Back to resultsA Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
Primary Purpose
Xeroderma Pigmentosum
Status
Recruiting
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
Afamelanotide
Sponsored by
About this trial
This is an interventional treatment trial for Xeroderma Pigmentosum
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.
Sites / Locations
- CLINUVEL investigative siteRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Afamelanotide
Arm Description
Outcomes
Primary Outcome Measures
Change in minimal erythema dose (MED).
MED is the lowest dose of UV light that causes reddening of the skin.
Secondary Outcome Measures
Change in UV-induced DNA damage and repair capacity.
Analysis of UV photoproducts and DNA repair mechanisms.
Change in skin disease severity (A).
The higher the score, the more severe the disease.
Change in skin disease severity (B).
The higher the score, the more severe the disease.
Change in skin disease severity (C).
The higher the score, the more severe the disease.
Change in dermal melanin density.
Non-invasive quantitative skin reflectance measurement.
Change in quality of life assessed by a disease specific tool (A)
Higher scores represent worse health-related quality of life.
Change in quality of life assessed by a validated global quality of life tool (B)
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Full Information
NCT ID
NCT05159752
First Posted
November 9, 2021
Last Updated
June 15, 2023
Sponsor
Clinuvel Europe Limited
1. Study Identification
Unique Protocol Identification Number
NCT05159752
Brief Title
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
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 19, 2021 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
October 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Clinuvel Europe Limited
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Xeroderma Pigmentosum
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Afamelanotide
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Afamelanotide
Intervention Description
Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.
Primary Outcome Measure Information:
Title
Change in minimal erythema dose (MED).
Description
MED is the lowest dose of UV light that causes reddening of the skin.
Time Frame
From Baseline to Day 76.
Secondary Outcome Measure Information:
Title
Change in UV-induced DNA damage and repair capacity.
Description
Analysis of UV photoproducts and DNA repair mechanisms.
Time Frame
From Baseline to Day 76.
Title
Change in skin disease severity (A).
Description
The higher the score, the more severe the disease.
Time Frame
From Baseline to Day 238.
Title
Change in skin disease severity (B).
Description
The higher the score, the more severe the disease.
Time Frame
From Baseline to Day 238.
Title
Change in skin disease severity (C).
Description
The higher the score, the more severe the disease.
Time Frame
From Baseline to Day 238.
Title
Change in dermal melanin density.
Description
Non-invasive quantitative skin reflectance measurement.
Time Frame
From Baseline to Day 238.
Title
Change in quality of life assessed by a disease specific tool (A)
Description
Higher scores represent worse health-related quality of life.
Time Frame
From Baseline to Day 238.
Title
Change in quality of life assessed by a validated global quality of life tool (B)
Description
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Time Frame
From Baseline to Day 238.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Head of Clinical Operations
Phone
+441372860765
Email
mail@clinuvel.com
Facility Information:
Facility Name
CLINUVEL investigative site
City
Regensburg
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Head of Clinical Operations
Email
mail@clinuvel.com
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
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