Clinical Trial Results:
A 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.
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Summary
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EudraCT number |
2018-004164-60 |
Trial protocol |
NL |
Global end of trial date |
28 Dec 2023
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Results information
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Results version number |
v1(current) |
This version publication date |
05 Jan 2025
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First version publication date |
05 Jan 2025
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
CUV040
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05854784 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
CLINUVEL (UK)
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Sponsor organisation address |
The Old Post Office, 33 Station Road, Egham, Surrey, United Kingdom, TW20 9LA
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Public contact |
Director, Global Clinical Affairs, CLINUVEL (UK) LTD, 0044 1372860765, mail@clinuvel.com
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Scientific contact |
Director, Global Clinical Affairs, CLINUVEL (UK) LTD, 0044 1372860765, mail@clinuvel.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
10 May 2024
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
28 Dec 2023
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Global end of trial reached? |
Yes
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Global end of trial date |
28 Dec 2023
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP).
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Protection of trial subjects |
This study was conducted in accordance with the Declaration of Helsinki, its revisions (Scotland, October 2000 and incorporating Notes of Clarification – Washington, 2002, Tokyo, 2004 and Seoul 2008) and ICH guidelines for Good Clinical Practice (GCP) governing the conduct of studies, and all applicable local regulations. The investigator assured that the study was conducted in accordance with prevailing local laws and regulations. The investigator was responsible for reporting to the Sponsor, the authorities and the Ethics Committee any modifications, safety updates, amendments, and violations of the protocol that impact on subject safety.
Prior to any study specific screening procedures, the Investigator had explained to each participant and/or to his/her legal representative, if necessary, the nature of the study, its purpose, procedures to be performed, the necessity for withdrawal of prohibited medication, expected duration, and the benefits and risks of study participation. After this explanation and before any study specific procedures were performed, the subject had voluntarily signed an informed consent statement.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
28 Mar 2023
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Switzerland: 3
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Country: Number of subjects enrolled |
Netherlands: 3
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Worldwide total number of subjects |
6
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EEA total number of subjects |
3
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
5
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
Recruitment was conducted as per the protocol. | ||||||
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Pre-assignment
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Screening details |
Screening was conducted as per protocol. | ||||||
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Period 1
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Period 1 title |
Overall Period
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
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Arms
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Arm title
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SCENESSE® (afamelanotide 16mg) | ||||||
Arm description |
Six eligible patients received six doses of afamelanotide 16mg, one dose every 28 days, as a controlled-release formulation. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
SCENESSE® (afamelanotide 16mg)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Implant
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
16mg implant, 1 dose every 28 days, 6 doses in total
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Baseline characteristics reporting groups
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Reporting group title |
Overall Period
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Reporting group description |
- | ||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
SCENESSE® (afamelanotide 16mg)
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Reporting group description |
Six eligible patients received six doses of afamelanotide 16mg, one dose every 28 days, as a controlled-release formulation. | ||
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End point title |
The change in disease severity in patients with VP as measured by a scoring system (A). [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
From baseline to Day 168.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Statistics are not provided because they are not mandatory for a single arm study. |
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in disease severity in patients with VP as measured by a scoring system (B). | ||||||||
End point description |
A lower score indicates a reduced severity of the disease.
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in disease severity in patients with VP as measured by a scoring system (C). | ||||||||
End point description |
A lower score indicates a reduced severity of the disease.
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in disease severity in patients with VP as measured by a scoring system (D). | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in disease severity in patients with VP as measured by a scoring system (E). | ||||||||
End point description |
A lower score indicates a reduced severity of the disease.
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in number of new skin lesions formed. | ||||||||
End point description |
A lower score indicates a reduced severity of the disease.
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in the Quality of Life in patients with VP as measured by a validated global quality of life tool (A). | ||||||||
End point description |
Percent activity impairment due to health. A lower score indicates a reduced impairment.
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End point type |
Secondary
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End point timeframe |
Median change from baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in the Quality of Life in patients with VP as measured by a disease specific tool (B). | ||||||||
End point description |
A lower score indicates a reduced severity of the disease.
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End point type |
Secondary
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End point timeframe |
From baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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End point title |
The change in the Quality of Life in patients with VP as measured by disease specific questionnaire (C). | ||||||
End point description |
Not appropriate to calculate total scores.
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End point type |
Secondary
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End point timeframe |
From baseline to Day 168.
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| No statistical analyses for this end point | |||||||
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End point title |
The change in outdoor light exposure over time (Daily Diary). | ||||||||
End point description |
Median number of daily hours of sunlight exposure.
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End point type |
Secondary
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End point timeframe |
From baseline to Day 168.
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| No statistical analyses for this end point | |||||||||
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Adverse events information [1]
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Timeframe for reporting adverse events |
All adverse events were monitored from baseline until 3 months after end of treatment.
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Assessment type |
Non-systematic | ||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||
Dictionary version |
26.1
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Reporting groups
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Reporting group title |
Arm 1
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Reporting group description |
To avoid potential breach of confidentiality and privacy, adverse event information is not released as a rare disease patient may be individually identifiable from a small study. | ||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||
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| Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: To avoid potential breach of confidentiality and privacy, adverse event information is not released as a rare disease patient may be individually identifiable from a small study. |
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
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