Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)
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| ClinicalTrials.gov Identifier: NCT01526356 |
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Recruitment Status :
Completed
First Posted : February 3, 2012
Last Update Posted : June 11, 2015
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Sponsor:
The University of Texas Health Science Center, Houston
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston
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Brief Summary:
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.
The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Angiofibromas Tuberous Sclerosis | Drug: Placebo Drug: Rapamycin | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 177 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy |
| Study Start Date : | May 2012 |
| Actual Primary Completion Date : | August 2014 |
| Actual Study Completion Date : | August 2014 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Tuberous sclerosis complex
MedlinePlus related topics:
Tuberous Sclerosis
| Arm | Intervention/treatment |
|---|---|
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Placebo Comparator: Placebo
Cream only
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Drug: Placebo
Study cream is applied nightly to the affected areas on the face.
Other Name: Rapamycin |
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Active Comparator: 0.1 % Rapamycin
0.1% Rapamycin cream
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Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose |
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Active Comparator: 1% Rapamycin
1% Rapamycin cream
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Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose |
Primary Outcome Measures :
- Reduction in lesion size and appearance [ Time Frame: 6 months ]Photographic, visual, and measurable reduction in the size and severity of the lesions.
Secondary Outcome Measures :
- Confirm the lack of systemic uptake of topically applied rapamycin. [ Time Frame: 6 months ]Blood levels checked to confirm the lack of systemic rapamycin.
- Dermatologic sensitivity at the site of application. [ Time Frame: 6 months ]Pain, erythema, or pruritis at the application site.
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| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Subjects must be willing and able to comply with all trial requirements.
- Subject has a diagnosis of TSC and has visible facial angiofibromas.
- Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods .
Exclusion Criteria:
- Subject is currently 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 investigational drug.
- Subject has a known hypersensitivity to either the vehicle or Rapamycin.
- Subject is a pregnant or nursing female.
- 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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01526356
Locations
| United States, Alabama | |
| University of Alabama at Birmingham | |
| Birminham, Alabama, United States, 35294 | |
| United States, California | |
| UCLA Mattel Children's Hospital | |
| Los Angeles, California, United States, 90095 | |
| Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center | |
| Oakland, California, United States, 94609 | |
| United States, Maryland | |
| Kennedy Krieger Institute | |
| Baltimore, Maryland, United States, 21205 | |
| United States, Massachusetts | |
| Herscot Center for Adults and Children with TSC Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Minnesota | |
| Clinic Without Walls | |
| Saint Paul, Minnesota, United States, 55102-2697 | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Texas | |
| Texas Scottish Rite Hospital | |
| Dallas, Texas, United States, 75219 | |
| The University of Texas Medical School at Houston | |
| Houston, Texas, United States, 77030 | |
| Australia, New South Wales | |
| Sydney Children's Hospital | |
| Sydney, New South Wales, Australia | |
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Investigators
| Principal Investigator: | Mary Kay Koenig, MD | The University of Texas Medical School at Houston | |
| Principal Investigator: | Hope Northrup, MD | The University of Texas Medical School at Houston |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Mary Kay Koenig, Associate Professor, The University of Texas Health Science Center, Houston |
| ClinicalTrials.gov Identifier: | NCT01526356 History of Changes |
| Other Study ID Numbers: |
HSC-MS-11-0501 Department of Defense USAMRMC ( Other Grant/Funding Number: W81XWH-11-1-0240 ) |
| First Posted: | February 3, 2012 Key Record Dates |
| Last Update Posted: | June 11, 2015 |
| Last Verified: | June 2015 |
Keywords provided by Mary Kay Koenig, The University of Texas Health Science Center, Houston:
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Angiofibroma Tuberous Sclerosis Rapamycin Sirolimus mTOR |
Additional relevant MeSH terms:
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Tuberous Sclerosis Angiofibroma Sclerosis Pathologic Processes Hamartoma Neoplasms Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Malformations of Cortical Development, Group I Malformations of Cortical Development Nervous System Malformations Nervous System Diseases Neurocutaneous Syndromes Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Congenital Abnormalities Genetic Diseases, Inborn Neoplasms, Vascular Tissue Neoplasms by Histologic Type Sirolimus Everolimus Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |