Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

The University of Texas System (UT)

Status and phase: Completed; Phase 2

Conditions: Angiofibromas; Tuberous Sclerosis

Treatments: Drug: Placebo; Drug: Rapamycin

Study type: Interventional

Funder types: Other

Identifiers

NCT01526356

HSC-MS-11-0501

Department of Defense USAMRMC (Other Grant/Funding Number)

Details and patient eligibility

About

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.

Enrollment: 179 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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179 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Cream only

Treatment:

Drug: Placebo

0.1 % Rapamycin

Active Comparator group

Description:

0.1% Rapamycin cream

Treatment:

Drug: Rapamycin

Drug: Rapamycin

1% Rapamycin

Active Comparator group

Description:

1% Rapamycin cream

Treatment:

Drug: Rapamycin

Drug: Rapamycin