New drug targets Cancer's fuel supply in rare genetic tumors

NCT ID NCT03872427

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 31, 2026 · Updated 2 times

Summary

This phase II trial tests a drug called telaglenastat (CB-839) in people with advanced solid tumors that have specific genetic changes, including NF1 mutations and malignant peripheral nerve sheath tumors. The drug works by blocking an enzyme that cancer cells need to grow. Researchers will measure how well the drug shrinks or stabilizes tumors over six months, and monitor for side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: telaglenastat hydrochloride (CB-839 HCl)
What this could lead to: If it works, this could point toward a new treatment option for people with NF1-related cancers and other solid tumors with specific genetic mutations.
What could go wrong: This is an early-phase trial with only 54 participants, so results may not apply to everyone. The drug may cause side effects or fail to shrink tumors.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

54 people

The number who actually took part.

Started

Dec 2019

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients must have histologically confirmed malignancy that is metastatic or unresectable * Patient must have histopathologic confirmation of advanced solid tumor with NF1 mutation, NF1 mutant MPNST, KEAP1/NRF2 mutant and STK11/LKB1 mutant tumors (molecular profiling performed in any Clinical Laboratory Improvement Act \[CLIA\] certified lab \[including tumor and circulating cell-free (cf)DNA\], e.g. Caris, FoundationOne, FoundationAct, Oncomine, Guardant etc.) * NOTE: For all cohorts annotation for actionability will be performed by the PRECISION ONCOLOGY DECISION SUPPORT (PODS) TEAM SHEIKH KHALIFA BIN ZAYED AL NAHYAN INSTITUTE FOR PERSONALIZED CANCER THERAPY (IPCT) THE UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER 6565 MD ANDERSON BLVD, HOUSTON, TX 77030 * Patient must have no standard therapies available * Patient must be aged greater than 18 years old for all cohorts * Patients for NF1 mutant MPNST and NF1 mutant non-MPNST cohorts must be \>= 40 kg * Patient must be at least 4 weeks since any prior surgery or radiotherapy * Females of childbearing potential must have a negative serum pregnancy test (=\< 14 days) prior to start of trial treatment * Response Evaluation Criteria in Solid Tumors (RECIST) measurable disease and biopsiable targetable lesion * Patients must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) as \>= 20 mm (\>= 2 cm) by chest x-ray or as \>= 10 mm (\>= 1 cm) with CT scan, magnetic resonance imaging (MRI), or calipers by clinical exam * Patients with treated brain metastases are eligible if there is no evidence of progression for at least 4 weeks after central nervous system (CNS)-directed treatment, as ascertained by clinical examination and brain imaging (MRI or CT) during the screening period * Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%) * Leukocytes \>= 3,000/mcL * Absolute neutrophil count \>= 1,000/mcL * Platelets \>= 100,000/mcL * Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN) and up to 3 ml/dL for patients with Gilbert's disease * Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3 x institutional ULN and =\< 5 x institutional ULN for patients with liver metastases * Creatinine =\< institutional ULN, as age appropriate OR * Glomerular filtration rate (GFR) \>= 30 mL/min/1.73 m\^2 for patients with creatinine levels above institutional normal * The effects of telaglenastat (CB-839) HCl on the developing human fetus are unknown. For this reason and because anti-metabolic agents like telaglenastat (CB-839) HCl 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 and for the duration of study participation. 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 telaglenastat (CB-839) HCl administration * Ability to understand and the willingness to sign a written informed consent document Exclusion Criteria: * Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study * Patients who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities \> grade 1) * Patients who are receiving any other investigational agents * History of allergic reactions attributed to compounds of similar chemical or biologic composition to telaglenastat (CB-839) HCl * Patients with glioma will be excluded * Patients with active or prior history of hepatitis B or C will be excluded * Telaglenastat (CB-839) HCl is a weak in vitro inhibitor of CYP2C9. Therefore, patients receiving any medications or substances that are substrates of CYP2C9 are eligible, but should use caution with substrates that have a narrow therapeutic index. 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 * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Pregnant women are excluded from this study because telaglenastat (CB-839) HCl is anti-metabolic 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 telaglenastat (CB-839) HCl, breastfeeding should be discontinued if the mother is treated with telaglenastat (CB-839) HCl

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Conditions

The condition(s) this trial relates to.

cancer malignant peripheral nerve sheath tumor metastatic malignant neoplasm Neoplasm Metastasis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Advanced malignant solid neoplasm Metastatic malignant solid neoplasm NF1 mutation positive malignant peripheral nerve sheath tumor Unresectable malignant solid neoplasm

Contacts and locations

Locations

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
HaysMed

Hays, Kansas, 67601, United States
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Mercy Hospital Pittsburg

Pittsburg, Kansas, 66762, United States
Moffitt Cancer Center

Tampa, Florida, 33612, United States
Moffitt Cancer Center - McKinley Campus

Tampa, Florida, 33612, United States
Moffitt Cancer Center-International Plaza

Tampa, Florida, 33607, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
National Cancer Institute Developmental Therapeutics Clinic

Bethesda, Maryland, 20892, United States
National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States
Northwestern University

Chicago, Illinois, 60611, United States
Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States
Salina Regional Health Center

Salina, Kansas, 67401, United States
The University of Kansas Cancer Center - Olathe

Olathe, Kansas, 66061, United States
UM Sylvester Comprehensive Cancer Center at Aventura

Aventura, Florida, 33180, United States
UM Sylvester Comprehensive Cancer Center at Coral Gables

Coral Gables, Florida, 33146, United States
UM Sylvester Comprehensive Cancer Center at Deerfield Beach

Deerfield Beach, Florida, 33442, United States
UM Sylvester Comprehensive Cancer Center at Kendall

Miami, Florida, 33176, United States
UM Sylvester Comprehensive Cancer Center at Plantation

Plantation, Florida, 33324, United States
UPMC Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States
UT MD Anderson Cancer Center

Houston, Texas, 77030, United States
University Health Truman Medical Center

Kansas City, Missouri, 64108, United States
University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States
University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States
University of Kansas Clinical Research Center

Fairway, Kansas, 66205, United States
University of Kansas Health System Saint Francis Campus

Topeka, Kansas, 66606, United States
University of Kansas Hospital-Westwood Cancer Center

Westwood, Kansas, 66205, United States
University of Kentucky/Markey Cancer Center

Lexington, Kentucky, 40536, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States
Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States
Wake Forest University at Clemmons

Clemmons, North Carolina, 27012, United States

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