Doxycycline Injection of Cutaneous Schwannoma in Neurofibromatosis Type 2

  • STATUS
    Recruiting
  • End date
    Dec 10, 2024
  • participants needed
    19
  • sponsor
    Massachusetts Eye and Ear Infirmary
Send
Updated on 10 October 2022
See if I qualify

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.

Description

Study Design: Interventional Clinical Trial, single-arm, open-label

Estimated Enrollment: Up to 19 participants with NF2-associated cutaneous or subcutaneous schwannomas

Aims: To demonstrate change in peripheral schwannoma size following direct injection into the tumor with doxycycline hyclate.

Nineteen neurofibromatosis type 2-associated peripheral cutaneous or subcutaneous schwannomas between 0.5 cm and 4 cm in greatest diameter will be diagnosed clinically, photographed for documentation of location, and injected with between 0.1 ml to 4 ml of 10mg/ml doxycycline hyclate (Fresenius Kabi, Canton, MA). A maximum of three tumors per patient will be injected. 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. The tumor volume will be estimated by electronic caliper measure of the greatest tumor diameter (D). (V=4/3piR3 where R is the radius (R=D/2) is measured in millimeters and the dose calculated by Vmm3/1000 = injection volume in milliliters). Repeat measures will be made at 6-months and 1-year follow up (+/- 1 month) and documented with electronic calipers and photography. Injection of 1% lidocaine may be given pre-doxycycline to reduce injection pain.

Outcome: The primary outcome will be longitudinal change in tumor maximal diameter measured at 6-months and 1-year compared to baseline defined by a change of 25%. Secondary endpoints include patient reports of paresthesia, pain, skin breakdown or discoloration. Exploratory endpoints will be the change in the sum of maximal diameters.

Details
Condition Neurofibromatosis Type 2
Treatment Doxycycline Injection [Doxy]
Clinical Study IdentifierNCT05521048
SponsorMassachusetts Eye and Ear Infirmary
Last Modified on10 October 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

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
\. 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

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
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name
Edit Delete

Added by • 

 • 

Private

Edit Delete

Reply by • • Private
Edit Delete

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note