Patients with Xeroderma Pigmentosum C and V
Conditions
Interventions
Trade Name: SCENESSE
Product Name: Afamelanotide (16 mg implant)
Pharmaceutical Form: Implant
Sponsors
CLINUVEL EUROPE LIMITED
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Male or female patient with a molecular-genetically confirmed diagnosis of XPC or XPV. -Aged 18-75 years. -Providing written Informed Consent prior to the performance of any study-specific procedure. -Willing and able to comply with the conditions specified in the protocol and study procedures, in the opinion of the Investigator. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6
Exclusion criteria
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 (confirmed by positive urine ß-HCG pregnancy test) or lactating; •Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, 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 adequate 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; •Not suitable for trial participation in the opinion of the Investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XPC •Evaluate the impact of afamelanotide on MED in patients with XPV. ;Secondary Objective: •Evaluate the impact of afamelanotide on UV-induced DNA damage and repair capacity in patients with XPC. •Evaluate the impact of afamelanotide on UV-induced DNA damage and repair capacity in patients with XPV. •Evaluate the impact of afamelanotide on melanin density in patients with XPC. •Evaluate the impact of afamelanotide on melanin density in patients with XPV. •Evaluate the safety and tolerability of afamelanotide in patients with XP. •Evaluate the impact of afamelanotide on the skin disease severity of patients with XPC. •Evaluate the impact of afamelanotide on the skin disease severity of patients with XPV. •Evaluate the impact of afamelanotide on the quality of life of patients with XPC. •Evaluate the impact of afamelanotide on the quality of life of patients with XPV;Primary end point(s): The change in MED ;Timepoint(s) of evaluation of this end point: Analysis will compare the individual fold increase in MED from Day 0 to the post-treatment assessment at Day 76. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •The changes in UV-induced DNA damage and repair capacity from baseline to Day 76, as measured through analysis of UV photoproducts and DNA repair mechanisms on irradiated skin in comparison with non-irradiated skin; •The change in skin disease severity from baseline to Day 70, Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14) as measured by: 5-point IGA scale; 11-point Likert-type scale. •The change in (skin) disease severity from baseline to Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14) as measured by the European XP severity score. •The change in melanin density from baseline to Days 28, 56, 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14) as measured by spectrophotometry. •The change in Quality of Life from baseline to Days 70, 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14), as measured by the two instruments: WPAI:GH; XP-derived QOLEB. ;Timepoint(s) of evaluation of this end point: Please see above. | — |
Countries
Belgium, Spain
Contacts
Public ContactHead of clinical operations
CLINVEL EUROPE LIMITED
Outcome results
None listed