Neurofibromatosis Type 1Phase 1NCT00865644
Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
A Phase 1 study testing Imiquimod 5% Cream in people with Neurofibromatosis Type 1 and Cutaneous Neurofibromas, run by Massachusetts General Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- What is being tested?
- Imiquimod 5% Cream
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2009to Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 15Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
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.
Read the 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.
- Conditions
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- Being tested
- Imiquimod 5% Cream
- Keywords
- imiquimod 5% cream
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imiquimod 5% CreamDrug
Applied topically to three tumors 5 times per week for a full 6 weeks
| Study group | Role | What they receive |
|---|---|---|
| Imiquimod | Experimental | Not described Receives: Drug: Imiquimod 5% Cream |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the effect of topical imiquimod 5% cream on tumor volume of cutaneous neurofibromas in adult subjects with neurofibromatosis 1
Measured: 2 years
Secondary outcomes (1)
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
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2009 (estimated)
- Study startMar 2009
- Primary completionApr 2010
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Scott Randall Plotkin, MD, PhD, Massachusetts General Hospital
- Investigators
- Scott R. Plotkin, MD, PhD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00865644
- Sponsor's protocol ID
- 08-347
- Status last verified
- Jan 2017
- First submitted
- 17 Mar 2009
- Last updated
- 10 Jan 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
- ClinicalTrials.gov ID
- NCT00865644
- Acronym / sponsor ID
- 08-347
- Conditions
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- Keywords
- imiquimod 5% cream
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- Mar 2009
- Primary completion
- Apr 2010 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 19 Mar 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Imiquimod | Experimental | Drug: Imiquimod 5% Cream |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Patients must have a diagnosis of NF1 based on NIH criterial with two or more of the following characteristics:
- Participants must have at least four cutaneous neurofibromas on skin exam with the following qualities:
- No treatment with an investigation agent for cutaneous neurofibromas within the last 3 months
- 18 years of age or older
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imiquimod 5% CreamApplied topically to three tumors 5 times per week for a full 6 weeks | Drug | Imiquimod |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Scott R. Plotkin, MD, PhD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Imiquimod 5% Cream in people with Neurofibromatosis Type 1 and Cutaneous Neurofibromas, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2017.