Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1
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ClinicalTrials.gov Identifier: NCT00657202 |
Recruitment Status :
Completed
First Posted : April 14, 2008
Last Update Posted : July 7, 2016
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Neurofibromatosis Type 1 Cutaneous Neurofibromas | Drug: Ranibizumab | Early Phase 1 |
- Participants will receive one injection of ranibizumab into 3 tumors on their skin (one dose per tumor) on day 1 of treatment. One other tumor will be injected with normal saline solution. The saline solution-called a control-is necessary to determine whether injections (without medicine) can cause a tumor to shrink. Tumors will be measured and photographed prior to treatment. Ranibizumab tumors will be removed on days 8, 15, and 29; the saline treated tumor will be removed on day 29.
- Participants will come into the clinic once a week for a total of 4 weeks and then again on Days 35. 57 and 85 for post-treatment visits. Some of the following tests and procedures will be performed: physical examination (including photographs of tumors), review of current medications, vital signs, routine blood tests, serum chemistry blood tests, interstitial fluid pressure measurements and tumor samples.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 11 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1 |
Study Start Date : | March 2008 |
Actual Primary Completion Date : | October 2009 |
Actual Study Completion Date : | December 2013 |

Arm | Intervention/treatment |
---|---|
Experimental: Ranibizumab |
Drug: Ranibizumab
Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment.
Other Name: Lucentis |
- Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas. [ Time Frame: 2 years ]
- To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab. [ Time Frame: 2 years ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients must have diagnosis of NF1 based on NIH criteria with two or more of the following characteristics: a) Six or more cafe-au-lait macules b) Skin fold freckling in the axilla or groin c) Optic pathway glioma d) Two or more Lisch nodules of the iris e) Distinctive bony lesions such as dysplasia of the sphenoid wing or of a long bone such as the tibia f) Two or more neurofibromas of any type or 1 or more plexiform neurofibroma g) First degree relative with NF1
- At least four cutaneous neurofibromas on skin exam with the following qualities: a) the lesion must be discrete by clinical exam and must be at least 5mm away from another skin tumor b) the lesion must be amenable to measurement with calipers with minimum dimension of 5mm and maximum dimension of 20mm c)the lesions cannot be located on the face, scalp, or groin and must be located in an area that can be photographed d) histologic confirmation of tumor type is not required in the setting of compatible clinical setting
- Must be willing to have treated CNF surgically removed during the study
- 18 years of age or older
- Normal organ and marrow function as described in the protocol
Exclusion Criteria:
- Pregnant or breast-feeding women
- Chemotherapy or radiotherapy within 6 weeks prior to entering the study
- Receiving any other investigational agent
- History of allergic reactions attributed to compounds of similar chemical or biologic composition as ranibizumab
- Hypertension that cannot be controlled by medications
- Known coagulopathy that increases risk of bleeding or a history of clinically significant hemorrhages in the past
- NCI CTCAE grade 3 hemorrhage within 4 weeks of starting the study treatment
- Pre-existing thyroid abnormality with thyroid function that cannot be maintained in the normal range with medication
- Uncontrolled intercurrent illness

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00657202
United States, Massachusetts | |
Massachusetts General Hospital | |
Boston, Massachusetts, United States, 02114 |
Principal Investigator: | Scott Plotkin, MD, PhD | Massachusetts General Hospital |
Responsible Party: | Scott Randall Plotkin, MD, PhD, Associate Professor, Massachusetts General Hospital |
ClinicalTrials.gov Identifier: | NCT00657202 History of Changes |
Other Study ID Numbers: |
07-332 |
First Posted: | April 14, 2008 Key Record Dates |
Last Update Posted: | July 7, 2016 |
Last Verified: | July 2016 |
Keywords provided by Scott Randall Plotkin, MD, PhD, Massachusetts General Hospital:
NF1 neurofibroma ranibizumab Lucentis |
Additional relevant MeSH terms:
Neurofibromatoses Neurofibromatosis 1 Neurofibroma Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neoplasms by Histologic Type Neoplasms Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn |
Peripheral Nervous System Diseases Neuromuscular Diseases Peripheral Nervous System Neoplasms Nervous System Neoplasms Ranibizumab Immunologic Factors Physiological Effects of Drugs Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Growth Inhibitors Antineoplastic Agents |