Neurofibromatosis 1 study: Cabozantinib Oral Tablet vs Selumetinib Oral Capsule
Official title Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
ClinicalTrials.gov ID: NCT06502171
What this study is testing
What is Cabozantinib Oral Tablet?
An investigational once‑daily tablet being studied for treating neurofibromatosis 1 (NF1) related tumors. is designed to inhibit multiple tyrosine kinases involved in tumor growth and angiogenesis.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether the combination of selumetinib and cabozantinib is safe and works for people with NF1 who have growing or painful plexiform neurofibromas.
- Who it's for
- You may be able to join if you are 16 or older, have NF1, and have a plexiform neurofibroma that is getting bigger or causing symptoms.
- What's involved
- You would take two pills every day, one of each drug. You will have regular check‑ups, blood tests, and scans to see how you feel and how the tumors change.
- Time commitment
- The study lasts about 2 years, with visits every few weeks for the first 16 weeks and then every few months for the rest of the study.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 and older
You may be able to join if
- Males: (140 - age) x weight (kg)/(serum creatinine [mg/dL] × 72)
- Females: [(140 - age) x weight (kg)/(serum creatinine [mg/dL] × 72)] × 0.85 Urine protein/creatinine ratio (UPCR) ≤ 1 mg/mg (≤ 113.2 mg/mmol), or...
You likely can't join if
- 2.1. Evidence of an optic pathway or other low-grade glioma, high-grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor...
- 2.2. Patients with high-grade glioma, atypical or malignant peripheral nerve sheath tumor, or other malignancy who received treatment in the last 12...
- 2.3. Dental braces or prosthesis that interfere with volumetric analysis of the neurofibroma(s).
- 2.4. Prior Therapy: Participants may have received treatment for a PN or other tumor/malignancy but must have fully recovered to baseline or CTCAE ≤...
- Myelosuppressive chemotherapy: Must not have received within 28 days of entry onto this study.
- 2.5. Hematopoietic growth factors: Must not have received a growth factor that supports platelet, red or white cell number or function within 14 days...
See the full eligibility criteria
- Males: (140 - age) x weight (kg)/(serum creatinine [mg/dL] × 72)
- Females: [(140 - age) x weight (kg)/(serum creatinine [mg/dL] × 72)] × 0.85 Urine protein/creatinine ratio (UPCR) ≤ 1 mg/mg (≤ 113.2 mg/mmol), or 24-h urine protein ≤ 1 g. 1.7.3.
- 2.1. Evidence of an optic pathway or other low-grade glioma, high-grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor requiring treatment with chemotherapy, biologic therapy or radiation therapy.
- 2.2. Patients with high-grade glioma, atypical or malignant peripheral nerve sheath tumor, or other malignancy who received treatment in the last 12 months.
- 2.3. Dental braces or prosthesis that interfere with volumetric analysis of the neurofibroma(s).
- 2.4. Prior Therapy: Participants may have received treatment for a PN or other tumor/malignancy but must have fully recovered to baseline or CTCAE ≤ Grade 1 from acute toxicities from prior therapies except alopecia.
- Myelosuppressive chemotherapy: Must not have received within 28 days of entry onto this study.
- 2.5. Hematopoietic growth factors: Must not have received a growth factor that supports platelet, red or white cell number or function within 14 days of initiation of therapy.
- 2.6. Biologic (anti-neoplastic agent): At least 28 days (or 5 half-lives whichever is longer) since the completion of therapy with a biologic agent.
- 2.7. Investigational Drugs: At least 28 days (or 5 half-lives whichever is longer) since the completion of therapy with an investigational drug or systemic anticancer treatment.
- \- Prior treatment with cabozantinib or selumetinib is permitted for participants on Phase 1 of this study only (not Phase 1b/2).
- 2.8. Radiation therapy: 6 months from involved field radiation to index PN(s) must have elapsed prior to study entry
- \>12 weeks must have elapsed between systemic treatment with radionuclides and first dose of study treatment.
- Participants with clinically relevant ongoing complications from prior radiation therapy are not eligible.
- 2.9.1. Participants are not eligible if complete resection of a PN with acceptable morbidity is feasible, or if a participant with a feasible surgical option with minimal risk for surgical morbidity refuses surgery.
- 2.9.2. Any major surgery within 3 months before first dose of study treatment.
