Facial tumors (angiofibromas) associated with tuberous sclerosis complex.
Conditions
Interventions
Trade Name: Rapamune 1 mg/ml solución oral
Pharmaceutical Form: Oral solution
INN or Proposed INN: RAPAMUNE
Current Sponsor code: SIROLIMUS
Concentration unit: mg/ml milligram(s)/millilitre
Concentrat
Sponsors
Fundación Investigación Hospital Ramón y Cajal
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Patients, who, after receiving information about the design, the purpose of the study, the potential risks it can occur and that at any time may refuse your help, give written consent to participate in the study and for the provision material for cellular and molecular studies. -Subject is male or female and over 13 years of age. -Subject has a diagnosis of TSC and has visible fibromatosus lesions (angiofibromas or angifibromata) -women of childbearing age should get a negative pregnancy test the screening visit; further agree to use adequate contraception (among them are considered: double barrier as condom + diaphragm, surgical sterilization) within 14 days following administration of study drug. Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Pregnant, intention to become pregnant during treatment phase of the trial, or breastfeeding. 2. Patient is currently participating or has participated within the last two months in any clinical trial. 3.Patient is actually receiving or has received in the last three mounths any m-TOR inhibitor treatment or any form of immunosuppression drug. 4. Hypersensitivity to rapamycin 5.Any serious medical or psychiatric illness currently ongoing, or experienced within the past three months, that in the opinion of the investigator would compromise the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the clinical effectiveness of a topical treatment with rapamycin solution 1mg/ml for the treatment of facial angiofibromas associated to TSC.;Secondary Objective: To evaluate the safety of the treatment treatment with rapamycin solution 1mg/ml for the treatment of facial angiofibromas associated to TSC. To evaluate treatment efficiency through the characterization of tumor recurrence timing.;Primary end point(s): The proportion of participants with clinical evidence of "success" (defined as reduction of at least 50% in size and number of tumors and a decrease in the puntuaction of the FASI index of at least 40% at the end of the treatmen period (three months). Success includes parcial and total responses.;Timepoint(s) of evaluation of this end point: 1ªweek month1 month2 month 4 month 7 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1.Proportion of patients achieving a total response at the end of the treatment period (3 months) 2.Proportion of patients achieving a total response at the first month of the study. 3.Proportion of patients achieving a complete or a parcial response after one week of treatment. 4.Proportion of patients achieving a total response at the first week of the study. 5.Proportion of patients achieving a complete or a parcial response after one month of treatment. 6.Time of relapse Safety secondary endpoint 1.Proportion of patients who suffer any AE 2.Proportion of local reactions 3.Proportion of patiens whose serum rapamycin level is detectable 4.Proportion of patients whose laboratory tests became abnormal when at screening were not. 5.Proportion of patients whose blood preasure or whose respiratory/ heart rate became abnormal;Timepoint(s) of evaluation of this end point: 1ªweek month1 month2 month 4 month 7 | — |
Countries
Spain
Contacts
Public ContactFundación Hospital Ramón y Cajal
Fundación Investigación Hospital Ramón y Cajal
Outcome results
None listed