Variegate Porphyria Clinical Trial
Official title:
Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)-Related Skin Disease.
Verified date | January 2024 |
Source | Clinuvel Pharmaceuticals Limited |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 6 |
Est. completion date | December 28, 2023 |
Est. primary completion date | December 28, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 70 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Netherlands | CLINUVEL investigational site | Rotterdam |
Lead Sponsor | Collaborator |
---|---|
Clinuvel (UK) Ltd. |
Netherlands,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The change in disease severity in patients with VP as measured by a scoring system (A). | The higher the score, the less severe the disease | From baseline to Day 168 | |
Secondary | The change in disease severity in patients with VP as measured by a scoring system (B). | The higher the score, the more severe the disease | From baseline to Day 168 | |
Secondary | The change in disease severity in patients with VP as measured by a scoring system (C) | The higher the score, the more severe the disease | From baseline to Day 168 | |
Secondary | The change in disease severity in patients with VP as measured by a scoring system (D) | The higher the score, the less severe the disease | From baseline to Day 168 | |
Secondary | The change in disease severity in patients with VP as measured by a scoring system (E) | The higher the score, the more severe the disease | From baseline to Day 168 | |
Secondary | The change in number of new skin lesions formed | The higher the number of new lesions, the more severe the disease | From baseline to Day 168 | |
Secondary | The change in the Quality of Life in patients with VP as measured by a validated global quality of life tool (A) | Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life | From baseline to Day 168 | |
Secondary | The change in the Quality of Life in patients with VP as measured by a disease specific tool (B) | Higher scores represent worse health-related quality of life | From baseline to Day 168 | |
Secondary | The change in the Quality of Life in patients with VP as measured by a disease specific questionnaire (C) | Higher scores indicate a better quality of life | From baseline to Day 168 | |
Secondary | The change in outdoor light exposure over time (Daily Diary) | Daily diaries recording outdoor light exposure and trauma events | From baseline to Day 168 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
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