Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

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Brief Title

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

Official Title

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

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 Phase

Early Phase 1

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.

Condition

Neurofibromatosis Type 1

Intervention

Ranibizumab

Study Arms / Comparison Groups

 Ranibizumab
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

11

Start Date

March 2008

Completion Date

December 2013

Primary Completion Date

October 2009

Eligibility 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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Scott Plotkin, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00657202

Organization ID

07-332


Responsible Party

Principal Investigator

Study Sponsor

Massachusetts General Hospital


Study Sponsor

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


Verification Date

July 2016