Tuberous SclerosisPhase 1NCT01031901

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

A Phase 1 study testing Skincerity, Skincerity plus sirolimus/rapamycin and Skinercity plus sirolimus/rapamycin in people with Tuberous Sclerosis and Neurofibromatoses, run by The University of Texas Health Science Center, Houston at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 13 years and olderHealthy volunteers can take part
Conditions the study is for: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma
What is being tested?: Skincerity, Skincerity plus sirolimus/rapamycin and Skinercity plus sirolimus/rapamycin
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 52 participants
Where: 1 siteUnited States
Study dates: Dec 2009to Jun 2011
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 13 years and older
All sexes
Healthy volunteers accepted
Child, Adult, Older adult

Who may be able to join

Subject is willing and able to comply with all trial requirements
Subject is male or female and over 13 years of age
Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial

Who cannot join

Subject is currently receiving therapy with rapamycin or sirolimus
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 any clinical trial involving an investigational drug
Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
Subject is a pregnant or nursing female

Trial locations and contacts

1 site in 1 country.

United States1 site

The University of Texas Health Science Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months.

The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.

Conditions: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Being tested: Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
Keywords: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus
Medical subject headings: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SkincerityDrug
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
Skincerity plus sirolimus/rapamycinDrug
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
Skinercity plus sirolimus/rapamycinDrug
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas

Study groups in NCT01031901
Study group	Role	What they receive
TSC Placebo Arm	Placebo comparator	TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas Receives: Drug: Skincerity
TSC 1% Arm	Experimental	TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas Receives: Drug: Skincerity plus sirolimus/rapamycin
TSC 5% Arm	Experimental	TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas Receives: Drug: Skinercity plus sirolimus/rapamycin
NF1 Placebo Arm	Placebo comparator	NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas Receives: Drug: Skincerity
NF1 1% Arm	Experimental	NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas Receives: Drug: Skincerity plus sirolimus/rapamycin
NF1 5% Arm	Experimental	NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas Receives: Drug: Skinercity plus sirolimus/rapamycin

What is being measured

The primary outcome is the main question the study is designed to answer.

Rapamycin level
Measured: 6 months
Complete blood count
Measured: 6 months
Total cholesterol
Measured: 6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
Measured: 6 months

Secondary outcomes (1)

Reduction in lesion size and appearance
Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2009Jun 2011

First posted15 Dec 2009 (estimated)
Study startDec 2009
Primary completionFeb 2011
Study completionJun 2011

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Tuberous SclerosisStudies of the same condition, open and under way.
More trials run by The University of Texas Health Science Center, HoustonThe 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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 52 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01031901
Sponsor's protocol ID: HSC-MS-09-0259
Status last verified: Feb 2012
First submitted: 10 Dec 2009
Last updated: 27 Feb 2012
Results posted: No
Data monitoring committee: No

Publications

Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.
PubMed 22934754

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Study of the Disease Process of Lymphangioleiomyomatosis
Lung Disease, Pneumothorax
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingObservational study
Study of Skin Tumors in Tuberous Sclerosis
Tuberous Sclerosis
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex

All Tuberous Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 27 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1
ClinicalTrials.gov ID: NCT01031901
Acronym / sponsor ID: HSC-MS-09-0259
Conditions: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Keywords: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 52 participants (actual)
Lead sponsor: The University of Texas Health Science Center, HoustonUniversity, hospital or other
Collaborators: Society for Pediatric Dermatology
Study start: Dec 2009
Primary completion: Feb 2011 (actual)
Study completion: Jun 2011 (actual)
First posted: 15 Dec 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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 6

Arm	Type	Receives
TSC Placebo Arm	Placebo comparator	Drug: SkincerityTSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
TSC 1% Arm	Experimental	Drug: Skincerity plus sirolimus/rapamycinTSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
TSC 5% Arm	Experimental	Drug: Skinercity plus sirolimus/rapamycinTSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
NF1 Placebo Arm	Placebo comparator	Drug: SkincerityNF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
NF1 1% Arm	Experimental	Drug: Skincerity plus sirolimus/rapamycinNF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
NF1 5% Arm	Experimental	Drug: Skinercity plus sirolimus/rapamycinNF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas

Who may qualify

Ages: 13 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

Subject is willing and able to comply with all trial requirements
Subject is male or female and over 13 years of age
Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial

Key exclusion criteria (5 listed)

Subject is currently receiving therapy with rapamycin or sirolimus
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 any clinical trial involving an investigational drug
Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
Subject is a pregnant or nursing female

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
SkincerityStudy subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas	Drug	NF1 Placebo Arm, TSC Placebo Arm
Skincerity plus sirolimus/rapamycinStudy subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas	Drug	NF1 1% Arm, TSC 1% Arm
Skinercity plus sirolimus/rapamycinStudy subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas	Drug	NF1 5% Arm, TSC 5% Arm

Primary outcome

Rapamycin level
Time frame: 6 months
Complete blood count
Time frame: 6 months
Total cholesterol
Time frame: 6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
Time frame: 6 months

1 secondary outcome

Reduction in lesion size and appearance
Time frame: 6 months

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 27 Feb 2012.
Derived from this study: PubMed 22934754Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.
UT TSC Clinic HomeLink provided by the sponsor.

Related trials for Tuberous Sclerosis

RecruitingObservational study
Study of the Disease Process of Lymphangioleiomyomatosis
Lung Disease, Pneumothorax
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingObservational study
Study of Skin Tumors in Tuberous Sclerosis
Tuberous Sclerosis
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex

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.

Mary Kay Koenig, MD principal investigator

The University of Texas Health Science Center, Houston

Hope Northrup, MD principal investigator

The University of Texas Health Science Center, Houston

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 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 27 Feb 2012. 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. Some participants in this study may receive a placebo. 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?

Rapamycin level (time frame: 6 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01031901, which also lists 1 linked publication.

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 27 Feb 2012.