumors
Receives: Drug: RTX-240; Drug: Pembrolizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety Assessment: Measured by incidence of Treatment Emergent Adverse Events (TEAEs)

    Measured: Up to 38 months

  2. Dose limiting toxicities (DLTs) of study treatment as determined by incidence and severity of adverse events (AEs)

    Measured: Up to 38 months

Secondary outcomes (10)

  1. PK of study treatment as measured by detection of the number of cells positive for both 4-1BBL and IL-15 using flow cytometry.

    Measured: Assessed From the 1st dose of RTX-240 until 30 days after last of study treatment

  2. Determination of the Immunogenicity of study treatment Measured by the incidence of antibodies to RTX-240

    Measured: Assessed From the 1st dose of RTX-240 until 30 days after last of study treatment

  3. Anti-tumor activity of study treatment measured by clinical benefit rate (CBR) (% of patients who achieve CR, PR or stable disease [SD])

    Measured: Up to 38 months

  4. Anti-tumor activity of study treatment measured by duration of response (DoR)

    Measured: Up to 38 months

  5. Anti-tumor activity of study treatment measured by progression free survival (PFS)

    Measured: Up to 38 months

  6. Anti-tumor activity of study treatment measured by overall survival (OS)

    Measured: Up to 38 months

  7. Anti-tumor activity of study treatment measured by time to response (TTR).

    Measured: Up to 38 months

  8. Anti-tumor activity of study treatment measured by time to progression (TTP)

    Measured: Up to 38 months

  9. Anti-Tumor activity of study treatment Measured by Objective Response Rate (ORR)

    Measured: Up to 38 months

  10. Proportion of AML patients with CR, CR with incomplete recovery (CRi), morphologic leukemia-free state, or PR

    Measured: Up to 38 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 May 2020
  2. Study start6 May 2020
  3. Primary completion30 Nov 2022
  4. Study completion30 Nov 2022

Why it stopped: Sponsor terminated study during expansion on 11/30/22. RTX-240 was well-tolerated in multiple indications, combinations, and dose levels (69pts). No DLTs, related deaths or SAEs were reported. RTX-240 cleared circulation rapidly.

Resources

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
69 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04372706
Sponsor's protocol ID
RTX-240-01
Status last verified
Dec 2022
First submitted
23 Apr 2020
Last updated
13 Dec 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing RTX-240 and Pembrolizumab in people with Solid Tumor, AML Adult, run by Rubius Therapeutics at 11 sites in United States.

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 16 inclusion and 12 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 13 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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: Sponsor terminated study during expansion on 11/30/22. RTX-240 was well-tolerated in multiple indications, combinations, and dose levels (69pts). No DLTs, related deaths or SAEs were reported. RTX-240 cleared circulation rapidly.). 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?

Safety Assessment: Measured by incidence of Treatment Emergent Adverse Events (TEAEs) (time frame: Up to 38 months). The study lists 2 primary outcomes in all. 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 NCT04372706.

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 13 Dec 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in