Variegate PorphyriaPhase 2NCT05854784
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
A Phase 2 study testing Afamelanotide 16 MG in people with Variegate Porphyria, run by Clinuvel Pharmaceuticals Limited at 1 site in Netherlands.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Variegate Porphyria
- What is being tested?
- Afamelanotide 16 MG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteNetherlands
- Study dates
- 28 Mar 2023to 28 Dec 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients with confirmed diagnosis of VP.
- Patients with VP-related skin symptoms.
- Aged 18-70 years.
Who cannot join
- 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.
Show all 11Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
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.
- Conditions
- Variegate Porphyria
- Being tested
- Afamelanotide 16 MG
- Medical subject headings
- Porphyria, Variegate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Afamelanotide 16 MGDrug
Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.
| Study group | Role | What they receive |
|---|---|---|
| Afamelanotide | Experimental | Not described Receives: Drug: Afamelanotide 16 MG |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to Day 168
Secondary outcomes (9)
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.
Measured: Median change from baseline to Day 168.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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)?"
Measured: 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.
Measured: From baseline to Day 168.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2023
- Study start28 Mar 2023
- Primary completion28 Dec 2023
- Study completion28 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Variegate PorphyriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05854784
- Sponsor's protocol ID
- CUV040
- Status last verified
- Apr 2025
- First submitted
- 3 May 2023
- Last updated
- 27 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT05854784
- Acronym / sponsor ID
- CUV040
- Conditions
- Variegate Porphyria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Clinuvel Pharmaceuticals LimitedIndustry (pharma, biotech, devices)
- Study start
- 28 Mar 2023 (actual)
- Primary completion
- 28 Dec 2023 (actual)
- Study completion
- 28 Dec 2023 (actual)
- First posted
- 11 May 2023 (actual)
- Results
- Posted 27 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Afamelanotide | Experimental | Drug: Afamelanotide 16 MG |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female patients with confirmed diagnosis of VP.
- Patients with VP-related skin symptoms.
- Aged 18-70 years.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Afamelanotide 16 MGEligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation. | Drug | Afamelanotide |
Primary outcome
The Change in Disease Severity in Patients With VP as Measured by CGIC.
Time frame: From baseline to Day 168
The higher the score, the less severe the disease. Scale ranges from +3 to -3.
9 secondary outcomes
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.
The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.
Time frame: Median change from baseline to Day 168
The Change in Disease Severity in Patients With VP as Measured by PGIC.
Time frame: Median change from baseline to Day 168
The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.
Time frame: Median change from baseline to Day 168
The Change in Number of New Skin Lesions Formed.
Time frame: Median change from baseline to Day 168.
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
The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB.
Time frame: From baseline to Day 168
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.
The Change in Outdoor Light Exposure Over Time (Daily Diary)
Time frame: From baseline to Day 168.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Variegate Porphyria
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
Active, not recruitingObservational study
Acute Porphyria Biomarkers for Disease Activity
Acute Intermittent Porphyria, Hereditary Coproporphyria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Afamelanotide 16 MG in people with Variegate Porphyria, run by Clinuvel Pharmaceuticals Limited at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Change in Disease Severity in Patients With VP as Measured by CGIC. (time frame: From baseline to Day 168). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2025.