Doxycycline in Cutaneous Schwannoma (NF2)

NCT05521048 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: 2022P000534
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

In this research study the investigators want to learn more about an alternate, local treatment for skin schwannomas. Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for NF2-related skin schwannomas, ultimately reducing the risks and costs associated with standard surgical removal of such skin tumors if successful.

Conditions

Neurofibromatosis Type 2

Keywords

Cutaneous schwannoma; Doxycycline

Outcome Measures

Primary Outcomes (1)

• Tumor Maximal Diameter

  Longitudinal change in tumor maximal diameter

  6-months, 1-year

Study Arms (1)

Single Arm Open Label

EXPERIMENTAL

Open Label

Drug: Doxycycline Injection [Doxy]

Interventions

Doxycycline Injection [Doxy] DRUG

Injections between 0.1 ml to 4 ml of 10mg/ml doxycycline hyclate will be administered to a maximum of three tumors per patient. The dose administered will be calculated based on tumor size, not to exceed the estimated volume of the tumor or a maximum of 4 ml. Injection of 1% lidocaine may be given pre-doxycycline to reduce injection pain.

Also known as: Doxycycline Hyclate

Single Arm Open Label

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have a confirmed diagnosis of neurofibromatosis 2 by fulfilling National Institute of Health (NIH) criteria or Manchester criteria, or by detection of a causative mutation in the NF2 gene.
• The NIH criteria include presence of:
• Bilateral vestibular schwannomas, OR
• First-degree relative with NF2 and EITHER unilateral eighth nerve mass OR
• Two of the following: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity.
• The Manchester criteria include presence of:
• Bilateral vestibular schwannomas, OR First-degree relative with NF2 and EITHER unilateral eighth nerve mass OR - Two of the following: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity OR
• Unilateral vestibular schwannoma AND any two of: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity, OR
• Multiple meningiomas (two or more) AND unilateral vestibular schwannoma OR
• Any two of: schwannoma, glioma, neurofibroma, cataract.
• Patients must have measurable disease, defined as at least one cutaneous/subcutaneous schwannoma with the following qualities:
• Maximal tumor diameter \> 0.5 cm to \< 4.0 cm that can be accurately measured by electronic calipers
• Up to a maximum of 3 tumors/subject may be injected
• Not located on the face
• Age ≥ 8 years on day 1 of treatment.

You may not qualify if:

• Allergy to doxycycline or tetracycline
• Tumors located on the face or major motor nerves
• Patients currently receiving medical anticancer therapies or who have received medical anticancer therapies within 4 weeks of the start of study drug (including chemotherapy and molecular targeted agents), as these may interfere with the study drug
• Radiation therapy to a study target tumor within 1 year prior to enrollment, or any radiation therapy within 4 weeks prior to enrollment, as these may interfere with our ability to assess response to study drug
• Prior treatment with any investigational drug within the preceding 4 weeks, as they may interfere with the study drug
• Unstable or rapidly progressive disease, including patients who require glucocorticoids for symptomatic control of brain or spinal tumors, as this would represent a high risk for inability to comply with the study requirements
• Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
• symptomatic congestive heart failure of New York heart Association Class III or IV
• unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease
• severely impaired lung function as defined as spirometry and DLCO that is 50% of the normal predicted value and/or O2 saturation that is 90% or less at rest on room air
• active (acute or chronic) or uncontrolled severe infections liver disease, such as cirrhosis or severe hepatic impairment (Child-Pugh class C)
• Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. Adequate contraception (oral contraceptives, contraceptive implants, vaginal ring, or intrauterine devices (IUDs)) must be used at the time of injection but does not need to be carried out past the 1st month of observation.
• History of significant noncompliance with follow-up that would jeopardize the study evaluation.
• Patients unwilling to or unable to comply with the study protocol.

Sponsors & Collaborators

• Massachusetts Eye and Ear Infirmary: lead

Study Sites (1)

Massachusetts Eye and Ear

Boston, Massachusetts, 02214, United States

Location

Related Publications (28)

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MeSH Terms

Conditions

Neurofibromatosis 2

Interventions

Doxycycline

Condition Hierarchy (Ancestors)

Neuroma, Acoustic; Neurilemmoma; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neuroma; Neoplastic Syndromes, Hereditary; Vestibulocochlear Nerve Diseases; Retrocochlear Diseases; Ear Diseases; Otorhinolaryngologic Diseases; Otorhinolaryngologic Neoplasms; Cranial Nerve Neoplasms; Cranial Nerve Diseases; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Tetracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• D. Bradley Welling, MD, PhD

  Massachusetts Eye and Ear Infirmary

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor Harvard Department of Otolaryngology-Head and Neck Surgery

Study Record Dates

• First Submitted: August 26, 2022
• First Posted: August 30, 2022
• Study Start: September 19, 2022
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: December 8, 2025

Record last verified: 2025-12

Locations

US (1)