Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Facies

Tuberous Sclerosis

Angiofibroma

Disease Attributes

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Neoplasms, Vascular Tissue

Neoplasms by Histologic Type

Tuberous Sclerosis Complex

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Sirolimus

Temsirolimus

MTOR Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Immunosuppressive Agents

Immunologic Factors

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

Placebo cream topical, applied once daily before bed on affected area for 26 weeks

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

rapamycin

placebo

Ioana Stanescu

The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).

Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to be well tolerated.

The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over

Individual Participant Data is not planned to be shared with other researchers

Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4:

0=Clear

1. Almost Clear
2. Mild
3. Moderate
4. Severe

The time elapsed from the first dose to the time of treatment success, according to the Investigator's Global Assessment (IGA) scale. The total time of treatment was 26 weeks, although Covid-19 visit delays led to an extension of up to 2 weeks (28 weeks total) for some patients. Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4:

0=Clear

1. Almost Clear
2. Mild
3. Moderate
4. Severe

The change in grading on the Investigator's Global Assessment (IGA) scale from baseline.

IGA scores range from 0-4:

0=Clear

1. Almost Clear
2. Mild
3. Moderate
4. Severe

The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline. FASI grades lesions according to their erythema, size and extent by summing the scores of each category. The final FASI scores range from (mild) 2-9 (severe).

Erythema Skin color 0 Light Red 1 Red 2 Dark Red/purple 3 Size None 0 Small (\< 5mm) 1 Large (\> 5mm) 2 Confluent 3 Extension \<50 % cheek surface 2 \>50% cheek surface 3

Percentage change in facial angiofibroma since beginning treatment, as assessed by the participant or parent/caregiver. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where the participant or parent/caregiver estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.

Percentage improvement in facial angiofibroma since beginning treatment, as assessed by the clinician. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where clinicians estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.

Change in facial angiofibroma since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver. This was a single assessment time-point, where the participant or parent/caregiver evaluated the change in the facial angiofibroma lesion appearance from their perspective since baseline.

Dermatology Specialties Limited Partnership

Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

rapamycin: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

0.5% Rapamycin Cream, Topical

Rapamycin cream topical, 1.0% w/w, applied once daily before bed on affected area for 26 weeks

rapamycin: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

1.0% Rapamycin Cream, Topical

Placebo cream topical, applied once daily before bed on affected area for 26 weeks

placebo: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Placebo

Total

Age, Continuous

Age group

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Study Site

Height

Weight

Rapamycin concentration

The Investigator's Global Assessment (IGA) measures the severity of facial angiofibroma, with scores between 0 (clear) and 4 (severe).

0 = Clear

1. = Almost Clear
2. = Mild
3. = Moderate
4. = Severe

Investigator's Global Assessment (IGA)

Facial Angiofibroma Severity Index (FASI) grades lesions according to their erythema, size and extent. Scores range between 2 and 9. Scores are summed from the following measures:

Erythema Skin color 0 Light Red 1 Red 2 Dark Red/purple 3 Size None 0 Small (\< 5mm) 1 Large (\> 5mm) 2 Confluent 3 Extension \<50 % cheek surface 2 \>50% cheek surface 3

Facial Angiofibroma Severity Index (FASI)

Covid-19 pandemic halted recruitment, leading to a smaller number of patients recruited than intended (107/120).

Spots Global Cancer Trial Database for Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

Useful links and downloads for this trial