Neurofibromatosis Type 2Phase 1/2NCT05521048
Doxycycline in Cutaneous Schwannoma (NF2)
A Phase 1/2 study testing Doxycycline Injection [Doxy] in people with Neurofibromatosis Type 2, run by Massachusetts Eye and Ear Infirmary at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Neurofibromatosis Type 2
- What is being tested?
- Doxycycline Injection [Doxy]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Sep 2022to 30 Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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:
Show all 20Show fewer
- 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.
- Life expectancy of greater than 1 year
- Lansky/Karnofsky performance status ≥ 60
- Fully recovered from acute toxic effects of any prior chemotherapy, biological modifiers or radiotherapy
- Any neurologic deficits must be stable for ≥1 week
- Patient or parent/legal guardian must be able to provide signed informed consent and assent (as applicable for minors)
Who cannot join
- 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
Show all 14Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts Eye and Ear
Boston, Massachusetts, 02214
About this study
Official title: Doxycycline Injection of Cutaneous Schwannoma in Neurofibromatosis Type 2
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.
Read the detailed 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.
- Conditions
- Neurofibromatosis Type 2
- Being tested
- Doxycycline Injection [Doxy]
- Keywords
- Cutaneous schwannoma, Doxycycline
- Medical subject headings
- Neurofibromatosis 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Doxycycline Injection [Doxy]Drug
Also called Doxycycline Hyclate
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.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm Open Label | Experimental | Open Label Receives: Drug: Doxycycline Injection [Doxy] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tumor Maximal Diameter
Longitudinal change in tumor maximal diameter
Measured: 6-months, 1-year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2022
- Study start19 Sep 2022
- Primary completion31 Dec 2026 (estimated)
- Study completion30 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (28)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis Type 2Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts Eye and Ear Infirmary
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, D. Bradley Welling, MD, PhD, Massachusetts Eye and Ear Infirmary
- Investigators
- D. Bradley Welling, MD, PhD, Principal investigator, Massachusetts Eye and Ear Infirmary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05521048
- Sponsor's protocol ID
- 2022P000534
- Status last verified
- Oct 2026
- First submitted
- 26 Aug 2022
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ren Y, Chari DA, Vasilijic S, Welling DB, Stankovic KM. New developments in neurofibromatosis type 2 and vestibular schwannoma. Neurooncol Adv. 2020 Nov 16;3(1):vdaa153. doi: 10.1093/noajnl/vdaa153. eCollection 2021 Jan-Dec.
PubMed 33604573Evans DG. Neurofibromatosis type 2 (NF2): a clinical and molecular review. Orphanet J Rare Dis. 2009 Jun 19;4:16. doi: 10.1186/1750-1172-4-16.
PubMed 19545378Ostrom QT, Cioffi G, Waite K, Kruchko C, Barnholtz-Sloan JS. CBTRUS Statistical Report: Primary Brain and Other Central Nervous System Tumors Diagnosed in the United States in 2014-2018. Neuro Oncol. 2021 Oct 5;23(12 Suppl 2):iii1-iii105. doi: 10.1093/neuonc/noab200.
PubMed 34608945Balasubramaniam A, Shannon P, Hodaie M, Laperriere N, Michaels H, Guha A. Glioblastoma multiforme after stereotactic radiotherapy for acoustic neuroma: case report and review of the literature. Neuro Oncol. 2007 Oct;9(4):447-53. doi: 10.1215/15228517-2007-027. Epub 2007 Aug 17.
PubMed 17704364Coy S, Rashid R, Stemmer-Rachamimov A, Santagata S. Correction to: An update on the CNS manifestations of neurofibromatosis type 2. Acta Neuropathol. 2020 Apr;139(4):667. doi: 10.1007/s00401-019-02044-6.
PubMed 31432207Petrilli AM, Garcia J, Bott M, Klingeman Plati S, Dinh CT, Bracho OR, Yan D, Zou B, Mittal R, Telischi FF, Liu XZ, Chang LS, Welling DB, Copik AJ, Fernandez-Valle C. Ponatinib promotes a G1 cell-cycle arrest of merlin/NF2-deficient human schwann cells. Oncotarget. 2017 May 9;8(19):31666-31681. doi: 10.18632/oncotarget.15912.
PubMed 28427224Cosetti MK, Golfinos JG, Roland JT Jr. Quality of Life (QoL) Assessment in Patients with Neurofibromatosis Type 2 (NF2). Otolaryngol Head Neck Surg. 2015 Oct;153(4):599-605. doi: 10.1177/0194599815573002. Epub 2015 Mar 16.
PubMed 25779467Marchi E, Teixeira LR, Vargas FS. Management of malignancy-associated pleural effusion: current and future treatment strategies. Am J Respir Med. 2003;2(3):261-73. doi: 10.1007/BF03256654.
