Back to resultsStudy to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
Primary Purpose
Variegate Porphyria
Status
Active
Phase
Phase 2
Locations
Netherlands
Study Type
Interventional
Intervention
Afamelanotide 16 MG
Sponsored by
About this trial
This is an interventional treatment trial for Variegate Porphyria
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.
Sites / Locations
- CLINUVEL investigational site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Afamelanotide
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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
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
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
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
The change in number of new skin lesions formed
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)
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)
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)
Higher scores indicate a better quality of life
The change in outdoor light exposure over time (Daily Diary)
Daily diaries recording outdoor light exposure and trauma events
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05854784
Brief Title
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
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.
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
March 28, 2023 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
March 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Clinuvel (UK) Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Variegate Porphyria
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Afamelanotide
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Afamelanotide 16 MG
Intervention Description
Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
Primary Outcome Measure Information:
Title
The change in disease severity in patients with VP as measured by a scoring system (A).
Description
The higher the score, the less severe the disease
Time Frame
From baseline to Day 168
Secondary Outcome Measure Information:
Title
The change in disease severity in patients with VP as measured by a scoring system (B).
Description
The higher the score, the more severe the disease
Time Frame
From baseline to Day 168
Title
The change in disease severity in patients with VP as measured by a scoring system (C)
Description
The higher the score, the more severe the disease
Time Frame
From baseline to Day 168
Title
The change in disease severity in patients with VP as measured by a scoring system (D)
Description
The higher the score, the less severe the disease
Time Frame
From baseline to Day 168
Title
The change in disease severity in patients with VP as measured by a scoring system (E)
Description
The higher the score, the more severe the disease
Time Frame
From baseline to Day 168
Title
The change in number of new skin lesions formed
Description
The higher the number of new lesions, the more severe the disease
Time Frame
From baseline to Day 168
Title
The change in the Quality of Life in patients with VP as measured by a validated global quality of life tool (A)
Description
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life
Time Frame
From baseline to Day 168
Title
The change in the Quality of Life in patients with VP as measured by a disease specific tool (B)
Description
Higher scores represent worse health-related quality of life
Time Frame
From baseline to Day 168
Title
The change in the Quality of Life in patients with VP as measured by a disease specific questionnaire (C)
Description
Higher scores indicate a better quality of life
Time Frame
From baseline to Day 168
Title
The change in outdoor light exposure over time (Daily Diary)
Description
Daily diaries recording outdoor light exposure and trauma events
Time Frame
From baseline to Day 168
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
CLINUVEL investigational site
City
Rotterdam
Country
Netherlands
12. IPD Sharing Statement
Learn more about this trial
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
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