CompletedPhase 2

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

Netherlands6 participantsStarted 2023-03-28

Plain-language 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 patients with VP.

Who can participate

Age range

18 Years – 70 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: From baseline to Day 168

Trial details

NCT IDNCT05854784

SponsorClinuvel Pharmaceuticals Limited

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-12-28

Results submitted2024-12-20

View on ClinicalTrials.gov More Variegate Porphyria trials