Trial Outcomes & Findings for Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) (NCT NCT00979745)
NCT ID: NCT00979745
Last Updated: 2021-10-12
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
74 participants
Primary outcome timeframe
From baseline to Day 270
Results posted on
2021-10-12
Participant Flow
Participant milestones
| Measure |
Experimental: Afamelanotide
administered afamelanotide implants on Days 0, 60, 120, 180 and 240.
|
Placebo
administered placebo implants on Days 0, 60, 120, 180 and 240.
|
|---|---|---|
|
Overall Study
STARTED
|
38
|
36
|
|
Overall Study
COMPLETED
|
34
|
34
|
|
Overall Study
NOT COMPLETED
|
4
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Baseline characteristics by cohort
| Measure |
Experimental: Afamelanotide
n=38 Participants
administered afamelanotide implants on Days 0, 60, 120, 180 and 240.
|
Placebo
n=36 Participants
administered placebo implants on Days 0, 60, 120, 180 and 240.
|
Total
n=74 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
38.3 years
STANDARD_DEVIATION 13 • n=5 Participants
|
38.6 years
STANDARD_DEVIATION 11.6 • n=7 Participants
|
38.45 years
STANDARD_DEVIATION 12.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From baseline to Day 270Outcome measures
| Measure |
Experimental: Afamelanotide
n=38 Participants
administered afamelanotide implants on Days 0, 60, 120, 180 and 240.
|
Placebo
n=36 Participants
administered placebo implants on Days 0, 60, 120, 180 and 240.
|
|---|---|---|
|
The Duration of Direct Sunlight Exposure Between 10:00 and 15:00 Hours on Days When Patients Did Not Report Phototoxicity-related Pain (Likert Pain Scale Score of 0)
|
6.0 Hours
Interval 0.0 to 192.5
|
0.75 Hours
Interval 0.0 to 34.8
|
SECONDARY outcome
Timeframe: 9 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 9 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 9 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 9 monthsOutcome measures
Outcome data not reported
Adverse Events
Experimental: Afamelanotide
Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Experimental: Afamelanotide
n=38 participants at risk
Afamelanotide implants administered on Days 0, 60, 120, 180 and 240.
|
Placebo
n=36 participants at risk
Placebo implants administered on Days 0, 60, 120, 180 and 240.
|
|---|---|---|
|
General disorders
Implant site discolouration
|
10.5%
4/38
|
0.00%
0/36
|
|
Investigations
Blood urine present
|
5.3%
2/38
|
5.6%
2/36
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/38
|
8.3%
3/36
|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
2/38
|
0.00%
0/36
|
|
Gastrointestinal disorders
Nausea
|
15.8%
6/38
|
8.3%
3/36
|
|
Pregnancy, puerperium and perinatal conditions
Dizziness
|
5.3%
2/38
|
0.00%
0/36
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
7.9%
3/38
|
0.00%
0/36
|
|
Nervous system disorders
Headache
|
18.4%
7/38
|
19.4%
7/36
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place