Trial · NCT04578496
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
NCT04578496, Phase 3, Completed, Clinuvel Pharmaceuticals Limited; results first posted 2020-11-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP). Trials, record NCT04578496. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04578496
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
- NCT number
- NCT04578496
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 16 participants, actual
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Afamelanotide
- First posted
- 2020-10-08
- Last updated
- 2020-11-19
- Results first posted
- 2020-11-02
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life. |
| Primary | Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | Intent-to-treat population | EPP-QoL score | MEDIAN |
| PRIMARY | Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable) | Intent-to-treat population | DLQI score | MEDIAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Afamelanotide | Day 60 | 27.8 | Full Range -56 to 75 | EPP-QoL score |
| Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Afamelanotide | Day 120 | 59.7 | Full Range 11 to 85 | EPP-QoL score |
| Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). | Afamelanotide | Day 180 or ET | 56.9 | Full Range 8 to 67 | EPP-QoL score |
| Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Afamelanotide | Day 60 | -5 | Full Range -17 to 1 | DLQI score |
| Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Afamelanotide | Day 120 | -4.5 | Full Range -17 to 0 | DLQI score |
| Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI) | Afamelanotide | Day 180 or ET | -5 | Full Range -17 to 0 | DLQI score |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Afamelanotide | 16 | |
| Overall Study | COMPLETED | Afamelanotide | 12 | |
| Overall Study | NOT COMPLETED | Afamelanotide | 4 |
Source ClinicalTrials.gov
Adverse events1
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 | Headache | Nervous system disorders | Afamelanotide | 1 | 16 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Afamelanotide | ||
| Total, other adverse events | Afamelanotide | 1 | 16 |
| Total, serious adverse events | Afamelanotide | 0 | 16 |
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT04578496
GET https://conductscience.org/api/trials/v1/NCT04578496/registered