Erythropoietic Protoporphyria
Conditions
Brief summary
The purpose of this study is to evaluate the concentration of afamelanotide in serum after the administration of afamelanotide in adolescent and adult EPP patients.
Interventions
DRUGAfamelanotide 16mg implant
afamelanotide 16mg implant will be administered to each patient during the study.
Sponsors
Clinuvel Europe Limited
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* EPP patients aged between 12 and 70 years * BMI between 15 and 30 kg/m2 * \>50 kg
Exclusion criteria
* Any personal or direct family history of melanoma * Any significant history of allergy and/or sensitivity to any of the contents of study drug product or lignocaine or other local anaesthetics if used * Any significant illness during the four weeks before the study screening period * Any evidence of hepatic or renal impairment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cmax (Maximum Plasma Concentration) | Baseline to Day 7 | Blood samples will be collected for analysis of afamelanotide plasma concentrations and understand what the maximum concentration in plasma is. |
| AUC(0-t) (Area Under the Curve From Administration to Last Observed Concentration at Time t) | Baseline to Day 7 | Blood samples will be collected for analysis of afamelanotide plasma concentrations. |
Secondary
| Measure | Time frame |
|---|---|
| AUC(0-∞) (Area Under the Curve Extrapolated to Infinite Time) | Baseline to Day 7 |
Countries
Belgium, Netherlands
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 14 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 14 Participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 28 |
| other Total, other adverse events | 23 / 28 |
| serious Total, serious adverse events | 0 / 28 |
Outcome results
None listed