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Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

Primary Purpose

Neurofibromatosis Type 1, Cutaneous Neurofibromas

Status

Completed

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

Ranibizumab

About this trial

This is an interventional treatment trial for Neurofibromatosis Type 1 focused on measuring NF1, neurofibroma, ranibizumab, Lucentis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Massachusetts General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ranibizumab

Arm Description

Outcomes

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.

Secondary Outcome Measures

To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.

Full Information

NCT ID

NCT00657202

First Posted

March 27, 2008

Last Updated

July 6, 2016

Sponsor

Massachusetts General Hospital

1. Study Identification

Unique Protocol Identification Number

NCT00657202

Brief Title

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2016

Overall Recruitment Status

Completed

Study Start Date

March 2008 (undefined)

Primary Completion Date

October 2009 (Actual)

Study Completion Date

December 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Massachusetts General Hospital

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Neurofibromatosis Type 1, Cutaneous Neurofibromas

Keywords

NF1, neurofibroma, ranibizumab, Lucentis

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

11 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Ranibizumab

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Ranibizumab

Other Intervention Name(s)

Lucentis

Intervention Description

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

Primary Outcome Measure Information:

Title

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

Secondary Outcome Measure Information:

Title

To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.

Time Frame

2 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Scott Plotkin, MD, PhD

Organizational Affiliation

Massachusetts General Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

12. IPD Sharing Statement

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Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

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