Afamelanotide as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer Sodium

Sponsor
Clinuvel Pharmaceuticals Limited (Industry)
Overall Status
Completed
CT.gov ID
NCT04425746
Collaborator
(none)
16
Enrollment
2
Arms
9.7
Actual Duration (Months)

Study Details

Study Description

Brief Summary

The purpose of this study was to evaluate the safety and efficacy of afamelanotide (previously developed as CUV1647) as adjunctive therapy in patients undergoing photodynamic therapy using porfimer sodium.

Condition or DiseaseIntervention/TreatmentPhase
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
16 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase II, Multicentre, Double-blind, Placebo Controlled, Pilot Study to Evaluate the Safety and Efficacy of CUV1647 (Afamelanotide) as Adjunctive Therapy in Patients Undergoing Photodynamic Therapy (PDT) Utilising Porfimer Sodium
Actual Study Start Date :
Aug 5, 2008
Actual Primary Completion Date :
May 28, 2009
Actual Study Completion Date :
May 28, 2009

Arms and Interventions

ArmIntervention/Treatment
Experimental: 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.

Drug: Afamelanotide

Placebo Comparator: 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.

Drug: Placebo

Outcome Measures

Primary Outcome Measures

  1. The First Day on Which Patients Are Free From Symptoms of Phototoxicity Following Phototesting [From Day 04 to Day 90]

Secondary Outcome Measures

  1. Change in Quality of Life [From Day 0 to Day 20, Day 60 and Day 90]

    Quality 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.

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  • Male or female Caucasian subjects undergoing photodynamic therapy with porfimer sodium;

  • Aged greater than 18 years;

  • Written informed consent prior to the performance of any study-specific procedure.

Exclusion Criteria:
  • Known allergy or hypersensitivity to afamelanotide or the polymer contained in the implant;

  • Non-Caucasian patients;

  • Personal history of melanoma or dysplastic nevus syndrome;

  • Current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions;

  • Female who is pregnant (confirmed by positive serum β-HCG pregnancy test prior to baseline) or lactating;

  • Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, intrauterine device);

  • Participation in a simultaneous clinical trial for another investigational agent or within 30 days prior to the screening visit;

  • Patients unable to give informed consent;

  • Patient needs for concomitant medication with potential photosensitizing effects e.g. antibiotics like tetracyclines.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Clinuvel Pharmaceuticals Limited

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Clinuvel Pharmaceuticals Limited
ClinicalTrials.gov Identifier:
NCT04425746
Other Study ID Numbers:
  • CUV025
First Posted:
Jun 11, 2020
Last Update Posted:
Oct 1, 2021
Last Verified:
Sep 1, 2021
Additional relevant MeSH terms:

Study Results

Participant Flow

Recruitment Details
Pre-assignment Detail
Arm/Group TitleAfamelanotidePlacebo
Arm/Group DescriptionSubjects 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.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.
Period Title: Overall Study
STARTED97
COMPLETED96
NOT COMPLETED01

Baseline Characteristics

Arm/Group TitleAfamelanotidePlaceboTotal
Arm/Group DescriptionSubjects 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.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 of all reporting groups
Overall Participants9716
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]
63.0
(9.3)
72.6
(8.8)
67.2
(10.0)
Sex: Female, Male (Count of Participants)
Female
0
0%
1
14.3%
1
6.3%
Male
9
100%
6
85.7%
15
93.8%

Outcome Measures

1. Primary Outcome
TitleThe First Day on Which Patients Are Free From Symptoms of Phototoxicity Following Phototesting
Description
Time FrameFrom Day 04 to Day 90

Outcome Measure Data

Analysis Population Description
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.
Arm/Group TitleAfamelanotidePlacebo
Arm/Group DescriptionSubjects 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. AfamelanotideSubjects 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
Measure Participants00
2. Secondary Outcome
TitleChange in Quality of Life
DescriptionQuality 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.
Time FrameFrom Day 0 to Day 20, Day 60 and Day 90

