NCT02634931

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

A Long-term, Single-arm, Open-label Trial of NPC-12G (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: 94 (actual)
• Sponsor: Nobelpharma · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label trial.

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 a TSC-specific facial skin lesion, 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 and surgical treatments, but there are problems as many relapses, deficiency of evidence, change of pigment, scar and risk of infection. This is a multicenter and open-label trial. The trial consists of two phase. In the first trial phase for 52 weeks, the efficacy as well as the safety is evaluated. For the second trial phase the trial will be continued until the date of approval of NDA for NPC-12G. The safety is evaluated during the second trial phase, but not the efficacy. Patients who meet all entry criteria for the trial apply 0.2% NPC-12G gel twice a day. Patients will visit at 4 to 5-week intervals for the first 6 months of the first trial phase, and then 3 months intervals thereafter.

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 52 weeks or longer

Timeline

• Start date: 2015-12-01
• Primary completion: 2018-10-01
• Completion: 2018-10-01
• First posted: 2015-12-18
• Last updated: 2019-02-20

Locations

1 site across 1 country: Japan