Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

July 6, 2016 updated by: Scott Randall Plotkin, MD, PhD, Massachusetts General Hospital

Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

11

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Ranibizumab
Drug: Ranibizumab
Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment.
Other Names:
  • Lucentis

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.
Time Frame: 2 years
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts General Hospital

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2008

Primary Completion (ACTUAL)

October 1, 2009

Study Completion (ACTUAL)

December 1, 2013

Study Registration Dates

First Submitted

March 27, 2008

First Submitted That Met QC Criteria

April 9, 2008

First Posted (ESTIMATE)

April 14, 2008

Study Record Updates

Last Update Posted (ESTIMATE)

July 7, 2016

Last Update Submitted That Met QC Criteria

July 6, 2016

Last Verified

July 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 07-332

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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