Outcome Measure Data

Analysis Population Description
[Not Specified]
Arm/Group TitleAfamelanotidePlacebo
Arm/Group DescriptionSubjects 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.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.
Measure Participants75
Day 20 - Day 0 (Physical functioning)
-5.7
8.0
Day 20 - Day 0 (Role Physical)
-14.6
0.0
Day 20 - Day 0 (Bodily Pain)
-1.4
-11.6
Day 20 - Day 0 (General Health)
2.0
-27.6
Day 20 - Day 0 (Vitality)
-1.5
-7.9
Day 20 - Day 0 (Social functioning)
1.2
7.5
Day 20 - Day 0 (Role - Emotional)
-7.1
10.0
Day 20 - Day 0 (Mental Health)
1.9
3.0
Day 20 - Day 0 (Physical Component Summary)
-2.6
-4.8
Day 20 - Day 0 (Mental Component Summary)
0.3
2.7
Day 60 - Day 0 (Physical Functioning)
-13.6
-10.0
Day 60 - Day 0 (Role- Physical)
-8.9
-13.8
Day 60 - Day 0 (Bodily Pain)
2.0
-21.4
Day 60 - Day 0 (General Health)
-4.2
-33.0
Day 60 - Day 0 (Vitality)
5.4
-5.4
Day 60 - Day 0 (Social Functioning)
0.0
-17.5
Day 60 - Day 0 (Role- Emotional)
6.0
-10.0
Day 60 - Day 0 (Mental Health)
2.9
-2.0
Day 60 - Day 0 (Physical Component Summary)
-4.7
-9.4
Day 60 - Day 0 (Mental Component Summary)
4.3
-2.5
Day 90 - Day 0 (Physical Functioning)
-13.6
-5.0
Day 90 - Day 0 (Role- Physical)
0.0
-3.8
Day 90 - Day 0 (Bodily pain)
-2.7
-13.8
Day 90 - Day 0 (General Health)
-4.1
-32.0
Day 90 - Day 0 (Social Functioning)
0.0
-12.5
Day 90 - Day 0 (Role- Emotional)
0.0
-11.7
Day 90 - Day 0 (Mental Health)
-2.1
-9.0
Day 90 - Day 0 (Physical Component Summary)
-3.0
-5.1
Day 90 - Day 0 (Mental Component Summary)
0.7
-6.3

Adverse Events

Time Frame
Adverse Event Reporting Description None of the serious adverse events were deemed to be related to study drug
Arm/Group TitleAfamelanotidePlacebo
Arm/Group DescriptionSubjects 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. AfamelanotideSubjects 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
All Cause Mortality
AfamelanotidePlacebo
Affected / at Risk (%)# EventsAffected / at Risk (%)# Events
Total/ (NaN) / (NaN)
Serious Adverse Events
AfamelanotidePlacebo
Affected / at Risk (%)# EventsAffected / at Risk (%)# Events
Total2/9 (22.2%) 3/7 (42.9%)
Gastrointestinal disorders
Haematemesis0/9 (0%) 1/7 (14.3%)
Oesophageal stenosis1/9 (11.1%) 0/7 (0%)
Infections and infestations
Subdiaphragmatic abscess0/9 (0%) 1/7 (14.3%)
Injury, poisoning and procedural complications
Overdose of Previscan (Fluindione)1/9 (11.1%) 0/7 (0%)
Drug toxicity (morphine)1/9 (11.1%) 0/7 (0%)
Vascular disorders
Thrombosis0/9 (0%) 1/7 (14.3%)
Other (Not Including Serious) Adverse Events
AfamelanotidePlacebo
Affected / at Risk (%)# EventsAffected / at Risk (%)# Events
Total6/9 (66.7%) 1/7 (14.3%)
Gastrointestinal disorders
Dyspepsia1/9 (11.1%) 0/7 (0%)
Nausea2/9 (22.2%) 0/7 (0%)
Nervous system disorders
Headache3/9 (33.3%) 0/7 (0%)
Skin and subcutaneous tissue disorders
Erythema1/9 (11.1%) 1/7 (14.3%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Name/TitleClinical Operations Manager
OrganizationCLINUVEL PHARMACEUTICALS LTD
Phone
Emailmail@clinuvel.com
Responsible Party:
Clinuvel Pharmaceuticals Limited
ClinicalTrials.gov Identifier:
NCT04425746
Other Study ID Numbers:
  • CUV025
First Posted:
Jun 11, 2020
Last Update Posted:
Oct 1, 2021
Last Verified:
Sep 1, 2021