Trial · NCT01097044
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
NCT01097044, Phase 2, Completed, Clinuvel Pharmaceuticals Limited; results first posted 2019-09-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP). Trials, record NCT01097044. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01097044
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
- NCT number
- NCT01097044
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 77 participants, actual
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Afamelanotide, Placebo
- First posted
- 2010-04-01
- Last updated
- 2019-10-28
- Results first posted
- 2019-09-17
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days | Daily for 6 months | 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 | Maximum Severity of Phototoxic Reaction Experienced by Participants | Daily for 6 months | 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 | Quality of Life Measured by Participant Completed Questionnaire | Day 0, Day 60, Day 120, Day 180 | 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. |
| Secondary | Change of Total Protoporphyrin IX Level in Participants | Baseline, Day 60, Day 120, Day 180 | 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. |
| Secondary | Number of Participants With Phototoxic Reactions With Likert Severity Scores ≥ 4 and ≥ 7 | Daily for 6 months | 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. |
| Secondary | Number of Phototoxic Reactions Experienced by Participants | Daily for 6 months | 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. |
| Secondary | Total Severity of Phototoxic Reactions Experienced by Participants Over the Entire Study | Daily for 6 months. | 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. |
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 | Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days | Daily for 6 months | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | Hours | MEDIAN |
| SECONDARY | Maximum Severity of Phototoxic Reaction Experienced by Participants | Daily for 6 months | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | score on a scale | MEDIAN |
| SECONDARY | Quality of Life Measured by Participant Completed Questionnaire | Day 0, Day 60, Day 120, Day 180 | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | units on a scale | MEDIAN |
| SECONDARY | Change of Total Protoporphyrin IX Level in Participants | Baseline, Day 60, Day 120, Day 180 | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | mcg/dL | MEAN |
| SECONDARY | Number of Participants With Phototoxic Reactions With Likert Severity Scores ≥ 4 and ≥ 7 | Daily for 6 months | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | Count of Participants | NUMBER |
| SECONDARY | Number of Phototoxic Reactions Experienced by Participants | Daily for 6 months | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | Episodes | MEDIAN |
| SECONDARY | Total Severity of Phototoxic Reactions Experienced by Participants Over the Entire Study | Daily for 6 months. | One active participant and one placebo participant withdrew after Baseline assessment and no data was provided for the analysis of this endpoint.~The overall number of participants analyzed differs from the total number of study participants because the data was either incomplete and/or missing. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days | Afamelanotide | 8.25 | Full Range 0 to 48.3 | Hours | |
| Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days | Placebo | 0.75 | Full Range 0 to 71.3 | Hours | |
| Maximum Severity of Phototoxic Reaction Experienced by Participants | Afamelanotide | 5.0 | Full Range 0 to 8 | score on a scale | |
| Maximum Severity of Phototoxic Reaction Experienced by Participants | Placebo | 5.0 | Full Range 0 to 10 | score on a scale | |
| Quality of Life Measured by Participant Completed Questionnaire | Afamelanotide | Day 0 | 34.7 | Full Range 3 to 67 | units on a scale |
| Quality of Life Measured by Participant Completed Questionnaire | Placebo | Day 0 | 22.2 | Full Range 3 to 78 | units on a scale |
| Quality of Life Measured by Participant Completed Questionnaire | Afamelanotide | Day 60 | 73.6 | Full Range 17 to 100 | units on a scale |
| Quality of Life Measured by Participant Completed Questionnaire | Placebo | Day 60 | 44.4 | Full Range 6 to 94 | units on a scale |
| Quality of Life Measured by Participant Completed Questionnaire | Afamelanotide | Day 120 | 84.7 | Full Range 6 to 100 | units on a scale |
| Quality of Life Measured by Participant Completed Questionnaire | Placebo | Day 120 | 55.6 | Full Range 6 to 100 | units on a scale |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Afamelanotide | 39 | |
| Overall Study | STARTED | Placebo | 38 | |
| Overall Study | COMPLETED | Afamelanotide | 34 | |
| Overall Study | COMPLETED | Placebo | 33 | |
| Overall Study | NOT COMPLETED | Afamelanotide | 5 | |
| Overall Study | NOT COMPLETED | Placebo | 5 |
Source ClinicalTrials.gov
Adverse events28
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 | 8 | 39 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 2 | 38 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Afamelanotide | 3 | 39 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Placebo | 2 | 38 | |
| other | Fatigue | General disorders | Afamelanotide | 3 | 39 | |
| other | Fatigue | General disorders | Placebo | 1 | 38 | |
| other | Implant Site Pain | General disorders | Afamelanotide | 1 | 39 | |
| other | Implant Site Pain | General disorders | Placebo | 5 | 38 | |
| other | Pyrexia | General disorders | Afamelanotide | 3 | 39 | |
| other | Pyrexia | General disorders | Placebo | 1 | 38 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 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 | 31 | 39 |
| Total, other adverse events | Placebo | 29 | 38 |
| Total, serious adverse events | Afamelanotide | 2 | 39 |
| Total, serious adverse events | Placebo | 2 | 38 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01097044
GET https://conductscience.org/api/trials/v1/NCT01097044/results?page=2