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

Primary Purpose

Neurofibromatosis Type 1, Cutaneous Neurofibromas

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Imiquimod 5% Cream

About this trial

This is an interventional treatment trial for Neurofibromatosis Type 1 focused on measuring imiquimod 5% cream

Eligibility Criteria

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

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.

Sites / Locations

Massachusetts General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Imiquimod

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

NCT ID

NCT00865644

First Posted

March 17, 2009

Last Updated

January 9, 2017

Sponsor

Massachusetts General Hospital

1. Study Identification

Unique Protocol Identification Number

NCT00865644

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

March 2009 (undefined)

Primary Completion Date

April 2010 (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 study is to determine if imiquimod cream can reverse the growth of neurofibromas. Imiquimod is a skin cream that works by stimulating the body's immune system to respond to tumors. Imiquimod cream is approved for use in patients with various skin lesions, including actinic keratosis, superficial basal cell carcinoma, and external genital warts. Information from these studies, as well as previous laboratory studies, suggest that imiquimod cream may help shrink neurofibromas or keep them from growing.

Detailed Description

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.

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

imiquimod 5% cream

7. Study Design

Primary Purpose

Treatment

Study Phase

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

Imiquimod

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Imiquimod 5% Cream

Intervention Description

Applied topically to three tumors 5 times per week for a full 6 weeks

Primary Outcome Measure Information:

Title

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 Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

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

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