Trial Outcomes & Findings for Afamelanotide as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer Sodium (NCT NCT04425746)
NCT ID: NCT04425746
Last Updated: 2021-10-01
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
From Day 04 to Day 90
Results posted on
2021-10-01
Participant Flow
Participant milestones
| Measure |
Afamelanotide
Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication, and the results of evaluation of phototoxicity.
|
Placebo
Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
7
|
|
Overall Study
COMPLETED
|
9
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Afamelanotide as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer Sodium
Baseline characteristics by cohort
| Measure |
Afamelanotide
n=9 Participants
Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication, and the results of evaluation of phototoxicity.
|
Placebo
n=7 Participants
Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
63.0 Years
STANDARD_DEVIATION 9.3 • n=99 Participants
|
72.6 Years
STANDARD_DEVIATION 8.8 • n=107 Participants
|
67.2 Years
STANDARD_DEVIATION 10.0 • n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: From Day 04 to Day 90Population: The planned primary efficacy endpoint was the number of days for patients to be free of phototoxic reactions following phototesting. This was recorded by patients through diaries, however these diaries were not completed appropriately, thus data could not be collected. Thus, the efficacy analysis was performed using the secondary efficacy endpoint, change in quality of life as measured by SF-36.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Day 0 to Day 20, Day 60 and Day 90Quality of life, as measured by the Short Form-36 Health survey (SF-36) (quality of life questionnaire). The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability.
Outcome measures
| Measure |
Afamelanotide
n=7 Participants
Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Afamelanotide
|
Placebo
n=5 Participants
Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Placebo
|
|---|---|---|
|
Change in Quality of Life
Day 20 - Day 0 (Physical functioning)
|
-5.7 Scores on a scale
Interval -35.0 to 0.0
|
8.0 Scores on a scale
Interval -15.0 to 30.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Role Physical)
|
-14.6 Scores on a scale
Interval -56.3 to 6.3
|
0.0 Scores on a scale
Interval -18.8 to 50.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Bodily Pain)
|
-1.4 Scores on a scale
Interval -28.0 to 78.0
|
-11.6 Scores on a scale
Interval -68.0 to 59.0
|
|
Change in Quality of Life
Day 20 - Day 0 (General Health)
|
2.0 Scores on a scale
Interval -25.0 to 60.0
|
-27.6 Scores on a scale
Interval -43.0 to -13.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Vitality)
|
-1.5 Scores on a scale
Interval -18.8 to 12.5
|
-7.9 Scores on a scale
Interval -18.8 to 0.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Social functioning)
|
1.2 Scores on a scale
Interval -50.0 to 50.0
|
7.5 Scores on a scale
Interval -37.5 to 37.5
|
|
Change in Quality of Life
Day 20 - Day 0 (Role - Emotional)
|
-7.1 Scores on a scale
Interval -50.0 to 0.0
|
10.0 Scores on a scale
Interval -25.0 to 50.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Mental Health)
|
1.9 Scores on a scale
Interval -20.0 to 35.0
|
3.0 Scores on a scale
Interval -10.0 to 15.0
|
|
Change in Quality of Life
Day 20 - Day 0 (Physical Component Summary)
|
-2.6 Scores on a scale
Interval -13.1 to 2.7
|
-4.8 Scores on a scale
Interval -18.4 to 10.6
|
|
Change in Quality of Life
Day 20 - Day 0 (Mental Component Summary)
|
0.3 Scores on a scale
Interval -12.1 to 10.4
|
2.7 Scores on a scale
Interval -6.1 to 10.3
|
|
Change in Quality of Life
Day 60 - Day 0 (Physical Functioning)
|
-13.6 Scores on a scale
Interval -80.0 to 0.0
|
-10.0 Scores on a scale
Interval -40.0 to 20.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Role- Physical)
|
-8.9 Scores on a scale
Interval -56.3 to 12.5
|
-13.8 Scores on a scale
Interval -43.8 to 31.3
|
|
Change in Quality of Life
Day 60 - Day 0 (Bodily Pain)
|
2.0 Scores on a scale
Interval -28.0 to 52.0
|
-21.4 Scores on a scale
Interval -68.0 to 19.0
|
|
Change in Quality of Life
Day 60 - Day 0 (General Health)
|
-4.2 Scores on a scale
Interval -32.0 to 18.0
|
-33.0 Scores on a scale
Interval -47.0 to -20.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Vitality)
|
5.4 Scores on a scale
Interval -6.3 to 12.5
|
-5.4 Scores on a scale
Interval -25.0 to 12.5
|
|
Change in Quality of Life
