Topical Rapamycin to Erase Angiofibromas in TSC

Learn more about:
Related Clinical Trial
Doxycycline In Lymphangioleiomyomatosis (LAM) Effect of Fasting on the Size of Abdominal Lymphatic Tumors in Women Turmeric as Treatment in Epilepsy Study of the Disease Process of Lymphangioleiomyomatosis RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM The Effectiveness and Safety of Vagus Nerve Stimulation for TRE The Effectiveness and Safety of Resective Epilepsy Surgery for TRE Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) Safety of Simvastatin in LAM and TSC Studies of Autistic Patients: Gene Networks and Clinical Subtypes Topical Rapamycin to Erase Angiofibromas in TSC Rapalogues for Autism Phenotype in TSC: A Feasibility Study A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Everolimus for Cancer With TSC1 or TSC2 Mutation Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex The Cognitive Variability in NF1 and TSC Monozygotic Twins Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC) Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy – Tuberous Sclerosis Complex A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC) Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipoma in the Great West Region of France Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Complex Characterization of Patients With Tuberous Sclerosis Complex, Lymphangioleiomyomatosis and Angiomyolipoma Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex JASPER Early Intervention for Tuberous Sclerosis Studies in Patients With Tuberous Sclerosis Complex Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM) Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation Relationship Genetic Heterogeneity and Genotype-phenotype Correlation of Children and Adults With Tuberous Sclerosis Complex (TuScCom) Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC) Tuberous Sclerosis Complex Natural History Study: Renal Manifestations Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) Study of Skin Tumors in Tuberous Sclerosis

Brief Title

Topical Rapamycin to Erase Angiofibromas in TSC

Official Title

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

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.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score

Secondary Outcome

 Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient

Condition

Angiofibromas

Intervention

Placebo

Study Arms / Comparison Groups

 Placebo
Description:  Cream only

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

179

Start Date

May 2012

Completion Date

August 2014

Primary Completion Date

August 2014

Eligibility 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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Mary Kay Koenig, MD, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT01526356

Organization ID

HSC-MS-11-0501

Secondary IDs

Department of Defense USAMRMC

Responsible Party

Principal Investigator

Study Sponsor

The University of Texas Health Science Center, Houston


Study Sponsor

Mary Kay Koenig, MD, Principal Investigator, The University of Texas Medical School at Houston


Verification Date

September 2020