Facial AngiofibromaPhase 1NCT01853423
Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream
A Phase 1 study testing Rapamune in people with Facial Angiofibroma, run by Le Bonheur Children's Hospital at 1 site in United States.
Trial summary
The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment. Read more about this study
- Who is it for?
- Ages 3 to 45 years
- Conditions the study is for
- Facial Angiofibroma
- What is being tested?
- Rapamune
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2013to Jul 2016
- 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 3 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient or his / her legally authorize representative (LAR) must sign and date the approved informed consent prior to study participation or initiation of study procedures. If appropriate, the patient will give written or verbal consent. Surrogate consent will be obtained utilizing the "legally authorized representative (LAR)". The LAR must be an adult who has exhibited special care and concern for the subject, who is familiar with the subject's personal values, who is reasonably available, and who is willing to serve. No person who is identified as a protective order or other court order that directs that person to avoid contact with the subject shall be eligible to serve as the subject's LAR. Identification of LAR should normally be made using the following order of descending preference: Conservator, guardian, attorney in fact, subject's spouse (unless legally separated), the subject's adult child, the subject's parent, the subject's adult sibling, any other adult relative of the subject, or other adult who is familiar with the patient's personal values, who is reasonably available, and who is willing to serve.
- Patient must have a diagnosis of Tuberous Sclerosis Complex.
- Female or male patients over the age of 3 years.
- Female subjects of child bearing potential must not be pregnant and must undergo a pregnancy test, and must agree to use appropriate contraceptive methods
Who cannot join
- Patients will be excluded from entry into the study if any of the following are true:
- Patient has a history of drug allergy to rapamune.
- Patient is pregnant or lactating.
- Subject is receiving therapy with Rapamycin.
- Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction.
- Subject is currently participating in or has participated within the Last 30 days in a clinical trial involving an investigative drug.
- Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
- Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.
Trial locations and contacts
1 site in 1 country.
United States1 site
LeBonheur Children's Hospital
Memphis, Tennessee, 38103
About this study
The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment.
Read the detailed description
This is an open label, prospective single center study. Patients that are seen in our regional Tuberous Sclerosis Complex Clinic and have facial angiofibromas that would benefit from this cream and who wish to be involved in this study would be enrolled. We anticipate enrolling 12-15 patients the first year. Patients will have pictures of their facial angiofibromas taken immediately prior to entry into the study. They will then be prescribed 0.1% rapamune topical formulation to apply twice daily to their skin for the first two weeks and then once daily. If level is above 0, will re-check serum lipids, cbc, and repeat levels every three-four months and monitor the patient for symptoms. Patients will return at one month and three months to obtain serum rapamune levels along with pictures, then they will be seen intermittently throughout the first year with photographs of their face taken at the one month visit, 3 month visit, six month visit and twelve month visit. We will ask the parents about any side effects from the medication during the entire time patients are using the topical rapamune. We will monitor the facial lesions as well. We will assess for skin sensitivity, pain, erythema, or pruritus. If skin irritation results from the drug, then we will stop use of the drug. Parents will sign an informed consent allowing their child's picture to be taken and allowing us to summarize the data for publication at the end of this study.
This study will be the first study using a consistent formulation to make a 0.1% rapamune cream, and this will be performed under the direction of myself and Dr. Almoazen in the University of Tennessee Health Science Center(UTHSC) College of Pharmacy compounding laboratory. Only patients who are thought to be reliable for the follow up visits and to use the cream appropriately will be enrolled in the study. This study meets the FDA criteria for an Investigational New Drug(IND) exemption.
The adverse events noted in the package insert for rapamune cream are all based on systemic administration of the medication, primarily to adults with other diseases. In our clinical experience, with topical use there is no absorption and we have not noted any side effects.
- Conditions
- Facial Angiofibroma
- Being tested
- Rapamune
- Keywords
- Rapamune, Topical, Pediatric, Safety, Efficiency, Angiofibroma, Skin Cream
- Medical subject headings
- Tuberous Sclerosis, Angiofibroma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamuneDrug
Also called Sirolimus
Small amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily.
| Study group | Role | What they receive |
|---|---|---|
| Rapamune | Experimental | Small amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily. Receives: Drug: Rapamune |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of facial angiofibroma with use of rapamune facial skin cream
- Tolerability: Number of participants with adverse events
- Efficacy: Skin assessment to measure improvement of facial angiofibromas
- Safety: Rapamune levels (serum)
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2013 (estimated)
- Study startOct 2013
- Primary completionMay 2016
- Study completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facial AngiofibromaStudies of the same condition, open and under way.
- 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
- Le Bonheur Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer, University of Tennessee
- Responsible party
- Principal investigator, James W. Wheless, Le Bonheur Children's Hospital
- Investigators
- James W Wheless, MD, Principal investigator, LeBonheur Children's Hospital- Neuroscience Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01853423
- Sponsor's protocol ID
- UTHSC TSC01
- Other IDs
- 12-2008
- Status last verified
- Aug 2016
- First submitted
- 12 Apr 2013
- Last updated
- 19 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream
- ClinicalTrials.gov ID
- NCT01853423
- Acronym / sponsor ID
- UTHSC TSC01
- Conditions
- Facial Angiofibroma
- Keywords
- Rapamune, Topical, Pediatric, Safety, Efficiency, Angiofibroma, Skin Cream
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Le Bonheur Children's HospitalUniversity, hospital or other
- Collaborators
- Pfizer, University of Tennessee
- Study start
- Oct 2013
- Primary completion
- May 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 15 May 2013 (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 objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment.
Detailed description
This is an open label, prospective single center study. Patients that are seen in our regional Tuberous Sclerosis Complex Clinic and have facial angiofibromas that would benefit from this cream and who wish to be involved in this study would be enrolled. We anticipate enrolling 12-15 patients the first year. Patients will have pictures of their facial angiofibromas taken immediately prior to entry into the study. They will then be prescribed 0.1% rapamune topical formulation to apply twice daily to their skin for the first two weeks and then once daily. If level is above 0, will re-check serum lipids, cbc, and repeat levels every three-four months and monitor the patient for symptoms. Patients will return at one month and three months to obtain serum rapamune levels along with pictures, then they will be seen intermittently throughout the first year with photographs of their face taken at the one month visit, 3 month visit, six month visit and twelve month visit. We will ask the parents about any side effects from the medication during the entire time patients are using the topical rapamune. We will monitor the facial lesions as well. We will assess for skin sensitivity, pain, erythema, or pruritus. If skin irritation results from the drug, then we will stop use of the drug. Parents will sign an informed consent allowing their child's picture to be taken and allowing us to summarize the data for publication at the end of this study.
This study will be the first study using a consistent formulation to make a 0.1% rapamune cream, and this will be performed under the direction of myself and Dr. Almoazen in the University of Tennessee Health Science Center(UTHSC) College of Pharmacy compounding laboratory. Only patients who are thought to be reliable for the follow up visits and to use the cream appropriately will be enrolled in the study. This study meets the FDA criteria for an Investigational New Drug(IND) exemption.
The adverse events noted in the package insert for rapamune cream are all based on systemic administration of the medication, primarily to adults with other diseases. In our clinical experience, with topical use there is no absorption and we have not noted any side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rapamune | Experimental | Drug: RapamuneSmall amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily. |
Who may qualify
- Ages
- 3 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patient or his / her legally authorize representative (LAR) must sign and date the approved informed consent prior to study participation or initiation of study procedures. If appropriate, the patient will give written or verbal consent. Surrogate consent will be obtained utilizing the "legally authorized representative (LAR)". The LAR must be an adult who has exhibited special care and concern for the subject, who is familiar with the subject's personal values, who is reasonably available, and who is willing to serve. No person who is identified as a protective order or other court order that directs that person to avoid contact with the subject shall be eligible to serve as the subject's LAR. Identification of LAR should normally be made using the following order of descending preference: Conservator, guardian, attorney in fact, subject's spouse (unless legally separated), the subject's adult child, the subject's parent, the subject's adult sibling, any other adult relative of the subject, or other adult who is familiar with the patient's personal values, who is reasonably available, and who is willing to serve.
Key exclusion criteria (8 listed)
- Patients will be excluded from entry into the study if any of the following are true:
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 |
|---|---|---|
| RapamuneAlso: SirolimusSmall amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily. | Drug | Rapamune |
Primary outcome
Reduction of facial angiofibroma with use of rapamune facial skin cream
Time frame: 1 year
1. Tolerability: Number of participants with adverse events 2. Efficacy: Skin assessment to measure improvement of facial angiofibromas 3. Safety: Rapamune levels (serum)
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 19 Aug 2016.
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.
James W Wheless, MD principal investigator
LeBonheur Children's Hospital- Neuroscience Institute
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 Rapamune in people with Facial Angiofibroma, run by Le Bonheur Children's 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 4 inclusion and 8 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 19 Aug 2016. 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?
Reduction of facial angiofibroma with use of rapamune facial skin cream (time frame: 1 year). 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 19 Aug 2016.