Day 60 - Day 0 (Social Functioning)
|
0.0 Scores on a scale
Interval -50.0 to 37.5
|
-17.5 Scores on a scale
Interval -50.0 to 25.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Role- Emotional)
|
6.0 Scores on a scale
Interval -50.0 to 91.7
|
-10.0 Scores on a scale
Interval -50.0 to 25.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Mental Health)
|
2.9 Scores on a scale
Interval -10.0 to 10.0
|
-2.0 Scores on a scale
Interval -20.0 to 10.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Physical Component Summary)
|
-4.7 Scores on a scale
Interval -23.7 to 7.7
|
-9.4 Scores on a scale
Interval -18.4 to 4.0
|
|
Change in Quality of Life
Day 60 - Day 0 (Mental Component Summary)
|
4.3 Scores on a scale
Interval -8.6 to 34.2
|
-2.5 Scores on a scale
Interval -17.6 to 4.8
|
|
Change in Quality of Life
Day 90 - Day 0 (Physical Functioning)
|
-13.6 Scores on a scale
Interval -80.0 to 5.0
|
-5.0 Scores on a scale
Interval -40.0 to 30.0
|
|
Change in Quality of Life
Day 90 - Day 0 (Role- Physical)
|
0.0 Scores on a scale
Interval -12.5 to 6.3
|
-3.8 Scores on a scale
Interval -37.5 to 31.3
|
|
Change in Quality of Life
Day 90 - Day 0 (Bodily pain)
|
-2.7 Scores on a scale
Interval -16.0 to 19.0
|
-13.8 Scores on a scale
Interval -69.0 to 19.0
|
|
Change in Quality of Life
Day 90 - Day 0 (General Health)
|
-4.1 Scores on a scale
Interval -27.0 to 15.0
|
-32.0 Scores on a scale
Interval -60.0 to 13.0
|
|
Change in Quality of Life
Day 90 - Day 0 (Social Functioning)
|
0.0 Scores on a scale
Interval -37.5 to 37.5
|
-12.5 Scores on a scale
Interval -62.5 to 25.0
|
|
Change in Quality of Life
Day 90 - Day 0 (Role- Emotional)
|
0.0 Scores on a scale
Interval -50.0 to 91.7
|
-11.7 Scores on a scale
Interval -75.0 to 33.3
|
|
Change in Quality of Life
Day 90 - Day 0 (Mental Health)
|
-2.1 Scores on a scale
Interval -10.0 to 10.0
|
-9.0 Scores on a scale
Interval -40.0 to 15.0
|
|
Change in Quality of Life
Day 90 - Day 0 (Physical Component Summary)
|
-3.0 Scores on a scale
Interval -23.7 to 8.2
|
-5.1 Scores on a scale
Interval -14.7 to 6.4
|
|
Change in Quality of Life
Day 90 - Day 0 (Mental Component Summary)
|
0.7 Scores on a scale
Interval -14.3 to 34.2
|
-6.3 Scores on a scale
Interval -30.2 to 7.3
|
Adverse Events
Afamelanotide
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Afamelanotide
n=9 participants at risk
Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Afamelanotide
|
Placebo
n=7 participants at risk
Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Placebo
|
|---|---|---|
|
Gastrointestinal disorders
Haematemesis
|
0.00%
0/9
None of the serious adverse events were deemed to be related to study drug
|
14.3%
1/7
None of the serious adverse events were deemed to be related to study drug
|
|
Gastrointestinal disorders
Oesophageal stenosis
|
11.1%
1/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Injury, poisoning and procedural complications
Overdose of Previscan (Fluindione)
|
11.1%
1/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Injury, poisoning and procedural complications
Drug toxicity (morphine)
|
11.1%
1/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Infections and infestations
Subdiaphragmatic abscess
|
0.00%
0/9
None of the serious adverse events were deemed to be related to study drug
|
14.3%
1/7
None of the serious adverse events were deemed to be related to study drug
|
|
Vascular disorders
Thrombosis
|
0.00%
0/9
None of the serious adverse events were deemed to be related to study drug
|
14.3%
1/7
None of the serious adverse events were deemed to be related to study drug
|
Other adverse events
| Measure |
Afamelanotide
n=9 participants at risk
Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Afamelanotide
|
Placebo
n=7 participants at risk
Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity.
Placebo
|
|---|---|---|
|
Gastrointestinal disorders
Dyspepsia
|
11.1%
1/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Gastrointestinal disorders
Nausea
|
22.2%
2/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Nervous system disorders
Headache
|
33.3%
3/9
None of the serious adverse events were deemed to be related to study drug
|
0.00%
0/7
None of the serious adverse events were deemed to be related to study drug
|
|
Skin and subcutaneous tissue disorders
Erythema
|
11.1%
1/9
None of the serious adverse events were deemed to be related to study drug
|
14.3%
1/7
None of the serious adverse events were deemed to be related to study drug
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place