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

Phase 0
18 Years
Open (Enrolling)
Neurofibromatosis Type 1, Cutaneous Neurofibromas

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Trial Information

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

- 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.

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

Type of Study:


Study Design:

Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Scott Plotkin, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Institutional Review Board

Study ID:




Start Date:

March 2008

Completion Date:

December 2013

Related Keywords:

  • Neurofibromatosis Type 1
  • Cutaneous Neurofibromas
  • NF1
  • neurofibroma
  • ranibizumab
  • Lucentis
  • Neurofibroma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica



Massachusetts General Hospital Boston, Massachusetts  02114-2617