In short
Clinical trials are studying Afamelanotide in several patient groups, including early Parkinson's disease, stroke, erythropoietic protoporphyria, vitiligo, and healthy volunteers. These studies mainly look at safety, efficacy, and how the body handles the treatment. The trials include different phases, from Phase 1 to Phase 4.
Key points
- Clinical trials of Afamelanotide are being done in several groups, including people with early Parkinson's disease, acute arterial ischaemic stroke, erythropoietic protoporphyria, vitiligo, and healthy volunteers. The studies range from Phase 1 to Phase 4, showing both early research and later comparison studies. Main goals include safety, efficacy, pharmacokinetics, DNA damage changes, and repigmentation. Some trials are completed, some are authorised, and one early Parkinson's study was withdrawn. The trial data show that Afamelanotide is being tested in both patient groups and healthy volunteers for different research questions.
Clinical trial overview
The trial data show that Afamelanotide is being studied in different clinical settings, not as a general medicine description, but as a research treatment in specific trials.
Across the listed studies, the targets include Parkinson's disease, arterial ischaemic stroke, erythropoietic protoporphyria, vitiligo, and healthy volunteers.
The studies include Phase 1, Phase 2, and Phase 4 research, which means the program includes early testing, mid-stage testing, and later comparative research.
Most trials are interventional, meaning the researchers give a treatment and then measure what happens.
Early Parkinson's disease studies
Two trials studied Afamelanotide in people with early Parkinson's disease.
One was a Phase 2a open-label study focused on safety and was marked Withdrawn, with planned enrollment of 10 participants.
The other was a Phase 2 proof-of-concept study that was completed and enrolled 6 participants.
In both Parkinson's studies, the main goal was to assess treatment-emergent adverse events, which are health problems that appear after treatment starts, and to look at clinically significant changes in laboratory results.
A proof-of-concept study means an early trial that checks whether a treatment seems promising enough to study further.
Stroke study
One Phase 2 study looked at Afamelanotide in people with acute arterial ischaemic stroke (AIS), and it was completed with 12 enrolled participants.
This study was a safety study, and the main outcome was the monitoring and recording of treatment-emergent adverse events.
Acute stroke means the study focused on the early period after the stroke event.
Erythropoietic protoporphyria study
One Phase 1 study in erythropoietic protoporphyria (EPP) was completed and enrolled 34 adolescent and adult participants.
The study aimed to determine the pharmacokinetics of Afamelanotide, which means how the body processes the treatment over time.
The trial also aimed to compare the pharmacokinetic profiles in adolescent and adult EPP patients.
The primary outcomes were Cmax and AUC(0-t), two measures of drug exposure in the body.
Healthy volunteer study
One Phase 2 mechanistic study was authorised in healthy volunteers and planned to enroll 10 participants.
A mechanistic study looks at how a treatment affects a biological process, not just whether it helps a disease.
This trial examined the impact of Afamelanotide in aqueous solution on ultraviolet radiation-induced DNA damage and DNA repair capacity in skin.
The primary outcome was the change in UV photoproducts before and after treatment in UV-exposed skin and in non-irradiated skin.
Vitiligo study
One large study in vitiligo was authorised as a Phase 4 open-label trial and planned to enroll 200 participants.
This study compared SCENESSE and narrow-band ultraviolet B (NB-UVB) light versus NB-UVB light alone.
NB-UVB is a form of light treatment used in skin disease, and the trial tested whether adding SCENESSE improved repigmentation.
The main outcome was the percentage of participants who achieved repigmentation, meaning return of skin color, in the combination group compared with the light-only group.
Main outcomes measured
Several different outcomes were used across the Afamelanotide trials, depending on the condition and study phase.
- Safety outcomes: These included treatment-emergent adverse events and clinically significant laboratory changes in Parkinson's disease and stroke studies.
- Pharmacokinetic outcomes: In EPP, the study measured Cmax and AUC(0-t) to understand exposure to the treatment.
- Biological effect outcomes: In healthy volunteers, the study measured UV photoproduct changes to see the effect on DNA damage and repair.
- Clinical effect outcomes: In vitiligo, the study measured repigmentation to see whether the treatment combination improved skin color return.
The trial set shows that Afamelanotide is being explored for both safety and possible benefit in different patient groups, with study goals matched to each condition.