Primary outcomeMedian· Daily for 6 months
Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days
| Group | Value (Hours) |
|---|---|
| Afamelanotide | 8.25 |
| Placebo | 0.75 |
Trial · Interventional
CompletedPhase 2Results postedRegistry dataPhase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
NCTNCT01097044 (opens ClinicalTrials.gov in a new tab)Other idCUV030
Overview
- Status
- Completed
- Phase
- Phase 2
- Enrollment
- 77 participants (actual)
- Sponsor
- Clinuvel Pharmaceuticals Limited · Industry
- Design
- Randomized · Quadruple masking
- Countries
1 country
United States
- Start
- Apr 2010
- Primary completion
- Apr 2011
- Completion
- Apr 2011
- First posted
- 1 Apr 2010 (estimated)
- Last update posted
- 28 Oct 2019
- Conditions
- Erythropoietic Protoporphyria
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
201020112012
- Ph 2
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen: * Group A will be administered afamelanotide implants on Days 0, 60 and 120 * Group B will be administered placebo implants on Days 0, 60 and 120 To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured using the EPP specific questionnaire (EPP-QoL) every 60 days and the DLQI questionnaire every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.
Interventions
- AfamelanotideDrug
- PlaceboDrug
Arms2
Experimental
Afamelanotide
Dose: 16 mg implant; release of 16 mg over 7 to 10 days Mode of administration: Subcutaneous implantation Frequency: Every 60 days (on Days 0, 60 and 120)
Placebo comparator
Placebo
Dose: 16 mg implant; Mode of administration: Subcutaneous implantation Frequency: Every 60 days (on Days 0, 60 and 120)
Primary outcomes1
Outcomes the sponsor listed as primary.
Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days
Time frame · Daily for 6 months
Registry description
The amount of direct sunlight exposure between 10:00 and 15:00 hours on days when no pain was experienced (e.g. 11-point Likert pain score of 0). Time was recorded in a patient dairy using 15 minute time blocks. The pain score is measured by the 11-point Likert Pain scale with minimum of 0 and maximum of 10. Likert Pain scale of 0 represents no pain and 10 represents worst imaginable pain.
Secondary outcomes(6)
Maximum Severity of Phototoxic Reaction Experienced by Participants
Time frame · Daily for 6 months
Registry description
The days on which the participant experienced pain as a result of phototoxic reactions (caused by exposure to natural light) was recorded in a study diary. On each day such a reaction occurred, the participant scored the level of pain using an 11-point Likert pain scale, with minimum of 0 and maximum of 10. The 11-point Likert pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain. The maximum severity of a phototoxic reaction was determined by the highest daily 11-point Likert scale score that occurred during that phototoxic reaction.
Quality of Life Measured by Participant Completed Questionnaire
Time frame · Day 0, Day 60, Day 120, Day 180
Registry description
Erythropoietic Protoporphyria Quality of Life Measure (EPP-QoL) is used to measure the quality of life of participants. The total EPP-QoL score ranges from 0 to 100, with a score of 0 as the worst quality of life and score of 100 as the best quality of life.
Change of Total Protoporphyrin IX Level in Participants
Time frame · Baseline, Day 60, Day 120, Day 180
Registry description
This was an exploratory assessment only to analyze whether afamelanotide-induced change in sun exposure would result in a reduction of protoporphyrin IX. The changes of the Total Protoporphyrin IX Level (μg/dL) from Screening Visit (ITT Population) were measured between the two groups. The Protoporphyrin IX level is a laboratory parameter that is measured in specialist labs.
Number of Participants With Phototoxic Reactions With Likert Severity Scores ≥ 4 and ≥ 7
Time frame · Daily for 6 months
Registry description
The number of participants who experienced phototoxic reactions with Likert severity scores ≥ 4 and severity scores ≥7 were recorded. A derived endpoint was used. The number of participants who reported at least one phototoxic reaction with a Likert severity score of ≥ 4 was recorded. For severity scores ≥ 7, the number of patients who reported at least one phototoxic reaction with a Likert severity score of ≥ 7 was recorded. The 11-point Likert pain scale ranges from minimum of 0 to maximum of 10. The 11-point Likert pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain.
