NCT02635789

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

A Double-blind, Randomized, Placebo-controlled Phase III Trial to Investigate the Efficacy and Safety of NPC-12G Gel (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Nobelpharma · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)

Detailed description

Tuberous Sclerosis Complex (TSC) is an autosomal dominant hereditary disease that causes benign tumors on the almost whole body (including skin, brain, kidney, lung and heart), behavior disorder as autism, mental retardation and neurologic symptom as epilepsy. Angiofibroma is TSC-specific facial skin lesions, and hamartoma caused by increase of the component of skin connective tissues and blood vessels. Other skin lesions due to TSC are white macule(hypomelanotic macule), plaque, shagreen patch and ungual fibromas. Current therapeutic methods for angiofibroma are laser or surgical treatments, but there are problems as many relapses, deficiency of evidence, change of pigment, scar and risk of infection. This will be a multicenter, double-blind, randomized, placebo-controlled parallel group trial. The trial has three phases; the screening phase, double-blinded treatment phase, and post-treatment phase. The screening phase comprises a screening visit where subject's initial eligibility will be evaluated. During double-blinded treatment phase, patients who meet all entry criteria for the trial will be randomized into two groups, and they will apply 0.2% NPC-12G gel or placebo gel topically twice a day for 12 weeks.

Conditions

• Tuberous Sclerosis
• Angiofibroma
• Hypomelanotic Macule
• Plaque

Interventions

Type	Name	Description
DRUG	NPC-12G gel	NPC-12G gel is administered topically twice a day for 12 weeks
DRUG	Placebo gel	NPC-12G gel placebo is administered topically twice a day for 12 weeks

Timeline

• Start date: 2015-12-01
• Primary completion: 2016-10-01
• Completion: 2016-10-01
• First posted: 2015-12-21
• Last updated: 2017-02-02

Locations

1 site across 1 country: Japan