Clinical trial NCT05159752: A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
Official title: A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Xeroderma Pigmentosum
Lead sponsor: Clinuvel Pharmaceuticals Limited
Sponsor class: INDUSTRY
First posted: 2021-12-16
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. It will assess whether SCENESSE® increases the amount of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment.


Eligibility criteria: Inclusion Criteria:

* Male or female patient with a molecular-genetically confirmed diagnosis of XP-C;
* Aged 18-75 years.

Exclusion Criteria:

* Known allergy to afamelanotide or the polymer contained in the implant;
* Presence of severe hepatic disease or hepatic impairment;
* Renal impairment;
* Any other medical condition which may interfere with the study protocol;
* Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating;
* Females of child-bearing potential (pre-menopausal, not surgically sterile) not using highly effective contraceptive measures with a failure rate of less than 1% per year when used consistently and correctly (i.e. oral contraceptives, intrauterine device) or a life-style excluding pregnancy, for up to three months after the last implant administration;
* Sexually active man with a partner of child-bearing potential (pre-menopausal, not surgically sterile) who is not using highly effective contraceptive measures, as described above;
* Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening visit;
* Participation in a clinical trial for an investigational agent within 30 days prior to the Screening visit.

Primary outcomes:
measure: Change in minimal erythema dose (MED).
timeFrame: From Baseline to Day 76.
Secondary outcomes:
measure: Change in UV-induced DNA damage and repair capacity.
timeFrame: From Baseline to Day 76.
measure: Change in skin disease severity (A).
timeFrame: From Baseline to Day 238.
measure: Change in skin disease severity (B).
timeFrame: From Baseline to Day 238.
measure: Change in skin disease severity (C).
timeFrame: From Baseline to Day 238.
measure: Change in dermal melanin density.
timeFrame: From Baseline to Day 238.
measure: Change in quality of life assessed by a disease specific tool (A)
timeFrame: From Baseline to Day 238.
measure: Change in quality of life assessed by a validated global quality of life tool (B)
timeFrame: From Baseline to Day 238.
Other outcomes:
Locations:
city: Regensburg
country: Germany
facility: CLINUVEL investigative site
Study officials:
References: