Erythropoietic Protoporphyria
Conditions
Brief summary
The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients.
Interventions
DRUGAfamelanotide
Sponsors
Clinuvel Pharmaceuticals Limited
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies; * aged 18-75 years (inclusive); * provide written informed patient consent prior to the performance of any study-specific procedure.
Exclusion criteria
* any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant; * any allergy to lignocaine or other local anaesthetic to be used during the administration of the study medication; * EPP patients with significant hepatic involvement; * personal history of melanoma or dysplastic nevus syndrome; * current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions; * any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations; * acute history of drug or alcohol abuse (in the last 12 months); * female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (HCG) pregnancy test prior to baseline) 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); * sexually active men with partners of child-bearing potential not using barrier contraception during the trial and for a period of three months thereafter; * participation in a clinical trial for an investigational agent within 30 days prior to the screening visit; * prior and concomitant therapy with medications which may interfere with the objectives of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life. |
| Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | 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 | 16 |
| Total | 16 |
Baseline characteristics
| Characteristic | Afamelanotide |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 15 Participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 1 / 16 |
| serious Total, serious adverse events | 0 / 16 |
Outcome results
Primary
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (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: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
Population: Intent-to-treat population
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Afamelanotide | Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Day 60 | -5 DLQI score |
| Afamelanotide | Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Day 120 | -4.5 DLQI score |
| Afamelanotide | Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Day 180 or ET | -5 DLQI score |
Primary
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life.
Time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
Population: Intent-to-treat population
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Afamelanotide | Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Day 60 | 27.8 EPP-QoL score |
| Afamelanotide | Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Day 120 | 59.7 EPP-QoL score |
| Afamelanotide | Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Day 180 or ET | 56.9 EPP-QoL score |