NCT05854784已完成2 期
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.
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- The Change in Disease Severity in Patients With VP as Measured by CGIC.
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with confirmed diagnosis of VP.
- •Patients with VP-related skin symptoms.
- •Aged 18-70 years.
排除标准
- •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.
研究组 & 干预措施
Afamelanotide
Experimental
干预措施: Afamelanotide 16 MG (Drug)
结局指标
主要结局
The Change in Disease Severity in Patients With VP as Measured by CGIC.
时间窗: From baseline to Day 168
The higher the score, the less severe the disease. Scale ranges from +3 to -3.
次要结局
- The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.(Median change from baseline to Day 168.)
- The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.(Median change from baseline to Day 168)
- The Change in Disease Severity in Patients With VP as Measured by PGIC.(Median change from baseline to Day 168)
- The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.(Median change from baseline to Day 168)
- The Change in Number of New Skin Lesions Formed.(Median change from baseline to Day 168.)
- The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.(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.(From baseline to Day 168)
- The Change in Outdoor Light Exposure Over Time (Daily Diary)(From baseline to Day 168.)
- The Change in the Quality of Life in Patients With VP as Measured by VP QoL.(Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months.)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
2 期
Afamelanotide in Patients Suffering With Acne VulgarisAcne VulgarisNCT04943159Clinuvel Pharmaceuticals Limited3
已完成
2 期
Afamelanotide as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer SodiumPatients Undergoing Photodynamic Therapy Using Porfimer SodiumNCT04425746Clinuvel Pharmaceuticals Limited16
已完成
1 期
PEP005 Gel - Biological Effects in Actinic Keratosis Assessed by Reflectance Confocal MicroscopyActinic KeratosisNCT01449513LEO Pharma24
招募中
2 期
A Study to Assess the Changes in Pigmentation and Safety of Afamelanotide in Patients With Vitiligo on the FaceVitiligoNCT05210582Clinuvel, Inc.6
已完成
1 期
PEP005 Gel - Biological Effects in Actinic Keratosis Assessed by HistologyActinic KeratosisNCT01387711LEO Pharma27