Trial · NCT04053270
Multicentre Phase III Erythropoietic Protoporphyria Study
NCT04053270, Phase 3, Completed, Clinuvel Pharmaceuticals Limited; results first posted 2019-10-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Multicentre Phase III Erythropoietic Protoporphyria Study. Trials, record NCT04053270. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04053270
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Multicentre Phase III Erythropoietic Protoporphyria Study
- NCT number
- NCT04053270
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 100 participants, actual
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Afamelanotide, Placebo
- First posted
- 2019-08-12
- Last updated
- 2019-10-10
- Results first posted
- 2019-10-10
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | 0-360 days or Early Termination | The cumulative number of days where a phototoxic reaction occurred was recorded in the patient diary. The reported data represent a cumulative total for days of phototoxic reactions. 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 primary analysis population for efficacy was revised, to analyze only participants who reported cumulative total Likert pain scores of at least 26 during the study (0-360 days). This population, which was comprised of 60 patients, is identified as the Efficacy population. Participants with less than 26 Likert pain scores were not included in the analysis. |
| Primary | The Mean Number of Phototoxic Reactions (Study Efficacy Population) | 0-360 days or Early Termination | The mean number of phototoxic reactions that occurred whilst patients were on active compared with placebo implants. 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 primary analysis population for efficacy was revised, to analyze only participants who reported a cumulative total Likert pain score of at least 26 during the study (0-360 days). This population, which was comprised of 60 patients, is identified as the Efficacy population. Participants with less than 26 Likert pain scores were not included in the analysis. |
| Secondary | Cumulative Number of Days With Sunlight Exposure (Study Efficacy Population) | 0-360 days or Early Termination | The number of days with sunlight exposure was recorded in the patient diary. The sunlight exposures were divided into the following categories: none, \< 1 hour, 1 to 3 hours, 3 to 6 hours and \> 6 hours per day. |
| Secondary | Skin Melanin Density (Study Completers Population) | Day0, Day14, Day30, Day60, Day74, Day90, Day120, Day150, Day180, Day210, Day240, Day270, Day300, Day330, Day360 or Early Termination | Changes in melanin density (MD) (measured by spectrophotometry) at each visit by group. Participants had their skin pigmentation measured by a non-invasive quantitative skin chromaticity (reflectance) reading. Reflectance by the skin of light measured at the wavelengths of 400 nm and 420 nm was recorded using a Minolta cm-2500d spectrophotometer at the following skin sites: forehead, left cheek, right inside upper arm, left medial forearm, right side of abdomen (avoiding implant insertion site), left side of sacral region/buttock. Melanin density was determined for each skin site using the method of Dwyer et al 1998. |
| Secondary | Change in Quality of Life Using SF36 Questionnaire (Physical Component Score) for Study Completers Population | Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination | The Summary of SF36 change from Baseline of Physical Component Score (PCS) to the scores during treatment on Days 60, 120, 180, 240, 300 and 360 using the SF36 questionnaire. The SF-36 (The Short Form 36 Health Survey) consists of eight scaled scores, which are the weighted sums of the questions in each section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The higher scores represent better health-related quality-of-life. |
| Secondary | Change in Quality of Life Using SF36 Questionnaire (Mental Component Score) for Study Completers Population | Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination | The Summary of SF36 change from Baseline of Mental Component Score (MCS) to the scores during treatment on Days 60, 120, 180, 240, 300 and 360 using the SF36 questionnaire. The SF-36 (The Short Form 36 Health Survey) consists of eight scaled scores, which are the weighted sums of the questions in each section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The higher scores represent better health-related quality-of-life. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | 0-360 days or Early Termination | Participants who reported a cumulative total Likert pain score of at least 26 during the study (0-360 days), which was comprised of 60 patients, is identified as the Efficacy population. | Days | NUMBER |
| PRIMARY | The Mean Number of Phototoxic Reactions (Study Efficacy Population) | 0-360 days or Early Termination | The result is reported in a per sequence format with the different time points at which the interventions were switched.~Total of 60 Participants: 32 participants in Group A and 28 participants in Group B. | counts of episodes | MEAN |
| SECONDARY | Cumulative Number of Days With Sunlight Exposure (Study Efficacy Population) | 0-360 days or Early Termination | The primary analysis population for efficacy was revised, with an additional criterion of a total cumulative pain score of at least 26, and omitting patients who did not complete the study. This population, which was comprised of 60 patients, is identified as the Efficacy population. | Days | NUMBER |
| SECONDARY | Skin Melanin Density (Study Completers Population) | Day0, Day14, Day30, Day60, Day74, Day90, Day120, Day150, Day180, Day210, Day240, Day270, Day300, Day330, Day360 or Early Termination | The secondary analysis population for efficacy included those subjects who received all required doses of investigational product. This population is identified as the study completers population.~Calculated MD \<0 or \>6 were omitted. | unitless | MEAN |
| SECONDARY | Change in Quality of Life Using SF36 Questionnaire (Physical Component Score) for Study Completers Population | Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination | The secondary analysis population for efficacy included those subjects who received all required doses of investigational product. This population, which was comprised of 93 patients, is identified as the study completers population. | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life Using SF36 Questionnaire (Mental Component Score) for Study Completers Population | Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination | The secondary analysis population for efficacy included those subjects who received all required doses of investigational product. This population, which was comprised of 93 patients, is identified as the study completers population. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group88
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Afamelanotide | Severe Pain | 39 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Placebo | Severe Pain | 45 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Afamelanotide | Moderate Pain | 129 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Placebo | Moderate Pain | 179 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Afamelanotide | Mild Pain | 1126 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Placebo | Mild Pain | 1237 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Afamelanotide | No Pain | 8650 | Days | |
| Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) | Placebo | No Pain | 8484 | Days | |
| The Mean Number of Phototoxic Reactions (Study Efficacy Population) | Group A | Day 0 | 1.44 | Standard Deviation 3.25 | counts of episodes |
| The Mean Number of Phototoxic Reactions (Study Efficacy Population) | Group B | Day 0 | 2.64 | Standard Deviation 5.33 | counts of episodes |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group A | 49 | |
| Overall Study | STARTED | Group B | 51 | |
| Overall Study | COMPLETED | Group A | 47 | |
| Overall Study | COMPLETED | Group B | 46 | |
| Overall Study | NOT COMPLETED | Group A | 2 | |
| Overall Study | NOT COMPLETED | Group B | 5 |
Source ClinicalTrials.gov
Adverse events62
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 | Abdominal pain | Gastrointestinal disorders | Afamelanotide | 9 | 100 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 6 | 100 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Afamelanotide | 7 | 100 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 5 | 100 | |
| other | Diarrhoea | Gastrointestinal disorders | Afamelanotide | 13 | 100 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 13 | 100 | |
| other | Nausea | Gastrointestinal disorders | Afamelanotide | 34 | 100 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 19 | 100 | |
| other | Toothache | Gastrointestinal disorders | Afamelanotide | 4 | 100 | |
| other | Toothache | Gastrointestinal disorders | Placebo | 7 | 100 |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 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 | 94 | 100 |
| Total, other adverse events | Placebo | 88 | 100 |
| Total, serious adverse events | Afamelanotide | 3 | 100 |
| Total, serious adverse events | Placebo | 3 | 100 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 9521433 | RESULT |
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/NCT04053270
GET https://conductscience.org/api/trials/v1/NCT04053270/results?page=2