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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT05854784
Other study ID # CUV040
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date March 28, 2023
Est. completion date December 28, 2023

Study information

Verified date January 2024
Source Clinuvel Pharmaceuticals Limited
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
Netherlands CLINUVEL investigational site Rotterdam

Sponsors (1)

Lead Sponsor Collaborator
Clinuvel (UK) Ltd.

Country where clinical trial is conducted

Netherlands, 

Outcome

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
See also
  Status Clinical Trial Phase
Terminated NCT02922413 - Panhematin for Prevention of Acute Attacks of Porphyria Phase 2
Active, not recruiting NCT02935400 - Acute Porphyria Biomarkers for Disease Activity