Number of Phototoxic Reactions Experienced by Participants
Time frame · Daily for 6 months
Registry description
The days on which the participant experienced pain as a result of phototoxic reactions (caused by exposure to natural light) was recorded in a study diary. On each day such a reaction occurred, the participant scored the level of pain using an 11-point Likert pain scale, with minimum of 0 and maximum of 10. The 11-point Likert pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain. The number of phototoxic reactions was determined by counting the number of episodes on which participants report a 11-point Likert scale score of 4 or more for one or more consecutive days.
Total Severity of Phototoxic Reactions Experienced by Participants Over the Entire Study
Time frame · Daily for 6 months.
Registry description
The days on which the participant experienced pain as a result of phototoxic reactions (caused by exposure to natural light) was recorded in a study diary. On each day such a reaction occurred, the participant scored the level of pain using an 11-point Likert pain scale, with minimum of 0 and maximum of 10. The 11-point Likert Pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain. The total severity of phototoxic reactions was determined by the sum of daily 11-point Likert scale scores that occurred during phototoxic reactions. The overall sum of the severity per participant over the entire study was analyzed. The theoretical minimum score is 0 and the maximum possible score is 1800.
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(6)
Secondary outcomeMedian· Daily for 6 months
Maximum Severity of Phototoxic Reaction Experienced by Participants
| Group | Value (score on a scale) |
|---|---|
| Afamelanotide | 5.0 |
| Placebo | 5.0 |
Secondary outcomeMedian· Day 0, Day 60, Day 120, Day 180
Quality of Life Measured by Participant Completed Questionnaire
| Group | Value (units on a scale) |
|---|---|
| Afamelanotide | 34.7 |
| Placebo | 22.2 |
Secondary outcomeMean· Baseline, Day 60, Day 120, Day 180
Change of Total Protoporphyrin IX Level in Participants
| Group | Value (mcg/dL) | Spread |
|---|---|---|
| Afamelanotide | -471.3 | 505.4 |
| Placebo | -628.5 | 837.5 |
Secondary outcomeNumber· Daily for 6 months
Number of Participants With Phototoxic Reactions With Likert Severity Scores ≥ 4 and ≥ 7
| Group | Value (Count of Participants) |
|---|---|
| Afamelanotide | 26 |
| Placebo | 24 |
Secondary outcomeMedian· Daily for 6 months
Number of Phototoxic Reactions Experienced by Participants
| Group | Value (Episodes) |
|---|---|
| Afamelanotide | 2.0 |
| Placebo | 1.0 |
Secondary outcomeMean· Daily for 6 months.
Total Severity of Phototoxic Reactions Experienced by Participants Over the Entire Study
| Group | Value (units on a scale) | Spread |
|---|---|---|
| Afamelanotide | 27.9 | 43.1 |
| Placebo | 37.2 | 102.6 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| AfamelanotideAfamelanotide: One 16mg subcutaneous implant every 2 months for 6 months. (3 implants in total) | – | 2 / 39 | 31 / 39 |
| PlaceboPlacebo: One placebo subcutaneous implant every 2 months for 6 months. (3 implants in total) | – | 2 / 38 | 29 / 38 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 18 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
* Male or female subjects with characteristic photosensitivity of EPP symptoms and positive diagnosis of EPP confirmed by laboratory result of elevated total protoporphyrin IX.
* Aged 18 years old and above (inclusive).
* Able to understand and sign the written Informed Consent Form.
* Willing to take precautions to prevent pregnancy until completion of the study (Day 180).
Exclusion Criteria:
* Any allergy to afamelanotide or the polymer contained in the implant or to lidocaine or other local anesthetic 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 other photodermatosis such as PLE, DLE or solar urticaria.
* 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 6 months).
* Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history, allergic to local anesthetics, faints when given injections or giving blood).
* 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, including drugs that cause photosensitivity or skin pigmentation within 60 days prior to the screening visit.
* Female who is pregnant (confirmed by positive serum β-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).
PepBank is a research reference. Talk to a doctor before you use any peptide.