PubMed 14720007Godfrey KJ, Kally P, Dunbar KE, Campbell AA, Callahan AB, Lo C, Freund R, Lisman RD. Doxycycline Injection for Sclerotherapy of Lower Eyelid Festoons and Malar Edema: Preliminary Results. Ophthalmic Plast Reconstr Surg. 2019 Sep/Oct;35(5):474-477. doi: 10.1097/IOP.0000000000001332.
PubMed 30882591Shaye DA, Burks CA, Gadkaree SK, Ncogoza I, Tuyishimire G, Nyabyenda V, Gassore A. Self-compounded Doxycycline Sclerotherapy for the Treatment of Lymphatic Malformations in Low-Resource Settings. World J Surg. 2020 Nov;44(11):3616-3619. doi: 10.1007/s00268-020-05667-z. Epub 2020 Jul 8.
PubMed 32642795Farnoosh S, Don D, Koempel J, Panossian A, Anselmo D, Stanley P. Efficacy of doxycycline and sodium tetradecyl sulfate sclerotherapy in pediatric head and neck lymphatic malformations. Int J Pediatr Otorhinolaryngol. 2015 Jun;79(6):883-887. doi: 10.1016/j.ijporl.2015.03.024. Epub 2015 Apr 6.
PubMed 25887132Tang SJ, Sreenarasimhaiah J, Tang L, Rollins N, Purdy PD. Endoscopic injection sclerotherapy with doxycycline for mediastinal and esophageal lymphangiohemangioma. Gastrointest Endosc. 2007 Dec;66(6):1196-200. doi: 10.1016/j.gie.2007.06.023.
PubMed 18061720Woon JTK, Hoon D, Graydon A, Flint M, Doyle AJ. Aneurysmal bone cyst treated with percutaneous doxycycline: is a single treatment sufficient? Skeletal Radiol. 2019 May;48(5):765-771. doi: 10.1007/s00256-019-03188-y. Epub 2019 Feb 26.
PubMed 30809704Zhu C, Yan X, Yu A, Wang Y. Doxycycline synergizes with doxorubicin to inhibit the proliferation of castration-resistant prostate cancer cells. Acta Biochim Biophys Sin (Shanghai). 2017 Nov 1;49(11):999-1007. doi: 10.1093/abbs/gmx097.
PubMed 28985240Alsaadi M, Tezcan G, Garanina EE, Hamza S, McIntyre A, Rizvanov AA, Khaiboullina SF. Doxycycline Attenuates Cancer Cell Growth by Suppressing NLRP3-Mediated Inflammation. Pharmaceuticals (Basel). 2021 Aug 26;14(9):852. doi: 10.3390/ph14090852.
PubMed 34577552
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Doxycycline Injection of Cutaneous Schwannoma in Neurofibromatosis Type 2
- ClinicalTrials.gov ID
- NCT05521048
- Acronym / sponsor ID
- 2022P000534
- Conditions
- Neurofibromatosis Type 2
- Keywords
- Cutaneous schwannoma, Doxycycline
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (estimated)
- Lead sponsor
- Massachusetts Eye and Ear InfirmaryUniversity, hospital or other
- Study start
- 19 Sep 2022 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 Mar 2027 (estimated)
- First posted
- 30 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm Open Label | Experimental | Drug: Doxycycline Injection [Doxy]Open Label |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- 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.
- Patients must have measurable disease, defined as at least one cutaneous/subcutaneous schwannoma with the following qualities:
- Age ≥ 8 years on day 1 of treatment.
- Life expectancy of greater than 1 year
- Lansky/Karnofsky performance status ≥ 60
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Doxycycline Injection [Doxy]Also: Doxycycline HyclateInjections 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. | Drug | Single Arm Open Label |
Primary outcome
Tumor Maximal Diameter
Time frame: 6-months, 1-year
Longitudinal change in tumor maximal diameter
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Background: PubMed 33604573Ren Y, Chari DA, Vasilijic S, Welling DB, Stankovic KM. New developments in neurofibromatosis type 2 and vestibular schwannoma. Neurooncol Adv. 2020 Nov 16;3(1):vdaa153. doi: 10.1093/noajnl/vdaa153. eCollection 2021 Jan-Dec.
- Background: PubMed 19545378Evans DG. Neurofibromatosis type 2 (NF2): a clinical and molecular review. Orphanet J Rare Dis. 2009 Jun 19;4:16. doi: 10.1186/1750-1172-4-16.
