Trial Outcomes & Findings for A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) (NCT NCT04578496)
NCT ID: NCT04578496
Last Updated: 2020-11-19
Results Overview
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.
COMPLETED
PHASE3
16 participants
Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
2020-11-19
Participant Flow
Participant milestones
| Measure |
Afamelanotide
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Baseline characteristics by cohort
| Measure |
Afamelanotide
n=16 Participants
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)Population: Intent-to-treat population
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.
Outcome measures
| Measure |
Afamelanotide
n=13 Participants
|
|---|---|
|
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Day 60
|
27.8 EPP-QoL score
Interval -56.0 to 75.0
|
|
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Day 120
|
59.7 EPP-QoL score
Interval 11.0 to 85.0
|
|
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Day 180 or ET
|
56.9 EPP-QoL score
Interval 8.0 to 67.0
|
PRIMARY outcome
Timeframe: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)Population: Intent-to-treat population
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.
Outcome measures
| Measure |
Afamelanotide
n=13 Participants
|
|---|---|
|
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Day 60
|
-5 DLQI score
Interval -17.0 to 1.0
|
|
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Day 120
|
-4.5 DLQI score
Interval -17.0 to 0.0
|
|
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Day 180 or ET
|
-5 DLQI score
Interval -17.0 to 0.0
|
Adverse Events
Afamelanotide
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Afamelanotide
n=16 participants at risk
|
|---|---|
|
Nervous system disorders
Headache
|
6.2%
1/16 • Number of events 1
The adverse event reported below is an adverse event assessed to be related to the study drug by the investigator.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place