Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP) (NCT NCT05854784)
NCT ID: NCT05854784
Last Updated: 2025-04-27
Results Overview
The higher the score, the less severe the disease. Scale ranges from +3 to -3.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
From baseline to Day 168
Results posted on
2025-04-27
Participant Flow
Participant milestones
| Measure |
SCENESSE® (Afamelanotide 16mg)
Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
Baseline characteristics by cohort
| Measure |
SCENESSE® (Afamelanotide 16mg)
n=6 Participants
Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
1 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Race · White (non-Hispanic, Latino or Spanish)
|
4 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Race · White (Hispanic, Latino or Spanish)
|
1 Participants
n=99 Participants
|
|
Confirmed diagnosis of Varigate Porphyria
|
6 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: From baseline to Day 168The higher the score, the less severe the disease. Scale ranges from +3 to -3.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Disease Severity in Patients With VP as Measured by CGIC.
|
2.5 Scores on a scale
Interval 1.0 to 3.0
|
SECONDARY outcome
Timeframe: Median change from baseline to Day 168.A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.
|
-3.0 Scores on a scale
Interval -5.0 to 0.0
|
SECONDARY outcome
Timeframe: Median change from baseline to Day 168A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.
|
-2.0 Scores on a scale
Interval -3.0 to 1.0
|
SECONDARY outcome
Timeframe: Median change from baseline to Day 168The higher the score, the less severe the disease. Score ranges from +3 to -3.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Disease Severity in Patients With VP as Measured by PGIC.
|
2.5 Scores on a scale
Interval 1.0 to 3.0
|
SECONDARY outcome
Timeframe: Median change from baseline to Day 168A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.
|
-6.5 Scores on a scale
Interval -9.0 to -1.0
|
SECONDARY outcome
Timeframe: Median change from baseline to Day 168.A lower number indicates a reduced severity of the disease.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Number of New Skin Lesions Formed.
|
-6.5 Lesions
Interval -11.0 to 4.0
|
SECONDARY outcome
Timeframe: Median of change from baseline to Day 168Higher score indicates greater impairment and less productivity. Score is percentage impairment.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.
|
-10.00 Percentage of impairment time
Interval -80.0 to 40.0
|
SECONDARY outcome
Timeframe: From baseline to Day 168Higher scores represent worse health-related quality of life. Scale ranges from 0 to 45.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB.
|
-12.0 Score on a scale
Interval -23.0 to -3.0
|
SECONDARY outcome
Timeframe: 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)?"
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in the Quality of Life in Patients With VP as Measured by VP QoL.
Baseline
|
0 Participants
|
|
The Change in the Quality of Life in Patients With VP as Measured by VP QoL.
Day 168
|
5 Participants
|
SECONDARY outcome
Timeframe: From baseline to Day 168.Daily diaries recording outdoor light exposure.
Outcome measures
| Measure |
Afamelanotide
n=6 Participants
Afamelanotide 16 MG: Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
|
|---|---|
|
The Change in Outdoor Light Exposure Over Time (Daily Diary)
|
0.70 Change in number of hours of sunlight
Interval 0.2 to 2.3
|
Adverse Events
SCENESSE® (Afamelanotide 16mg)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
SCENESSE® (Afamelanotide 16mg)
n=6 participants at risk
Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation.
|
|---|---|
|
General disorders
General disorders
|
66.7%
4/6 • Number of events 10 • All adverse events were monitored from baseline until 3 months after end of treatment (approximately 9 months)
AE are reported as "General Disorders" to maintain confidentiality as the patients in this study are European Union citizens.The European Medicines Agency (EMA) has agreed that where single patient adverse event cases occur in a rare disease study there is a potential for identification of the patient.The EMA therefore considers such AE cases to be medical information on an individual and accepts redaction of the information on General Data Protection Regulation (Regulation(EU)2016/679) grounds.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place