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Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1

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

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Trial Information

Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1

- Three of the participant's tumors will be treated with imiquimod cream and one tumor
(out of all the remaining tumors) will be followed without treatment (control tumor).

- Participants will be given a tube of imiquimod cream and be asked to apply it to the
three tumors 5 times per week, for a full 6 weeks (Monday through Friday).

- Participants will be required to come to the hospital for examinations on Week 1, 2, 4
and 6 (+/- 3 calendar days) and after their last dose of imiquimod cream on Weeks 9, 12
and 18 (+/- 5 calendar days. The following tests and procedures will be performed: skin
test; vital signs and measurements and photographs of the tumors. Participants will be
asked to participate in an optional skin biopsy on Week 4 and research blood tests wil
lbe taken on Day 1.

Inclusion Criteria:

- Patients must have a diagnosis of NF1 based on NIH criterial with two or more of the
following characteristics:

- six or more cafe-au-lait macules (1.5cm or greater in size)

- skin fold freckling in the axilla or groin

- optic pathway glioma

- two or more Lisch nodules of the iris

- distinctive bony lesions such as dysplasia of the sphenoid wing or of a long
bone such as the tibia

- two or more neurofibromas of any type of 1 or more plexiform neurofibroma

- first degree relative with NF1

- Participants must have at least four cutaneous neurofibromas on skin exam with the
following qualities:

- the lesion must be discrete by clinical exam and must not be contact with
another skin tumor

- the lesion must be amenable to measurement with calipers with minimum dimension
of 5mm and maximum dimension of 30mm

- the lesions must be located on the trunk, neck, or extremities (excluding the
hands and feet) and must be located in an area that can be photographed

- histologic confirmation of tumor type is not required in the setting of
compatible clinical setting

- No treatment with an investigation agent for cutaneous neurofibromas within the last
3 months

- 18 years of age or older

Exclusion Criteria:

- Pregnant and nursing women

- Patients who have had chemotherapy or radiotherapy within 6 weeks prior to entering
the study or those who have not recovered from adverse events due to agents
administered more than 6 weeks earlier

- Patients may not be receiving any other investigational agents

- History of allergic reactions attributed to compounds of similar chemical or biologic
composition to imiquimod

- Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements.

Type of Study:


Study Design:

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

Outcome Measure:

To determine the effect of topical imiquimod 5% cream on tumor volume of cutaneous neurofibromas in adult subjects with neurofibromatosis 1

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Scott R. Plotkin, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Institutional Review Board

Study ID:




Start Date:

March 2009

Completion Date:

December 2013

Related Keywords:

  • Neurofibromatosis Type 1
  • Cutaneous Neurofibromas
  • imiquimod 5% cream
  • Neurofibroma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica



Massachusetts General HospitalBoston, Massachusetts  02114-2617