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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Afamelanotide | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Afamelanotide 16 MG | Drug | Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation. |
|
| Measure | Description | Time Frame |
|---|---|---|
| The Change in Disease Severity in Patients With VP as Measured by CGIC. | The higher the score, the less severe the disease. Scale ranges from +3 to -3. | From baseline to Day 168 |
| Measure | Description | Time Frame |
|---|---|---|
| The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA. | A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10. | Median change from baseline to Day 168. |
| The Change in Disease Severity in Patients With VP as Measured by 5-point IGA. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CLINUVEL investigational site | Rotterdam | Netherlands |
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| ID | Title | Description |
|---|---|---|
| FG000 | SCENESSE® (Afamelanotide 16mg) | Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | SCENESSE® (Afamelanotide 16mg) | Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | The Change in Disease Severity in Patients With VP as Measured by CGIC. | The higher the score, the less severe the disease. Scale ranges from +3 to -3. | Posted | Median | Full Range | Scores on a scale | From baseline to Day 168 |
|
|
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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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | SCENESSE® (Afamelanotide 16mg) | Six eligible patients received one dose of afamelanotide 16mg every 28 days, 6 doses in total, as a controlled-release formulation. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| General disorders | General disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Global Clinical Affairs | CLINUVEL UK LTD | +4401372860765 | mail@clinuvel.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 14, 2022 | Feb 6, 2025 | Prot_SAP_001.pdf |
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| ID | Term |
|---|---|
| D046350 | Porphyria, Variegate |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
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| ID | Term |
|---|---|
| C534526 | afamelanotide |
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A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4. |
| Median change from baseline to Day 168 |
| The Change in Disease Severity in Patients With VP as Measured by PGIC. | The higher the score, the less severe the disease. Score ranges from +3 to -3. | Median change from baseline to Day 168 |
| The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS. | A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10. | Median change from baseline to Day 168 |
| The Change in Number of New Skin Lesions Formed. | A lower number indicates a reduced severity of the disease. | Median change from baseline to Day 168. |
| The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH. | Higher score indicates greater impairment and less productivity. Score is percentage impairment. | Median of change from baseline to Day 168 |
| The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB. | Higher scores represent worse health-related quality of life. Scale ranges from 0 to 45. | From baseline to Day 168 |
| The Change in the Quality of Life in Patients With VP as Measured by VP QoL. | "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)?" | Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months. |
| The Change in Outdoor Light Exposure Over Time (Daily Diary) | Daily diaries recording outdoor light exposure. | From baseline to Day 168. |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Confirmed diagnosis of Varigate Porphyria | Count of Participants | Participants |
|
|
| Secondary | The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA. | A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10. | Posted | Median | Full Range | Scores on a scale | Median change from baseline to Day 168. |
|
|
|
| Secondary | The Change in Disease Severity in Patients With VP as Measured by 5-point IGA. | A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4. | Posted | Median | Full Range | Scores on a scale | Median change from baseline to Day 168 |
|
|
|
| Secondary | The Change in Disease Severity in Patients With VP as Measured by PGIC. | The higher the score, the less severe the disease. Score ranges from +3 to -3. | Posted | Median | Full Range | Scores on a scale | Median change from baseline to Day 168 |
|
|
|
| Secondary | The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS. | A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10. | Posted | Median | Full Range | Scores on a scale | Median change from baseline to Day 168 |
|
|
|
| Secondary | The Change in Number of New Skin Lesions Formed. | A lower number indicates a reduced severity of the disease. | Posted | Median | Full Range | Lesions | Median change from baseline to Day 168. |
|
|
|
| Secondary | The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH. | Higher score indicates greater impairment and less productivity. Score is percentage impairment. | Posted | Median | Full Range | Percentage of impairment time | Median of change from baseline to Day 168 |
|
|
|
| Secondary | The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB. | Higher scores represent worse health-related quality of life. Scale ranges from 0 to 45. | Posted | Median | Full Range | Score on a scale | From baseline to Day 168 |
|
|
|
| Secondary | The Change in the Quality of Life in Patients With VP as Measured by VP QoL. | "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)?" | Posted | Count of Participants | Participants | Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months. |
|
|
|
| Secondary | The Change in Outdoor Light Exposure Over Time (Daily Diary) | Daily diaries recording outdoor light exposure. | Posted | Median | Full Range | Change in number of hours of sunlight | From baseline to Day 168. |
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| 0 |
| 6 |
| 0 |
| 6 |
| 4 |
| 6 |
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D011164 | Porphyrias |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |