Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1 - Full Text View

Purpose

The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. It targets a substance in the body called VEGF (Vascular Endothelial Growth Factor). VEGF helps tumors to grow and survive by supporting the growth of blood vessels that bring nutrients to the tumor. VEGF is made by cancerous tumors and also by non-cancerous tumors such as neurofibromas.

Condition	Intervention	Phase
Neurofibromatosis Type 1 Cutaneous Neurofibromas	Drug: Ranibizumab	Phase 0


Study Type:	Interventional
Study Design:	Endpoint Classification: Pharmacodynamics Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1

Resource links provided by NLM:

Genetics Home Reference related topics: neurofibromatosis type 1 neurofibromatosis type 2

MedlinePlus related topics: Neurofibromatosis

Drug Information available for: Ranibizumab

Genetic and Rare Diseases Information Center resources: Neurofibromatosis Neurofibroma Neurofibromatosis Type 1

U.S. FDA Resources

Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab. [ Time Frame: 2 years ] [ Designated as safety issue: No ]


Enrollment:	11
Study Start Date:	March 2008
Study Completion Date:	December 2013
Primary Completion Date:	October 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Ranibizumab	Drug: Ranibizumab Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment. Other Name: Lucentis

Detailed Description:

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.

Eligibility


Ages Eligible for Study:	18 Years and older (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00657202

Locations


United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114

Sponsors and Collaborators

Massachusetts General Hospital

Investigators


Principal Investigator:	Scott Plotkin, MD, PhD	Massachusetts General Hospital

More Information


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
Study First Received:	March 27, 2008
Last Updated:	July 6, 2016
Health Authority:	United States: Institutional Review Board

Keywords provided by Massachusetts General Hospital:


NF1 neurofibroma ranibizumab Lucentis

Additional relevant MeSH terms:


Neurofibromatoses Neurofibroma Neurofibromatosis 1 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 Neoplasms Nervous System Neoplasms Peripheral Nervous System Diseases Neuromuscular Diseases Ranibizumab Immunologic Factors Physiological Effects of Drugs Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Growth Inhibitors Antineoplastic Agents

ClinicalTrials.gov processed this record on September 28, 2016