Primary outcomeMedian· Daily for 6 months
Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0).
| Group | Value (hours) |
|---|---|
| Afamelanotide | 69.4 |
| Placebo | 40.8 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataPhase III Confirmatory Study in Erythropoietic Protoporphyria
NCTNCT01605136 (opens ClinicalTrials.gov in a new tab)Other idCUV039
Overview
- Status
- Completed
- Phase
- Phase 3
- Enrollment
- 93 participants (actual)
- Sponsor
- Clinuvel Pharmaceuticals Limited · Industry
- Design
- Randomized · Quadruple masking
- Countries
1 country
United States
- Start
- May 2012
- Primary completion
- Jul 2013
- Completion
- Jul 2013
- First posted
- 24 May 2012 (estimated)
- Last update posted
- 19 Sept 2019
- Conditions
- Erythropoietic Protoporphyria
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
201220132014
- Ph 3
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). Between 75 and 100 eligible patients will be enrolled. Patients 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 The number and severity of phototoxic reactions, the type and duration of sun exposure, treatment-emergent adverse events and the use of concomitant medication will be recorded by patients in study diaries between Days 0 and 180. Quality of life will be measured using the DLQI and EPP-QoL at Days 0, 60, 120 and 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events. A subset of patients will be photoprovoked on the lower back and dorsal surface of the hand and the minimal symptom dose (MSD) will be determined on Days 0, 30, 60, 90 and 120.
Interventions
- AfamelanotideDrug
- PlaceboDrug
Arms2
Experimental
Afamelanotide
One 16mg subcutaneous implant every 2 months for 6 months.
Placebo comparator
Placebo
One placebo subcutaneous implant every 2 months for 6 months.
Primary outcomes1
Outcomes the sponsor listed as primary.
Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0).
Time frame · Daily for 6 months
Registry description
The amount of direct sunlight exposure between 10:00 and 18:00 hours on days when no pain was experienced (e.g.11-point Likert pain score of 0). Time was recorded in a patient diary 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)
Combined Sun Exposure and Phototoxic Pain
Time frame · Daily for 6 months
Registry description
Time in direct sunlight exposure between 10:00 and 18:00 hours on days when no or mild pain was experienced (Likert scores of 0 to 3). 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.
Sun Exposure
Time frame · Daily for 6 months
Registry description
Duration of direct sunlight exposure between 10:00 and 18:00 hours during the study.
Quality of Life Score
Time frame · Day 60, Day 120, and Day 180 or early termination.
Registry description
The Quality of life of participant is measured using DLQI and EPP QoL. The Dermatology Life Quality Index (DLQI) is a simple practical measure for routine clinical use. The DLQI ranges from 0 (no impact on life) to 30 (significant impact on life) . The Erthropoietic protoporphyria quality of life measure (EPP-QoL) scores range from 0 (worst imaginable QoL) to 100 (best possible QoL).
Photoprovocation
Time frame · Day 0, Day 30, Day 60, Day 90 and Day 120.
Registry description
A subset of subjects was photoprovoked on the dorsal surface of the hand (predilection place) and lower back and the minimum symptom dose (MSD) determined on Days 0, 30, 60, 90 and 120. The amount of radiation required to provoke the first clinical symptom was recorded.
Maximum Severity of Phototoxic Reaction Experienced by Participants
Time frame · Daily for 6 months
Registry description
The phototoxicity - phototoxic pain secondary endpoint has been divided into two secondary outcome measures. 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.
Total Number Phototoxic Reactions Experienced by Participants
Time frame · Daily for 6 months
Registry description
The phototoxicity - phototoxic pain secondary endpoint has been divided into two secondary outcome measures. The number of episodes was the endpoint. 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.
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
Combined Sun Exposure and Phototoxic Pain
| Group | Value (hours) |
|---|---|
| Afamelanotide | 80.0 |
| Placebo | 51.0 |
Secondary outcomeMedian· Daily for 6 months
Sun Exposure
| Group | Value (hours) |
|---|---|
| Afamelanotide | 83.50 |
| Placebo | 65.25 |
Secondary outcomeMedian· Day 60, Day 120, and Day 180 or early termination.
Quality of Life Score
| Group | Value (units on a scale) |
|---|---|
| Afamelanotide | 10.0 |
| Placebo | 11.0 |
Secondary outcomeMedian· Day 0, Day 30, Day 60, Day 90 and Day 120.
Photoprovocation
| Group | Value (J/cm^2) |
|---|---|
| Afamelanotide | 48.9 |
| Placebo | 21.0 |
Secondary outcomeMedian· Daily for 6 months
Maximum Severity of Phototoxic Reaction Experienced by Participants
| Group | Value (score on a scale) |
|---|---|
| Afamelanotide | 4.0 |
| Placebo | 5.0 |
Secondary outcomeMedian· Daily for 6 months
Total Number Phototoxic Reactions Experienced by Participants
| Group | Value (episodes) |
|---|---|
| Afamelanotide | 4 |
| Placebo | 6 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| AfamelanotideOne 16mg subcutaneous implant every 2 months for 6 months. Afamelanotide: One 16mg subcutaneous implant every 2 months for 6 months. | – | 3 / 48 | 45 / 48 |
| PlaceboOne placebo subcutaneous implant every 2 months for 6 months. Placebo: One placebo subcutaneous implant every 2 months for 6 months | – | 2 / 45 | 39 / 45 |
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
Studies1
Studies that cite this trial.
Studies with results
1 study on this page
Indexing hint: Randomized controlled trial · 2015
6 results
Afamelanotide for Erythropoietic Protoporphyria.
Langendonk et al. 2015The New England journal of medicinecited by 203
PMID 26132941PMC4780255DOI 10.1056/nejmoa1411481PUB-26132941
Rights unknown6 results
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 symptoms of EPP phototoxicity and a biochemically-confirmed diagnosis of EPP.
* 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 polymorphic light eruption, actinic prurigo, discoid lupus erythematosus, chronic actinic dermatitis 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.
* 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).
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