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NCT Number: NCT05854784

Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

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.

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Key information

Conditions

Variegate Porphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyria, Variegate Porphyrias Porphyrias, Hepatic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

CLINUVEL investigational site

Rotterdam, Netherlands

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Afamelanotide 16 MG

Drug

Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.

Primary outcomes

  1. The Change in Disease Severity in Patients With VP as Measured by CGIC.

    Time frame: From baseline to Day 168

    The higher the score, the less severe the disease. Scale ranges from +3 to -3.

Secondary outcomes

  1. The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.

    Time frame: Median change from baseline to Day 168.

    A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10.

  2. The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.

    Time frame: Median change from baseline to Day 168

    A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4.

  3. The Change in Disease Severity in Patients With VP as Measured by PGIC.

    Time frame: Median change from baseline to Day 168

    The higher the score, the less severe the disease. Score ranges from +3 to -3.

  4. The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.

    Time frame: Median change from baseline to Day 168

    A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10.

  5. The Change in Number of New Skin Lesions Formed.

    Time frame: Median change from baseline to Day 168.

    A lower number indicates a reduced severity of the disease.

  6. The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.

    Time frame: Median of change from baseline to Day 168

    Higher score indicates greater impairment and less productivity. Score is percentage impairment.

  7. The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB.

    Time frame: From baseline to Day 168

    Higher scores represent worse health-related quality of life. Scale ranges from 0 to 45.

  8. The Change in the Quality of Life in Patients With VP as Measured by VP QoL.

    Time frame: Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months.

    "Quality of life measured by the number of responses of "much better than a year ago" to the question "How would you judge the condition of your skin of your face and hands today, in comparison to the situation last year (12 months ago)?"

  9. The Change in Outdoor Light Exposure Over Time (Daily Diary)

    Time frame: From baseline to Day 168.

    Daily diaries recording outdoor light exposure.

Sponsors and collaborators

Lead sponsor

Clinuvel Pharmaceuticals Limited

Industry

Registry information

Official study 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.

Important dates

Study start
2023
Primary completion
2023
Study completion
2023
First posted
May 11, 2023
Registry last updated
Apr 27, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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