Phase 2
N=6
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
Variegate Porphyria
Bottom Line
View on ClinicalTrials.gov: NCT05854784 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Apr 2025
Primary outcome: Primary: The Change in Disease Severity in Patients With VP as Measured by CGIC. — 2.5 Scores on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Afamelanotide 16 MG (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Clinuvel Pharmaceuticals Limited
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Change in Disease Severity in Patients With VP as Measured by CGIC. |
2.5 | — |
| SECONDARY The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA. |
-3.0 | — |
| SECONDARY The Change in Disease Severity in Patients With VP as Measured by 5-point IGA. |
-2.0 | — |
| SECONDARY The Change in Disease Severity in Patients With VP as Measured by PGIC. |
2.5 | — |
| SECONDARY The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS. |
-6.5 | — |
| SECONDARY The Change in Number of New Skin Lesions Formed. |
-6.5 | — |
| SECONDARY The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH. |
-10.00 | — |
| SECONDARY The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB. |
-12.0 | — |
| SECONDARY The Change in the Quality of Life in Patients With VP as Measured by VP QoL. |
0; 5 | — |
| SECONDARY The Change in Outdoor Light Exposure Over Time (Daily Diary) |
0.70 | — |
Summary
The primary objective is to evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP). The secondary objectives are to evaluate the safety and tolerability of afamelanotide in patients with VP and evaluate the impact of afamelanotide on the quality of life of patients with VP.
Eligibility Criteria
Inclusion Criteria
- Male or female patients with confirmed diagnosis of VP.
- Patients with VP-related skin symptoms.
- Aged 18-70 years.
Exclusion Criteria
- Allergy to afamelanotide or polymer or to local anaesthetic to be used if applicable.
- Had two or more acute attacks of hepatic porphyria, as defined by the occurrence or acute porphyric symptoms within 12 months prior to the Screening period.
- Individual history of malignant or premalignant skin lesions.
- Individual or family history of melanoma.
- Presence of severe hepatic disease.
- Renal impairment.
- Female who is pregnant or lactating.
- Females of child-bearing potential not using adequate contraceptive or a life-style excluding pregnancy.
- Sexually active man with a partner of child-bearing potential not using adequate contraceptive measures.
- Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening period.
- Participation in a clinical trial within 30 days prior to the Screening period.
Data sourced from ClinicalTrials.gov (NCT05854784). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.