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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00865644
• Date of registration: 17/03/2009
• Prospective Registration: No
• Primary sponsor: Massachusetts General Hospital
• Public title: Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
• Scientific title: Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
• Date of first enrolment: March 2009
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00865644
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Scott R. Plotkin, MD, PhD
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cutaneous Neurofibromas

Neurofibromatosis Type 1

Intervention(s)

Drug: Imiquimod 5% Cream

Primary Outcome(s)

To determine the effect of topical imiquimod 5% cream on tumor volume of cutaneous neurofibromas in adult subjects with neurofibromatosis 1 [Time Frame: 2 years]

Secondary Outcome(s)

To correlate the inflammatory infiltrate adjacent to treated lesions during treatment with tumor response and to determine the number of circulating Tregs in this patient population [Time Frame: 2 years]

Secondary ID(s)

08-347

Source(s) of Monetary Support

Please refer to primary and secondary sponsors