Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
Study Details
Study Description
Brief 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Afamelanotide
|
Drug: Afamelanotide 16 MG
Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
Outcome Measures
Primary Outcome Measures
- The change in disease severity in patients with VP as measured by a scoring system (A). [From baseline to Day 168]
The higher the score, the less severe the disease
Secondary Outcome Measures
- The change in disease severity in patients with VP as measured by a scoring system (B). [From baseline to Day 168]
The higher the score, the more severe the disease
- The change in disease severity in patients with VP as measured by a scoring system (C) [From baseline to Day 168]
The higher the score, the more severe the disease
- The change in disease severity in patients with VP as measured by a scoring system (D) [From baseline to Day 168]
The higher the score, the less severe the disease
- The change in disease severity in patients with VP as measured by a scoring system (E) [From baseline to Day 168]
The higher the score, the more severe the disease
- The change in number of new skin lesions formed [From baseline to Day 168]
The higher the number of new lesions, the more severe the disease
- The change in the Quality of Life in patients with VP as measured by a validated global quality of life tool (A) [From baseline to Day 168]
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life
- The change in the Quality of Life in patients with VP as measured by a disease specific tool (B) [From baseline to Day 168]
Higher scores represent worse health-related quality of life
- The change in the Quality of Life in patients with VP as measured by a disease specific questionnaire (C) [From baseline to Day 168]
Higher scores indicate a better quality of life
- The change in outdoor light exposure over time (Daily Diary) [From baseline to Day 168]
Daily diaries recording outdoor light exposure and trauma events
Eligibility Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CLINUVEL investigational site | Rotterdam | Netherlands |
Sponsors and Collaborators
- Clinuvel (UK) Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CUV040