Clinical trial NCT05370235: A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V
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 C and V (XPC and XPV)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Xeroderma Pigmentosum
Lead sponsor: Clinuvel Pharmaceuticals Limited
Sponsor class: INDUSTRY
First posted: 2022-05-11
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 CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V 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 or XP-V;
* 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;
* Existing melanoma;
* Female who is pregnant or lactating;
* Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures;
* Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures;
* Use of any other prior and concomitant therapy which may interfere with the objective of the study;
* Participation in a clinical trial for an investigational agent.

Primary outcomes:
measure: Change in minimal erythema dose (MED) in patients with XP-C.
timeFrame: From baseline to day 76.
measure: Change in MED in patients with XP-V.
timeFrame: From baseline to day 76.
Secondary outcomes:
measure: Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-C.
timeFrame: From baseline to day 76.
measure: Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-C.
timeFrame: From baseline to day 76.
measure: Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-V.
timeFrame: From baseline to day 76.
measure: Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-V.
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-C (A).
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-V (A)
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-C (B).
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-V (B).
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-C (C).
timeFrame: From baseline to day 76.
measure: Change in skin disease severity in patients with XP-V (C).
timeFrame: From baseline to day 76.
measure: Change in quality of life assessed by a disease specific tool (A) in patients with XP-C.
timeFrame: From baseline to day 76.
measure: Change in quality of life assessed by a disease specific tool (A) in patients with XP-V.
timeFrame: From baseline to day 76.
measure: Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-C.
timeFrame: From baseline to day 76.
measure: Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-V.
timeFrame: From baseline to day 76.
Other outcomes:
Locations:
city: Clinuvel Investigational Site
country: Belgium
facility: CLINUVEL Investigational site
city: Clinuvel Investigational Site
country: Spain
facility: CLINUVEL Investigational site
Study officials:
References: