NCT04595747Active, not recruitingPhase 2
Testing the Anti-cancer Drug, Rogaratinib (BAY 1163877), for Treatment of Advanced Sarcoma With Alteration in Fibroblast Growth Factor Receptor (FGFR 1-4), and in Patients With SDH-deficient Gastrointestinal Stromal Tumor (GIST)
Phase 2 Study of Rogaratinib (BAY 1163877) in the Treatment of Patients With Sarcoma Harboring Alterations in Fibroblast Growth Factor Receptor (FGFR) 1-4 and SDH-Deficient Gastrointestinal Stromal Tumor (GIST)
Source: ClinicalTrials.gov · Last updated 31 Jul 2026
Brief summary (registry, in English)
This phase II trial studies the effect of rogaratinib in treating patients with sarcoma with a change in a group of proteins called fibroblast growth factor receptors (FGFRs) or SDH-deficient gastrointestinal stromal tumor (GIST). Rogaratinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 48 (estimated)
- First submitted
- 17 Oct 2020
- Start date
- 3 May 2021
- Primary completion date
- 31 Jul 2027
- Lead sponsor
- National Cancer Institute (NCI)
- Diseases
- Colorectal cancer, Sarcoma
- NCT number
- NCT04595747
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 48
- From 3 May 2021 to the primary completion date, 31 Jul 2027
- 18 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients who are receiving any other investigational agents
- Concomitant administration with sensitive substrates/narrow therapeutic index drugs of CYP3A4, P-gp BCRP, MATE1, and MATE2K, and strong inhibitors and inducers of CYP3A4 should be avoided. Use caution with strong inhibitors and inducers of P-glycoprotein (gp). Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
- Pregnant women are excluded from this study because rogaratinib (BAY 1163877) is kinase inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with rogaratinib (BAY 1163877), breastfeeding should be discontinued if the mother is treated with rogaratinib (BAY 1163877)
Other illness
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to rogaratinib (BAY 1163877)
- Patients with uncontrolled intercurrent illness
- Patients with psychiatric illness/social situations that would limit compliance with study requirements
Lab values
- Concomitant administration of medications that prolong QT/QTc interval is prohibited in accordance with the published FDA guidance "E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs".
- Patients with disturbed calcium and/or phosphate metabolism are excluded from this study
Who the study is looking for
- Participant must have histologically confirmed sarcoma with FGFR alteration identified by next-generation sequencing profiling with the exception of SDH-deficient GIST who can be enrolled regardless of FGFR status. Initial testing can be performed on archival tissue, if available. Patients must have locally advanced or metastatic disease that is not amenable to surgery
- Presence of measurable disease: Patient must have measurable disease
- Patients must have progressed following at least one standard prior chemotherapy regimen with the exception of SDH-deficient GIST for which there is no standard of care
- Participant must be willing to undergo pre-treatment biopsy if disease site is amenable to biopsy and low risk for the biopsy procedure. If biopsy is not possible, eligibility may be approved after discussion with the Study Chair. Of note, a minimum of 15 participants in each arm open to stage 2 should have disease amenable to biopsy. For those arms open in stage 1, all patients should have biopsiable disease.
- Age \>= 18 years
- Because no dosing or adverse event data are currently available on the use of rogaratinib (BAY 1163877) in patients \<18 years of age, children are excluded from this study
- Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%)
- Hemoglobin \>= 8.0 g/dL
- Absolute neutrophil count \>= 1,000/mcL
- Platelets \>= 100,000/mcL
- Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN)
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) =\< 3.0 x institutional ULN (unless liver metastases are present in which case it must be =\< 5 x ULN)
- Glomerular filtration rate (GFR) \>= 60 mL/min/1.73 m\^2 (using the Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] formula)
- Human immunodeficiency virus (HIV)-infected patients on effective non-CYP3A4 interacting anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
- For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
- Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
- Patients with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression
- Patients must be disease-free of prior invasive malignancies for \> 5 years with the exception of curatively-treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix
- NOTE: If there is a history of prior malignancy, patients must not be receiving other specific treatment for that cancer
- Patients should have completed prior treatment for their cancer: chemotherapy or radiotherapy must have been completed for greater than 2 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study
- Patients should have recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities \> grade 1) with the exception of alopecia
- Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association functional classification. To be eligible for this trial, patients should be class 2B or better
- Patients must have a QTc interval length of below 450 millisecond (msec)
- Participant is willing to comply with the protocol for the duration of the study including undergoing treatment and scheduled visits and examinations including follow up
- Participant must be able to swallow and maintain pills
- Women of childbearing potential must have a negative urine or serum pregnancy test within 28 days of initial dose of rogaratinib (BAY 1163877), and again within 7 days prior to treatment on day 1. If screening occurs within 7 days of day 1, only one pregnancy test is required
- The effects of rogaratinib (BAY 1163877) on the developing human fetus are unknown. For this reason and because kinase inhibitor agents are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and 4 months after completion of rogaratinib (BAY 1163877). Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of rogaratinib (BAY 1163877) administration
- Ability to understand and the willingness to sign a written informed consent document. Participants with impaired decision-making capacity (IDMC) who have a legally-authorized representative (LAR) and/or family member available will also be eligible
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (18)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Michigan Rogel Cancer Center Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| UCHealth University of Colorado Hospital Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| National Institutes of Health Clinical Center Coordinator of this site? Confirm its status | Bethesda | United States | Unknown status |
| National Cancer Institute Developmental Therapeutics Clinic Coordinator of this site? Confirm its status | Bethesda | United States | Unknown status |
| Dana-Farber Cancer Institute Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Northwestern University Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Ohio State University Comprehensive Cancer Center Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
| Siteman Cancer Center at West County Hospital Coordinator of this site? Confirm its status | Creve Coeur | United States | Unknown status |
| City of Hope Comprehensive Cancer Center Coordinator of this site? Confirm its status | Duarte | United States | Unknown status |
| UT MD Anderson Cancer Center Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| City of Hope at Irvine Lennar Coordinator of this site? Confirm its status | Irvine | United States | Unknown status |
| University of Miami Miller School of Medicine-Sylvester Cancer Center Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Vanderbilt University/Ingram Cancer Center Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Memorial Sloan-Kettering Cancer Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| UPMC Hillman Cancer Center Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| UM Sylvester Comprehensive Cancer Center at Plantation Coordinator of this site? Confirm its status | Plantation | United States | Unknown status |
| Siteman Cancer Center-South County Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
| Washington University School of Medicine Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
Related publications
This page is information, not medical advice. Discuss any trial with your doctor.