Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

NCT00657202 | Completed Early Phase 1 DMC

• 1 other identifier: 07-332
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neurofibromatosis Type 1; Cutaneous Neurofibromas

Keywords

NF1; neurofibroma; ranibizumab; Lucentis

Outcome Measures

Primary Outcomes (1)

• Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas.

  2 years

Secondary Outcomes (1)

• To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.

  2 years

Study Arms (1)

Ranibizumab

EXPERIMENTAL

Drug: Ranibizumab

Interventions

Ranibizumab DRUG

Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment.

Also known as: Lucentis

Ranibizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• years of age or older
• Normal organ and marrow function as described in the protocol

You may not qualify if:

• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma

Interventions

Ranibizumab

Condition Hierarchy (Ancestors)

Neurofibromatoses; 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; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Scott Plotkin, MD, PhD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: March 27, 2008
• First Posted: April 14, 2008
• Study Start: March 1, 2008
• Primary Completion: October 1, 2009
• Study Completion: December 1, 2013
• Last Updated: July 7, 2016

Record last verified: 2016-07

Locations

US (1)