Trial · NCT01605136
Phase III Confirmatory Study in Erythropoietic Protoporphyria
NCT01605136, Phase 3, Completed, Clinuvel Pharmaceuticals Limited; results first posted 2019-08-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase III Confirmatory Study in Erythropoietic Protoporphyria. Trials, record NCT01605136. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01605136
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase III Confirmatory Study in Erythropoietic Protoporphyria
- NCT number
- NCT01605136
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 93 participants, actual
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Afamelanotide, Placebo
- First posted
- 2012-05-24
- Last updated
- 2019-09-19
- Results first posted
- 2019-08-26
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). | Daily for 6 months | 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 | Combined Sun Exposure and Phototoxic Pain | Daily for 6 months | 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. |
| Secondary | Sun Exposure | Daily for 6 months | Duration of direct sunlight exposure between 10:00 and 18:00 hours during the study. |
| Secondary | Quality of Life Score | Day 60, Day 120, and Day 180 or early termination. | 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). |
| Secondary | Photoprovocation | Day 0, Day 30, Day 60, Day 90 and Day 120. | 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. |
| Secondary | Maximum Severity of Phototoxic Reaction Experienced by Participants | Daily for 6 months | 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. |
| Secondary | Total Number Phototoxic Reactions Experienced by Participants | Daily for 6 months | 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured 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). | Daily for 6 months | Number of subjects (ITT population, from Patient Diary Card) | hours | MEDIAN |
| SECONDARY | Combined Sun Exposure and Phototoxic Pain | Daily for 6 months | Number of subjects (ITT population, from Patient Diary Card) | hours | MEDIAN |
| SECONDARY | Sun Exposure | Daily for 6 months | Number of subjects (ITT population, from Patient Diary Card) | hours | MEDIAN |
| SECONDARY | Quality of Life Score | Day 60, Day 120, and Day 180 or early termination. | The number of participants analyzed differs from the overall number of participants analyzed because the data was either incomplete and/or missing. Only data from those who completed the Quality of Life assessments are included at each time point. | units on a scale | MEDIAN |
| SECONDARY | Photoprovocation | Day 0, Day 30, Day 60, Day 90 and Day 120. | J/cm^2 | MEDIAN | |
| SECONDARY | Maximum Severity of Phototoxic Reaction Experienced by Participants | Daily for 6 months | score on a scale | MEDIAN | |
| SECONDARY | Total Number Phototoxic Reactions Experienced by Participants | Daily for 6 months | episodes | MEDIAN |
Source ClinicalTrials.gov
Results by group46
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). | Afamelanotide | 69.4 | Full Range 0 to 650.50 | hours | |
| Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). | Placebo | 40.8 | Full Range 0.25 to 224.00 | hours | |
| Combined Sun Exposure and Phototoxic Pain | Afamelanotide | 80.0 | Full Range 0.5 to 825.0 | hours | |
| Combined Sun Exposure and Phototoxic Pain | Placebo | 51.0 | Full Range 1.25 to 251.0 | hours | |
| Sun Exposure | Afamelanotide | 83.50 | Full Range 0.50 to 825.00 | hours | |
| Sun Exposure | Placebo | 65.25 | Full Range 3.50 to 278.50 | hours | |
| Quality of Life Score | Afamelanotide | DLQI - Day 0 (baseline) | 10.0 | Full Range 0 to 26 | units on a scale |
| Quality of Life Score | Placebo | DLQI - Day 0 (baseline) | 11.0 | Full Range 0 to 22 | units on a scale |
| Quality of Life Score | Afamelanotide | DLQI - Day 60 | 2.0 | Full Range 0 to 21 | units on a scale |
| Quality of Life Score | Placebo | DLQI - Day 60 | 4.0 | Full Range 0 to 21 | units on a scale |
1 to 10 of 46Source ClinicalTrials.gov
See all 46 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Study Period | STARTED | Afamelanotide | 48 | |
| Study Period | STARTED | Placebo | 45 | |
| Study Period | COMPLETED | Afamelanotide | 45 | |
| Study Period | COMPLETED | Placebo | 42 | |
| Study Period | NOT COMPLETED | Afamelanotide | 3 | |
| Study Period | NOT COMPLETED | Placebo | 3 | |
| Safety Follow-Up Period | STARTED | Afamelanotide | 45 | |
| Safety Follow-Up Period | STARTED | Placebo | 42 | |
| Safety Follow-Up Period | COMPLETED | Afamelanotide | 44 | |
| Safety Follow-Up Period | COMPLETED | Placebo | 40 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events46
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nausea | Gastrointestinal disorders | Afamelanotide | 9 | 48 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 8 | 45 | |
| other | Pain | General disorders | Afamelanotide | 4 | 48 | |
| other | Pain | General disorders | Placebo | 4 | 45 | |
| other | Nasopharyngitis | Infections and infestations | Afamelanotide | 6 | 48 | |
| other | Nasopharyngitis | Infections and infestations | Placebo | 10 | 45 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Afamelanotide | 5 | 48 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Placebo | 2 | 45 | |
| other | Headache | Nervous system disorders | Afamelanotide | 19 | 48 | |
| other | Headache | Nervous system disorders | Placebo | 13 | 45 |
1 to 10 of 46Source ClinicalTrials.gov
See all 46 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Afamelanotide | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Afamelanotide | 45 | 48 |
| Total, other adverse events | Placebo | 39 | 45 |
| Total, serious adverse events | Afamelanotide | 3 | 48 |
| Total, serious adverse events | Placebo | 2 | 45 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26132941 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Afamelanotide vs Placebo: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01605136
GET https://conductscience.org/api/trials/v1/NCT01605136/results?page=2