NCT02635789.
| Trial name or title | Phase III study of topical formulation of sirolimus to skin lesions in people with TSC. |
| Methods | Double‐blind, randomised, placebo‐controlled phase III study. |
| Participants | People with TSC with angiofibroma, hypomelanotic macules, plaque. |
| Interventions | Participants are randomized into either: 1. placebo gel OR 2. NPC‐12G gel containing 0.2% sirolimus administered topically twice‐a‐day for 12 weeks. |
| Outcomes | Primary outcome: improvements in angiofibroma after 12 weeks (improvements comparing with baseline as assessed using photograph by the central photo‐judgement committee). Secondary outcomes: improvements in angiofibroma after 4, 8 and 16 weeks (improvements comparing with baseline is assessed using photograph by the central photo‐judgement committee); improvements in angiofibroma after 4, 8, 12 and 16 weeks (improvements comparing with baseline as assessed by the investigator); improvements in redness of angiofibroma (improvement comparing with baseline is assessed by the central photo‐judgement committee and the investigator); improvements in hypomelanotic macule and plaque of upper neck (improvement comparing with baseline is assessed by the central photo‐judgement committee and the investigator); the rate of patients who are evaluated as ''improvement'' or more (improvement rate) in primary outcome measure and in secondary outcome measures above outcome 1 to 5; change in total score from baseline for DLQI and CDLQI (DLQI for subjects 16 years old and greater, or CDLQI for children of less than 16 years old as assessed by participants); AEs during the study period; SAEs during the study period; laboratory findings during the study period; vital signs during the study period; sirolimus blood concentration as assessed by drug monitoring. |
| Starting date | December 2015. |
| Contact information | Mr Taihei Hio (Tel.: +81‐3‐5651‐1177, email: hio@nobelpharma.co.jp) and Mr. Kenji Shimizu (Tel.: +81‐3‐5651‐1177, email: shimizu@nobelpharma.co.jp). |
| Notes | Sponsored by Nobelpharma. |
AE: adverse event AEDs: anti‐epileptic drugs IQ: intelligence quotient QoL: quality of life SAEs: serious adverse events TSC: tuberous sclerosis complex