- Background: PubMed 34608945Ostrom QT, Cioffi G, Waite K, Kruchko C, Barnholtz-Sloan JS. CBTRUS Statistical Report: Primary Brain and Other Central Nervous System Tumors Diagnosed in the United States in 2014-2018. Neuro Oncol. 2021 Oct 5;23(12 Suppl 2):iii1-iii105. doi: 10.1093/neuonc/noab200.
- Background: PubMed 17704364Balasubramaniam A, Shannon P, Hodaie M, Laperriere N, Michaels H, Guha A. Glioblastoma multiforme after stereotactic radiotherapy for acoustic neuroma: case report and review of the literature. Neuro Oncol. 2007 Oct;9(4):447-53. doi: 10.1215/15228517-2007-027. Epub 2007 Aug 17.
- Background: PubMed 31432207Coy S, Rashid R, Stemmer-Rachamimov A, Santagata S. Correction to: An update on the CNS manifestations of neurofibromatosis type 2. Acta Neuropathol. 2020 Apr;139(4):667. doi: 10.1007/s00401-019-02044-6.
- Background: PubMed 28427224Petrilli AM, Garcia J, Bott M, Klingeman Plati S, Dinh CT, Bracho OR, Yan D, Zou B, Mittal R, Telischi FF, Liu XZ, Chang LS, Welling DB, Copik AJ, Fernandez-Valle C. Ponatinib promotes a G1 cell-cycle arrest of merlin/NF2-deficient human schwann cells. Oncotarget. 2017 May 9;8(19):31666-31681. doi: 10.18632/oncotarget.15912.
- Background: PubMed 25779467Cosetti MK, Golfinos JG, Roland JT Jr. Quality of Life (QoL) Assessment in Patients with Neurofibromatosis Type 2 (NF2). Otolaryngol Head Neck Surg. 2015 Oct;153(4):599-605. doi: 10.1177/0194599815573002. Epub 2015 Mar 16.
- Background: PubMed 14720007Marchi E, Teixeira LR, Vargas FS. Management of malignancy-associated pleural effusion: current and future treatment strategies. Am J Respir Med. 2003;2(3):261-73. doi: 10.1007/BF03256654.
- Background: PubMed 30882591Godfrey KJ, Kally P, Dunbar KE, Campbell AA, Callahan AB, Lo C, Freund R, Lisman RD. Doxycycline Injection for Sclerotherapy of Lower Eyelid Festoons and Malar Edema: Preliminary Results. Ophthalmic Plast Reconstr Surg. 2019 Sep/Oct;35(5):474-477. doi: 10.1097/IOP.0000000000001332.
- Background: PubMed 32642795Shaye DA, Burks CA, Gadkaree SK, Ncogoza I, Tuyishimire G, Nyabyenda V, Gassore A. Self-compounded Doxycycline Sclerotherapy for the Treatment of Lymphatic Malformations in Low-Resource Settings. World J Surg. 2020 Nov;44(11):3616-3619. doi: 10.1007/s00268-020-05667-z. Epub 2020 Jul 8.
- Background: PubMed 25887132Farnoosh S, Don D, Koempel J, Panossian A, Anselmo D, Stanley P. Efficacy of doxycycline and sodium tetradecyl sulfate sclerotherapy in pediatric head and neck lymphatic malformations. Int J Pediatr Otorhinolaryngol. 2015 Jun;79(6):883-887. doi: 10.1016/j.ijporl.2015.03.024. Epub 2015 Apr 6.
- Background: PubMed 18061720Tang SJ, Sreenarasimhaiah J, Tang L, Rollins N, Purdy PD. Endoscopic injection sclerotherapy with doxycycline for mediastinal and esophageal lymphangiohemangioma. Gastrointest Endosc. 2007 Dec;66(6):1196-200. doi: 10.1016/j.gie.2007.06.023.
Related trials for Neurofibromatosis Type 2
RecruitingObservational study
The NF Registry Sponsored by the Children's Tumor Foundation
Neurofibromatosis 1, Neurofibromatosis 2
RecruitingPhase 2
Vismodegib, FAK Inhibitor GSK2256098, Capivasertib, and Abemaciclib in Treating Patients With Progressive Meningiomas
Intracranial Meningioma, Recurrent Meningioma
RecruitingObservational study
Natural History Study of Patients With Neurofibromatosis Type 2
Neurofibromatosis
Active, not recruitingPhase 2
Study of Aspirin in Patients With Vestibular Schwannoma
Vestibular Schwannoma, Acoustic Neuroma
Active, not recruitingPhase 2
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
D. Bradley Welling, MD, PhD principal investigator
Massachusetts Eye and Ear Infirmary
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Doxycycline Injection [Doxy] in people with Neurofibromatosis Type 2, run by Massachusetts Eye and Ear Infirmary at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor Maximal Diameter (time frame: 6-months, 1-year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05521048, which also lists 28 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.