Study of Aspirin in Patients With Vestibular Schwannoma

Prospective, Randomized, Placebo-Controlled Phase II Trial of Aspirin for Vestibular Schwannomas

This is a phase II prospective, randomized, double-blind, longitudinal study evaluating whether the administration of aspirin can delay or slow tumor growth and maintain or improve hearing in VS patients.

Study Overview

• Status: Active, not recruiting
• Conditions: Neurofibromatosis 2; Vestibular Schwannoma; Acoustic Neuroma
• Intervention / Treatment: Drug: Aspirin; Drug: Placebo

Detailed Description

This is a phase II prospective, randomized, double-blind, longitudinal study evaluating whether the administration of aspirin can delay or slow tumor growth and maintain or improve hearing in VS patients. In the Randomized Treatment Phase, patients aged 12 years or older with VSs will be identified and recruited into one of two randomized groups who receive either 1) 325 mg aspirin twice daily (81 mg aspirin twice daily for pediatric patients <50 kg); or 2) a placebo. Patients will stay on aspirin/placebo as long as there is no more than 20% increase in VS volume. At tumor progression, patients will enter the Open Label Follow Up Phase. Patients who progress on placebo will be given the option to receive unblinded aspirin and will be followed until further progression, receiving a definitive treatment (e.g, surgery or radiation), or 42 months post-baseline, whichever occurs first. Patients who progress on aspirin (either blinded or unblinded) will be taken off study drug and followed until they receive a definitive treatment (e.g., surgery or radiation) or reach 42 months post-baseline, whichever comes first.

• Study Type: Interventional
• Enrollment (Actual): 97
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94304
      • Stanford Otolaryngology/HNS
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts Eye and Ear
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Utah
    • Salt Lake City, Utah, United States, 84101
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Radiographic diagnosis of vestibular schwannoma (NF2-associated or sporadic).
• Age≥12 years.
• Ability to provide informed consent.Pediatric patients must provide assent in addition to their parents'/guardians' consent. Adult patients who cannot consent for themselves will not be eligible to participate in this study.
• Ability to swallow tablets.

Exclusion Criteria:

• Inability to perform volumetric measurements of vestibular schwannoma(s).
• Inability to tolerate MRI with contrast.
• Daily use of aspirin within the last two months. Occasional use of aspirin for pain relief is not exclusionary.
• Known allergy to aspirin.
• Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of aspirin.
• Pregnant or lactating women.
• Patients with serious medical illnesses (e.g. severe asthma) that in the option of the investigator could prevent participation in the trial.
• Active bleeding diathesis.
• Hydrocephalus from brainstem compression.
• Febrile illness or flu-like illness in children and adolescents less than 18 years of age.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Aspirin; Patients on the experimental arm will receive blinded aspirin. Pediatric subjects who weigh less than 110 lbs will take 81mg aspirin twice a day. All other subjects will take 325mg aspirin twice a day.	Drug: Aspirin; Twice daily aspirin; Other Names: Bayer; Zoprin; Durlaza
Placebo Comparator: Placebo; Patients on the placebo arm will receive blinded placebo and take it twice a day.	Drug: Placebo; Twice daily placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression Free Survival	Progression Free Survival (PFS) is the length of time from start of study treatment to tumor growth of more than 20%.	Progression, or around 3.5 years

Collaborators and Investigators

• Sponsor: Massachusetts Eye and Ear Infirmary
• Collaborators: United States Department of Defense
• Investigators: Principal Investigator: Konstantina Stankovic, MD, PhD, Stanford University; Principal Investigator: D. Bradley Welling, MD, PhD, Massachusetts Eye and Ear

Study record dates

Study Major Dates

• Study Start (Actual): June 11, 2018
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): February 1, 2029

Study Registration Dates

• First Submitted: March 6, 2017
• First Submitted That Met QC Criteria: March 9, 2017
• First Posted (Actual): March 15, 2017

Study Record Updates

• Last Update Posted (Estimated): December 18, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Genetic Diseases, Inborn; Neoplasms by Histologic Type; Neurodegenerative Diseases; Otorhinolaryngologic Diseases; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neuroendocrine Tumors; Ear Diseases; Otorhinolaryngologic Neoplasms; Cranial Nerve Diseases; Neuroma; Cranial Nerve Neoplasms; Vestibulocochlear Nerve Diseases; Retrocochlear Diseases; Neurofibroma; Neurilemmoma; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurofibromatoses; Neuroma, Acoustic; Neurofibromatosis 2; Organic Chemicals; Hydrocarbons; Hydrocarbons, Cyclic; Hydrocarbons, Aromatic; Phenols; Benzene Derivatives; Salicylates; Hydroxybenzoates; Aspirin
• Other Study ID Numbers: 17-030; W81XWH-17-1-0644 (Other Grant/Funding Number: Congressionally Directed Medical Research Program)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No