Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Variegate Porphyria (VP)-related skin disease

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. - Phase IIa VP Study

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-004164-60-NL

Enrollment

Registered

2022-02-21

Start date

2022-05-19

Completion date

Unknown

Last updated

2025-02-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Variegate Porphyria (VP)-related skin disease

Interventions

Trade Name: SCENESSE Pharmaceutical Form: Implant

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Male or female patients with confirmed diagnosis of VP. Patients with VP-related skin symptoms assessed with a score equal or above 7 on the 11-point VAS IGA. Aged 18-70 years. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: Known allergy to afamelanotide or the polymer or to lignocaine/lidocaine or other local anaesthetic. Had two or more acute attacks of hepatic porphyria lasting more than two days, within 12 months prior to the Screening period. History of certain malignant and premalignant skin lesions. Individual or family history of melanoma. Severe hepatic disease. Renal impairment (eGFR (MDRD) < 30 ml/min*1.73m2). Female who is pregnant or lactating. Females of child-bearing potential not using adequate contraceptive measures. Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures. Not suitable for trial participation in the opinion of the Investigator.

Design outcomes

Primary

Measure	Time frame
Main Objective: determine whether afamelanotide implants can reduce the severity of the skin disease in patients with VP;Secondary Objective: Evaluate the safety and tolerability of afamelanotide in patients with VP. Evaluate the impact of afamelanotide on the quality of life of patients with VP.;Primary end point(s): E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.;Timepoint(s) of evaluation of this end point: E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.	—

Measure

Time frame

Main Objective: determine whether afamelanotide implants can reduce the severity of the skin disease in patients with VP;Secondary Objective: Evaluate the safety and tolerability of afamelanotide in patients with VP. Evaluate the impact of afamelanotide on the quality of life of patients with VP.;Primary end point(s): E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.;Timepoint(s) of evaluation of this end point: E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.

—

Secondary

Measure	Time frame
Secondary end point(s): E.5.2 Secondary End Point (repeat as necessary) - The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient’s Global Assessment using a VAS. - The change in the number of new skin lesions formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196 as counted by the Investigator. - The change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH, VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily Diary). Trauma events will be tabulated.;Timepoint(s) of evaluation of this end point: E.5.2 Secondary End Point (repeat as necessary) - The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient’s Global Assessment using a VAS. - The change in the number of new skin lesions formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196 as counted by the Investigator. - The change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH, VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily Diary). Trauma events will be tabulated.	—

Measure

Time frame

Secondary end point(s): E.5.2 Secondary End Point (repeat as necessary) - The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient’s Global Assessment using a VAS. - The change in the number of new skin lesions formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196 as counted by the Investigator. - The change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH, VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily Diary). Trauma events will be tabulated.;Timepoint(s) of evaluation of this end point: E.5.2 Secondary End Point (repeat as necessary) - The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient’s Global Assessment using a VAS. - The change in the number of new skin lesions formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196 as counted by the Investigator. - The change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH, VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily Diary). Trauma events will be tabulated.

—

Countries

Netherlands, Switzerland

Contacts

Public ContactClinical Project Manager

CLINUVEL (UK) LTD

mail@clinuvel.com00441372860765

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026