Study type

Study type

Clinical trial
Study drug and medical condition

Name of medicine

SCENESSE

Medical condition to be studied

Porphyria
Population studied

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)

Estimated number of subjects

200
Study design details

Main study objective

• Gather long-term safety data of SCENESSE®• Evaluate compliance with the risk minimization measures • Evaluate compliance with the controlled distribution program

Data analysis plan

The MAH deemed the sample size to be too small to arrive at a meaningful statistical comparisons. Consequently only descriptive statistics will be used in the analyses.Safety Data Analyses:-Changes in cutaneous efflorescence -Application site reactions -Allergy and hypersensitivity -Off-label use (in children or adults without EPP) -Use in pregnancy or lactation -Administration errors Assessment of Clinical Effectiveness:-Quality of Life (EPP-QoL) -Impact on Daily Activity-Continuity on treatment Risk minimisation measures:-Changes in cutaneous efflorescence-Application site reactions -Allergy and hypersensitivity-Off-label use (in children or adults without EPP) -Use in pregnancy or lactation-Administration error