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Status: Ongoing First registered on: 01/04/2016

Last updated on: 20/04/2022

1. Study identification

EU PAS Register NumberEUPAS13001

Official titleA 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)

Study title acronymSCENESSE® PASS-001

Study typeOther: non-interventional study

Brief description of the studyThis non-interventional study has been designed to gather long-term safety data and outcome endpoints. The objectives 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®

Was this study requested by a regulator?Yes: EMA

Is the study required by a Risk Management Plan (RMP)? EU RMP category 2 (specific obligation of marketing authorisation)

Regulatory procedure number (RMP Category 1 and 2 studies only)

Other study registration identification numbers and URLs as applicable

2. Research centres and Investigator details

Coordinating study entity

Centre nameErasmus Medical Centre

Centre locationRotterdam

Details of (Primary) lead investigator

Title Dr

Last name Bilbao

First name Pilar

Is this study being carried out with the collaboration of a research network?

Yes

Porphryia treatment centres

Other centres where this study is being conducted

Multiple centres

In total how many centres are involved in this Study?10

Countries in which this study is being conducted

International study

Austria

Finland

Italy

Netherlands

Slovenia

United Kingdom

3. Study timelines: initial administrative steps, progress reports and final report

PlannedActual

Date when funding contract was signed30/04/201619/06/2016

Start date of data collection31/05/201622/06/2016

Start date of data analysis

Date of interim report, if expected

Date of final study report01/04/2027

4. Sources of funding

Please provide estimates of the percentage of funding by source for this study

Names(s)Approximate % funding

Pharmaceutical companiesClinuvel100

Charities

Government body

Research councils

EU funding scheme

5. Contact details for enquiries

Scientific Enquiries

Title Dr

Last name Bilbao

First name Pilar

Address line 1Wesley House

Address line 2Bull Hill

Address line 3

CityLeatherhead

PostcodeKT22 7AH

CountryUnited Kingdom

Phone number (incl. country code)441372860765

Alternative phone number

Fax number (incl. country code)

Email address mail@clinuvel.com

Public Enquiries

Title Dr

Last name Bilbao

First name Pilar

Address line 1Wesley House

Address line 2Bull Hill

Address line 3

CityLeatherhead

PostcodeKT22 7AH

CountryUnited Kingdom

Phone number (incl. country code)441372860765

Alternative phone number

Fax number (incl. country code)

Email address mail@clinuvel.com

11. Scope of the study

What is the scope of the study?

The study has been designed to assess whether SCENESSE® can be used safely within designated treatment centres and to generate data to support the clinical effectiveness of SCENESSE®.

Primary scope : The study has been designed to assess whether SCENESSE® can be used safely within designated treatment centres and to generate data to support the clinical effectiveness of SCENESSE®.

12. Main objective(s)

What is the main objective of the study?

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

Are there primary outcomes?Yes

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

Are there secondary outcomes?Yes

• Evaluate adherence with the controlled distribution program • Generate data to contribute to knowledge about clinical benefits and to add data on potential clinical effectiveness of SCENESSE

13. Study design

What is the design of the study?

non-interventional study

14. Follow-up of patients

Will patients be followed up?Not applicable/no follow-up

15. Data analysis plan

Please provide a brief summary of the analysis method

All patients enrolled in the registry will be included in the safety assessment. In general longitudinal comparisons will be within Treated Groups (including separate groups that take into account patients who commence or discontinue treatment) and between groups, Treated versus Untreated. The number of participants with treatment-emergent adverse events will be summarized by MedDRA PT and body system. Adverse events will be further summarized by intensity, seriousness and outcome. Adverse events will also be summarized by 6 monthly time intervals from entry into the registry to assess the longitudinal effect of the treatment and EPP. Assessment of Clinical Effectiveness Continuity on treatment - together with logs detailing reasons for discontinuing (and those recommencing) Quality of Life (EPP-QoL) – will be measured using the EPP-QoL questionnaire, provided to the patient for completion at baseline prior to treatment in Year 1 and at each subsequent visit.