Local doxycycline treatment for skin tumors in neurofibromatosis type 2
Doxycycline Injection of Cutaneous Schwannoma in Neurofibromatosis Type 2
This study is testing if local injections of doxycycline can help shrink skin tumors in people with neurofibromatosis type 2, potentially reducing the need for surgery.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 19 (estimated) |
| Ages | 8 Years and up |
| Sex | All |
| Sponsor | Massachusetts Eye and Ear Infirmary Academic / other |
| Drugs / interventions | chemotherapy, Radiation |
| Locations | 1 site (Boston, Massachusetts) |
| Trial ID | NCT05521048 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the effectiveness of local doxycycline injections as a treatment for cutaneous schwannomas associated with neurofibromatosis type 2 (NF2). The study will involve up to 19 participants who will receive intra-tumoral injections of doxycycline hyclate, with the goal of reducing tumor size and minimizing the need for surgical removal. Participants will be monitored for changes in tumor size over a period of 6 months to 1 year, with assessments made using electronic calipers and photography. The trial is designed as a single-arm, open-label study.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with neurofibromatosis type 2 who have cutaneous or subcutaneous schwannomas between 0.5 cm and 4 cm in size.
Not a fit: Patients with tumors larger than 4 cm or those who do not meet the diagnostic criteria for neurofibromatosis type 2 may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a less invasive alternative to surgery for patients with NF2-related skin schwannomas.
How similar studies have performed: While the use of doxycycline for treating tumors is an innovative approach, similar studies have not been widely conducted, making this a novel investigation.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. 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. 2. 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 3. Age ≥ 8 years on day 1 of treatment. 4. Life expectancy of greater than 1 year 5. Lansky/Karnofsky performance status ≥ 60 6. Fully recovered from acute toxic effects of any prior chemotherapy, biological modifiers or radiotherapy 7. Any neurologic deficits must be stable for ≥1 week 8. Patient or parent/legal guardian must be able to provide signed informed consent and assent (as applicable for minors) Exclusion Criteria: 1. Allergy to doxycycline or tetracycline 2. Tumors located on the face or major motor nerves 3. 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 4. 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 5. Prior treatment with any investigational drug within the preceding 4 weeks, as they may interfere with the study drug 6. 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 7. 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) 8. 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. 9. History of significant noncompliance with follow-up that would jeopardize the study evaluation. 10. Patients unwilling to or unable to comply with the study protocol.
Where this trial is running
Boston, Massachusetts
- Massachusetts Eye and Ear — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: D. Bradley Welling, MD, PhD — Massachusetts Eye and Ear Infirmary
- Study coordinator: Christopher Wood
- Email: cgwood@meei.harvard.edu
- Phone: 6175733632
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.