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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Scott Randall Plotkin, MD, PhD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT00865644
First received: March 17, 2009
Last updated: March 4, 2013
Last verified: March 2013
  Purpose

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.


Condition Intervention
Neurofibromatosis Type 1
Cutaneous Neurofibromas
Drug: Imiquimod 5% Cream

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

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 [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Enrollment: 11
Study Start Date: March 2009
Estimated Study Completion Date: December 2013
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Imiquimod Drug: Imiquimod 5% Cream
Applied topically to three tumors 5 times per week for a full 6 weeks

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.
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00865644

Locations
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
Investigators
Principal Investigator: Scott R. Plotkin, MD, PhD Massachusetts General Hospital
  More Information

No publications provided

Responsible Party: Scott Randall Plotkin, MD, PhD, Associate Professor, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00865644     History of Changes
Other Study ID Numbers: 08-347
Study First Received: March 17, 2009
Last Updated: March 4, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Massachusetts General Hospital:
imiquimod 5% cream

Additional relevant MeSH terms:
Neurofibroma
Neurofibromatoses
Neurofibromatosis 1
Osteitis Fibrosa Cystica
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Bone Diseases, Endocrine
Bone Diseases
Musculoskeletal Diseases
Imiquimod
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Interferon Inducers

ClinicalTrials.gov processed this record on April 16, 2014