- 2.9.3. Any minor surgeries (e.g., the placement of an implanted vascular device, tooth extractions, biopsy, or an invasive operative procedure for procurement of tissue samples or body fluids using a needle or trocar...
- 2.9.4. Participants must have complete wound healing from major surgery or minor surgery before first dose of study treatment.
- 2.10. Concomitant anticoagulation with coumarin agents (e.g., warfarin), low molecular weight heparins, direct thrombin inhibitors (e.g., dabigatran), direct factor Xa inhibitor betrixaban, or platelet inhibitors (e.g...
- 2.11. Clinically significant hematuria, hematemesis, or hemoptysis of \>0.5 teaspoon (2.5ml) of red blood, or other history of significant bleeding (e.g., pulmonary hemorrhage) within 12 weeks before first dose of study...
- 2.12. Known cavitating pulmonary lesion(s) or known endotracheal or endobronchial disease manifestation.
- 2.13. Cardiovascular disorders: 2.13.1. Congestive heart failure New York Heart Association Class 2-4, unstable angina pectoris (Canadian Cardiovascular Society grade II-IV despite medical therapy), prior or current...
- 2.13.2. Stroke (including transient ischemic attack [TIA]), acute coronary syndrome or myocardial infarction (MI), or other ischemic event, or thromboembolic event (e.g., deep venous thrombosis, pulmonary embolism)...
- 2.13.3. Participants with a diagnosis of incidental, subsegmental PE or DVT within 6 months are allowed if stable, asymptomatic, and treated with a stable dose of permitted anticoagulation (aspirin) for at least 1 week...
- 2.13.4. Baseline QTc interval \>450 msec
- 2.14.1. The participant has evidence of tumor invading the GI tract, active peptic ulcer disease, inflammatory bowel disease (e.g., Crohn's disease), diverticulitis, cholecystitis, symptomatic cholangitis or...
- 2.14.2. Abdominal fistula, GI perforation, bowel obstruction, or intra-abdominal abscess within 6 months before first dose of study treatment.
- Note: Complete healing of an intra-abdominal abscess must be confirmed before first dose of study treatment.
- 2.15.1. Current or history of central serous retinopathy
- 2.15.2. Current or history of retinal vein occlusion
- 2.15.3. Known intraocular pressure (IOP) \>21 mmHg (or ULN adjusted by age) or uncontrolled glaucoma (irrespective of IOP).
- 2.15.4. Participants with any other significant abnormality on ophthalmic examination should be discussed with the Study Chair for potential eligibility.
- 2.15.5. Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor or strabismus) or long-standing orbito-temporal PN (such as visual loss, strabismus) will NOT be...
- 2.16.1. Serious non-healing wound, ulcer, or bone fracture.
- 2.16.2. Uncompensated/symptomatic hypothyroidism.
- 2.16.3. Moderate to severe hepatic impairment (Child-Pugh B or C).
- 2.16.4. Active infection
- 2.16.5. A known history of HIV seropositivity or known immunodeficiency. HIV testing will not be required as part of this trial, unless HIV is clinically suspected.
- 2.16.6. Uncontrolled diabetes, severe malnutrition, chronic liver or renal disease
- 2.16.7. History of organ transplant
- 2.17. Pregnant or lactating women.
- 2.18. Inability to swallow tablets.
- 2.19. Previously identified allergy or hypersensitivity to components of the study treatment formulations.
- 2.20. Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of cabozantinib or selumetinib (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea...
- 2.21. Participants who require chronic concomitant treatment of moderate/strong CYP3A4 inducers or inhibitors (Appendix XIII).
- 2.22. Participants with a history of significant noncompliance to medical regimens, are unwilling to or unable to comply with the protocol, or who in the opinion of the investigator may not be able to comply with the...
The study team makes the final eligibility decision.
Where it is enrolling
- Birmingham, Alabama, United States
- Los Angeles, California, United States
- Washington D.C., District of Columbia, United States
- Indianapolis, Indiana, United States
- Boston, Massachusetts, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
Compensation & support
A stipend or payment for your time may be offered.
No amount is published here because ClinicalTrials.gov has no reliable field for payment or travel support. Ask the study team what's actually covered (payment for your time, travel, parking, or other study costs), and never join a trial for the compensation alone.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team, so confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Birmingham, Alabama, United States; Los Angeles, California, United States; Washington D.C., District of Columbia, United States; Indianapolis, Indiana, United States; Boston, Massachusetts, United States; Cincinnati, Ohio, United States and 2 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.