Polymorphic Light Eruption
Conditions
Brief summary
This study was to evaluate the safety and efficacy of afamelanotide in patients suffering from polymorphic light eruption (PLE).
Interventions
DRUGAfamelanotide
DRUGPlacebo
Sponsors
Clinuvel Pharmaceuticals Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Aged greater than 18 years * Male or female patients with a documented history of PLE diagnosed or confirmed by a photodermatologist or photobiologist, with a history of PLE related pruritus symptoms. * Recurrent PLE episodes that occur at least once a year (as evidenced by PLE related pruritus symptoms) developing in their own country (to exclude patients affected only when traveling to sunnier climates) * Written informed consent prior to the performance of any study-specific procedure * Are willing and able to comply with the conditions specified in the protocol and study procedures in the opinion of the Investigator
Exclusion criteria
* Currently requiring treatment with systemic immunosuppressive agents * Documented history of other photosensitivity conditions which may be confused with PLE or interfere with the assessment of PLE episodes * Solarium use in the three months prior to study involvement and throughout the duration of the study * Use of immunosuppressive medications, drugs that cause hyperpigmentation or any other treatment that in the opinion of the Investigator may interfere with this study * Documented presence (\> 1 in 320) of Anti-Nuclear Antibody (ANA) and/or positive Extractable Nuclear Antibody (ENA); historical results from the 3 years prior to randomisation are acceptable if available * In the opinion of the Investigator, any evidence of clinically significant organ dysfunction, or any clinically significant deviation from normal in clinical or laboratory parameters * History of drug or alcohol abuse (in the last 1 year) * Female who is pregnant (confirmed by positive serum beta-Human chorionic gonadotropin (β-HCG) pregnancy test prior to baseline) or lactating * Female of child-bearing potential (pre-menopausal, not surgically sterile) that is not using or is not willing to use adequate contraceptive measures (e.g. oral contraceptives, condoms, diaphragm plus spermicide, intrauterine device) * Sexually active men with partners of child bearing potential not willing to use barrier contraception during the trial and for a period of three months thereafter * Participation in a clinical trial with another Investigational Medicinal Product (IMP) within 30 days prior to the Screening visit or during the study * Hypersensitivity to afamelanotide or any of its components
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale | From Day 0 to Day 120 | PLE related pruritus was recorded in paper patient diaries commencing using an 11-point Likert scale from 0 (no pruritus) to 10 (most severe pruritus). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Frequency (Number) of Documented PLE Episodes Recorded in Paper Patient Diaries | From Day 0 to Day 120 | — |
| Duration of PLE Episodes Recorded in Paper Patient Diaries | From Day 0 to Day 120 | — |
| Quality of Life Using the Dermatology Life Quality Index (DLQI) | At Day 0, Day 60 and Day 120 | The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Afamelanotide Afamelanotide implants were administered on Days 0 and 60. Patients returned to the clinic on Day 120 for the final visit.
Afamelanotide | 15 |
| Placebo Placebo implants were administered on Days 0 and 60. Patients returned to the clinic on Day 120 for the final visit.
Placebo | 16 |
| Total | 31 |
Baseline characteristics
| Characteristic | Afamelanotide | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 15 Participants | 16 Participants | 31 Participants |
| Sex: Female, Male Female | 11 Participants | 14 Participants | 25 Participants |
| Sex: Female, Male Male | 4 Participants | 2 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 7 / 15 | 3 / 16 |
| serious Total, serious adverse events | 0 / 15 | 0 / 16 |
Outcome results
Primary
Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale
PLE related pruritus was recorded in paper patient diaries commencing using an 11-point Likert scale from 0 (no pruritus) to 10 (most severe pruritus).
Time frame: From Day 0 to Day 120
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Afamelanotide | Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale | 0.25 score on a scale |
| Placebo | Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale | 0.47 score on a scale |
Secondary
Duration of PLE Episodes Recorded in Paper Patient Diaries
Time frame: From Day 0 to Day 120
Secondary
Frequency (Number) of Documented PLE Episodes Recorded in Paper Patient Diaries
Time frame: From Day 0 to Day 120
Secondary
Quality of Life Using the Dermatology Life Quality Index (DLQI)
The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Time frame: At Day 0, Day 60 and Day 120