Skip to content

Trial · Interventional

CompletedPhase 2Results postedRegistry data

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

NCTNCT05854784 (opens ClinicalTrials.gov in a new tab)Other idCUV040
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 2
Enrollment
6 participants (actual)
Sponsor
Clinuvel Pharmaceuticals Limited · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

Netherlands

Start
28 Mar 2023
Primary completion
28 Dec 2023
Completion
28 Dec 2023
First posted
11 May 2023
Last update posted
27 Apr 2025
Conditions
Variegate Porphyria

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20232024
  1. Ph 2

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

    NCT05854784

    28 Mar 2023 to 28 Dec 2023

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • Afamelanotide 16 MGDrug

Arms1

  • Experimental

    Afamelanotide

Primary outcomes1

Outcomes the sponsor listed as primary.

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

    Time frame · From baseline to Day 168

    Registry description

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

Secondary outcomes(9)

  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.

    Registry description

    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

    Registry description

    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

    Registry description

    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

    Registry description

    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.

    Registry description

    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

    Registry description

    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

    Registry description

    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.

    Registry description

    "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.

    Registry description

    Daily diaries recording outdoor light exposure.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMedian· From baseline to Day 168

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

GroupValue (Scores on a scale)
Afamelanotide2.5
Secondary and other outcome results(7)
Secondary outcomeMedian· Median change from baseline to Day 168.

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

GroupValue (Scores on a scale)
SCENESSE® (Afamelanotide 16mg)-3.0
Secondary outcomeMedian· Median change from baseline to Day 168

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

GroupValue (Scores on a scale)
SCENESSE® (Afamelanotide 16mg)-2.0
Secondary outcomeMedian· Median change from baseline to Day 168

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

GroupValue (Scores on a scale)
SCENESSE® (Afamelanotide 16mg)2.5
Secondary outcomeMedian· Median change from baseline to Day 168

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

GroupValue (Scores on a scale)
SCENESSE® (Afamelanotide 16mg)-6.5
Secondary outcomeMedian· Median change from baseline to Day 168.

The Change in Number of New Skin Lesions Formed.

GroupValue (Lesions)
SCENESSE® (Afamelanotide 16mg)-6.5
Secondary outcomeMedian· Median of change from baseline to Day 168

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

GroupValue (Percentage of impairment time)
SCENESSE® (Afamelanotide 16mg)-10.00
Secondary outcomeMedian· From baseline to Day 168

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

GroupValue (Score on a scale)
SCENESSE® (Afamelanotide 16mg)-12.0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
SCENESSE® (Afamelanotide 16mg)Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation.0 / 60 / 64 / 6

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years up to 70 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

PepBank is a research reference. Talk to a doctor before you use any peptide.