Clinical trial NCT05854784: Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Variegate Porphyria
Lead sponsor: Clinuvel Pharmaceuticals Limited
Sponsor class: INDUSTRY
First posted: 2023-05-11
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: The Change in Disease Severity in Patients With VP as Measured by CGIC.
timeFrame: From baseline to Day 168
Secondary outcomes:
measure: The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.
timeFrame: Median change from baseline to Day 168.
measure: The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.
timeFrame: Median change from baseline to Day 168
measure: The Change in Disease Severity in Patients With VP as Measured by PGIC.
timeFrame: Median change from baseline to Day 168
measure: The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.
timeFrame: Median change from baseline to Day 168
measure: The Change in Number of New Skin Lesions Formed.
timeFrame: Median change from baseline to Day 168.
measure: The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.
timeFrame: Median of change from baseline to Day 168
measure: The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB.
timeFrame: From baseline to Day 168
measure: The Change in the Quality of Life in Patients With VP as Measured by VP QoL.
timeFrame: Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months.
measure: The Change in Outdoor Light Exposure Over Time (Daily Diary)
timeFrame: From baseline to Day 168.
Other outcomes:
Locations:
city: Rotterdam
country: Netherlands
facility: CLINUVEL investigational site
Study officials:
References: