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A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05159752
Recruitment Status : Recruiting
First Posted : December 16, 2021
Last Update Posted : September 28, 2022
Sponsor:
Information provided by (Responsible Party):
Clinuvel Pharmaceuticals Limited ( Clinuvel Europe Limited )

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.

Condition or disease Intervention/treatment Phase
Xeroderma Pigmentosum Drug: Afamelanotide Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : October 19, 2021
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : January 2024


Arm Intervention/treatment
Experimental: Afamelanotide Drug: Afamelanotide
Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.




Primary Outcome Measures :
  1. Change in minimal erythema dose (MED). [ Time Frame: From Baseline to Day 76. ]
    MED is the lowest dose of UV light that causes reddening of the skin.


Secondary Outcome Measures :
  1. Change in UV-induced DNA damage and repair capacity. [ Time Frame: From Baseline to Day 76. ]
    Analysis of UV photoproducts and DNA repair mechanisms.

  2. Change in skin disease severity (A). [ Time Frame: From Baseline to Day 238. ]
    The higher the score, the more severe the disease.

  3. Change in skin disease severity (B). [ Time Frame: From Baseline to Day 238. ]
    The higher the score, the more severe the disease.

  4. Change in skin disease severity (C). [ Time Frame: From Baseline to Day 238. ]
    The higher the score, the more severe the disease.

  5. Change in dermal melanin density. [ Time Frame: From Baseline to Day 238. ]
    Non-invasive quantitative skin reflectance measurement.

  6. Change in quality of life assessed by a disease specific tool (A) [ Time Frame: From Baseline to Day 238. ]
    Higher scores represent worse health-related quality of life.

  7. Change in quality of life assessed by a validated global quality of life tool (B) [ Time Frame: From Baseline to Day 238. ]
    Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05159752


Contacts
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Contact: Head of Clinical Operations +441372860765 mail@clinuvel.com

Locations
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Germany
CLINUVEL investigative site Recruiting
Regensburg, Germany
Contact: Head of Clinical Operations       mail@clinuvel.com   
Sponsors and Collaborators
Clinuvel Europe Limited
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Responsible Party: Clinuvel Europe Limited
ClinicalTrials.gov Identifier: NCT05159752    
Other Study ID Numbers: CUV156
First Posted: December 16, 2021    Key Record Dates
Last Update Posted: September 28, 2022
Last Verified: September 2022

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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
Additional relevant MeSH terms:
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Xeroderma Pigmentosum
Ichthyosis
Skin Abnormalities
Congenital Abnormalities
Infant, Newborn, Diseases
Keratosis
Skin Diseases
Precancerous Conditions
Neoplasms
Skin Diseases, Genetic
Genetic Diseases, Inborn
Photosensitivity Disorders
Pigmentation Disorders
DNA Repair-Deficiency Disorders
Metabolic Diseases
Afamelanotide
Dermatologic Agents