SCENESSE® PASS-001 (Afamelanotide 16mg in Patients with EPP)
Research type
Research Study
Full title
A Post-Authorisation Disease Registry Safety Study to Generate Data on the Long-Term Safety and Clinical Effectiveness of SCENESSE® (Afamelanotide 16mg) in Patients with Erythropoietic Protoporphyria (EPP).
IRAS ID
287960
Contact name
Robert S. Dawe
Contact email
Sponsor organisation
CLINUVEL UK LTD
Clinicaltrials.gov Identifier
EUPAS13001, EU PAS Register Number
Duration of Study in the UK
9 years, 9 months, 27 days
Research summary
Erythropoietic protoporphyria (EPP) is an orphan indication. Other than SCENESSE® (afamelanotide 16mg) there are no medicinal products in the Community available for the therapeutic indication; there is currently a clear unmet medical need for treatment. In December 2014, the European Commission granted marketing authorisation for SCENESSE® under exceptional circumstances (Article 14(8) of Regulation (EC) No 726/2004). The establishment of a Disease Registry was imposed as a specific obligation with data to be collected from both adult EPP patients and physicians. The objectives of this non-interventional post-authorisation study are to assess whether SCENESSE® can be used safely within designated treatment centres and to generate data to support the clinical effectiveness derived from the use of SCENESSE®. Patients with a positive diagnosis for EPP will be eligible for study entry. The treated group will include those who are treated with SCENESSE® (aged 18 years or more and where treatment is not contraindicated in accordance with the approved SmPC). Those participating in the registry study but electing not to receive SCENESSE® or where treatment with SCENESSE® is contraindicated will act as controls. Clinical effectiveness of SCENESSE® will be assessed at regular intervals by evaluating:
- Longitudinal assessment of those remaining on treatment and the reasons for discontinuing (or recommencing) treatment
- Longitudinal assessment of activities able to be undertaken using the Daily Activity Inventory
- Quality of Life assessment using the EPP-QoL questionnaire
- Sun protection measures used and the number and severity of phototoxic reactions experienced
End of data collection will be determined by PRAC/CHMP.REC name
North of Scotland Research Ethics Committee 2
REC reference
21/NS/0013
Date of REC Opinion
18 Feb 2021
REC opinion
Further Information Favourable Opinion