Clinical trial NCT06462183: Study of Safety and Efficacy of RGT-61159 in Adults with Relapsed/Refractory Adenoid Cystic Carcinoma (ACC) or Colorectal Carcinoma (CRC)
Official title: A Phase 1a/1b, First-in-human, Multicenter Study to Assess the Efficacy and Safety of RGT-61159 for Treatment of Patients with Relapsed/Refractory Adenoid Cystic Carcinoma (ACC) or Colorectal Carcinoma (CRC)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Adenoid Cystic Carcinoma
Colorectal Cancer
Lead sponsor: Rgenta Therapeutics Inc
Sponsor class: INDUSTRY
First posted: 2024-06-17
Enrollment: 105
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: Open-label, ascending dose escalation followed by dose expansion
Primary purpose: TREATMENT

Brief summary: Phase 1 study to evaluate safety, tolerability and anti-tumor activity of RGT-61159 in patients with ACC or CRC

Detailed description: This first-in-human, Phase 1, multi-center, open-label, non-randomized study, is designed to evaluate safety, tolerability, and anti-tumor activity of once-daily RGT-61159 in patients with advanced R/R ACC or R/R CRC for whom standard therapy with proven clinical benefit does not exist, is no longer effective, or is not appropriate. RGT-61159 is an oral, small molecule MYB inhibitor.

Eligibility criteria: Inclusion Criteria:

* Histologically confirmed ACC or CRC
* Radiographically measurable disease as assessed per RECIST 1.1, with at least 1 site of disease that is measurable and that has not been previously irradiated; or, if the patient has had previous radiation to the target lesion(s), there must be evidence of progression since the radiation
* Patients with locally relapsed/refractory (R/R) advanced or metastatic ACC not amenable to potentially curative surgery or radiotherapy and progression of disease within 12 months at study entry
* Patients with CRC must have locally R/R advanced or metastatic disease not amenable to potentially curative surgery or radiotherapy; must have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidines-, oxaliplatin-, and irinotecan-based chemotherapies, anti-VEGF agents, and if RAS wild-type, an anti-EGFR therapy.
* Adequate hematologic status, organ function, renal function, liver function and prothrombin time (PT) or INR ≤ 1.5 × ULN and partial thromboplastin time (PTT) or activated partial thromboplastin time (aPTT) ≤ 1.5 × ULN
* Resolved acute effects of any prior therapy to baseline

Exclusion Criteria:

* Major surgery or significant traumatic injury within 28 days prior to Cycle 1 Day 1
* Chemotherapy within 14 days prior to Cycle 1 Day 1
* Use of nitrosoureas or mitomycin C within 6 weeks prior to Cycle 1 Day 1
* Radiation therapy within 21 days prior to Cycle 1 Day 1
* Investigational drug use, targeted therapy, or biologic therapy within 28 days or 5 half-lives, whichever is shorter, prior to Cycle 1 Day 1
* Ongoing systemic infection requiring treatment with antibiotic, antiviral, or antifungal treatment
* Active known second malignancy
* Clinically significant cardiac disease
* Infection with human immunodeficiency virus (HIV)-1 or HIV-2 unless it's well-controlled HIV (eg, cluster of differentiation 4 \[CD4\] \> 350/mm3 and undetectable viral load)
* Current active liver disease including hepatitis A (hepatitis A \[HepA\] virus immunoglobulin M \[IgM\] positive), hepatitis B (hepatitis B virus \[HBV\] surface antigen positive), or hepatitis C (hepatitis C virus \[HCV\] antibody positive, confirmed by HCV RNA)
* Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant small bowel resection that would preclude adequate absorption
* Uncontrolled diabetes
* Treatment with a long-acting hematopoietic growth factor within 14 days before Cycle 1 Day 1 or a short-acting hematopoietic growth factor within 7 days before Cycle 1 Day 1
* Treatment with high-dose chemotherapy and stem-cell rescue (autologous stem cell transplant) or allogeneic stem cell transplant within 90 days before Cycle 1 Day 1
* Patients with central nervous system (CNS) metastases are not eligible, unless they have completed local therapy and have discontinued the use of corticosteroid throughout this indication for at least 4 weeks before starting treatment in this study
* History of solid organ transplantation
* Coronavirus disease 2019 (COVID-19) vaccination within 14 days prior to first dose of study drug
* Prior treatment with a MYB inhibitor

Primary outcomes:
measure: Number and type of dose-limiting toxicities (DLTs) in first cycle of administration
timeFrame: 21 days
measure: Number and type of adverse events
timeFrame: Through study completion, estimated as 30 days after last dose of study drug
measure: Recommended Phase 2 Dose (RP2D)
timeFrame: Assessed at the end of Cycle 1 for each subject (each cycle 21 days)
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: St Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: Laura & Isaac Perlmutter Cancer Center at NYU Langone Health
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Fairfax
country: United States
facility: Next Oncology VA
state: Virginia
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Center
state: Washington
city: Ottawa
country: Canada
facility: Ottawa Hospital Cancer Centre
state: Ontario
city: Toronto
country: Canada
facility: Princess Margaret Cancer Center
state: Ontario
Study officials:
References: