A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

• Sponsor: Clinuvel Pharmaceuticals Limited (Industry)
• Overall Status: Completed
• CT.gov ID: NCT04578496
• Collaborator: (none)
• Enrollment: 16
• Arm: 1
• Actual Duration (Months): 31.6

Study Details

Study Description

Brief Summary

The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients.

Condition or Disease	Intervention/Treatment	Phase
Erythropoietic Protoporphyria	Drug: Afamelanotide	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 16 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
• Actual Study Start Date: Jul 11, 2011
• Actual Primary Completion Date: Feb 27, 2014
• Actual Study Completion Date: Feb 27, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Afamelanotide	Drug: Afamelanotide

Outcome Measures

Primary Outcome Measures

1. 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.

2. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies;

• aged 18-75 years (inclusive);

• provide written informed patient consent prior to the performance of any study-specific procedure.

Exclusion Criteria:

• any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant;

• any allergy to lignocaine or other local anaesthetic to be used during the administration of the study medication;

• EPP patients with significant hepatic involvement;

• 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;

• any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations;

• acute history of drug or alcohol abuse (in the last 12 months);

• female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (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);

• sexually active men with partners of child-bearing potential not using barrier contraception during the trial and for a period of three months thereafter;

• participation in a clinical trial for an investigational agent within 30 days prior to the screening visit;

• prior and concomitant therapy with medications which may interfere with the objectives of the study.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Clinuvel Pharmaceuticals Limited

Investigators

• Study Director: Pilar Bilbao, Clinuvel Pharmaceuticals Limited

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Clinuvel Pharmaceuticals Limited
• ClinicalTrials.gov Identifier: NCT04578496
• Other Study ID Numbers: CUV037
• First Posted: Oct 8, 2020
• Last Update Posted: Nov 19, 2020
• Last Verified: Nov 1, 2020

• Additional relevant MeSH terms: Protoporphyria, Erythropoietic; Afamelanotide

Study Results

Participant Flow

Arm/Group Title	Afamelanotide
Arm/Group Description
Period Title: Overall Study
STARTED	16
COMPLETED	12
NOT COMPLETED	4

Baseline Characteristics

Arm/Group Title	Afamelanotide
Arm/Group Description
Overall Participants	16
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	15 93.8%
>=65 years	1 6.3%
Sex: Female, Male (Count of Participants)
Female	11 68.8%
Male	5 31.3%

Outcome Measures

1. Primary Outcome

Title	Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Description	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.
Time Frame	Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Afamelanotide
Arm/Group Description
Measure Participants	13
Day 60	27.8
Day 120	59.7
Day 180 or ET	56.9

2. Primary Outcome

Title	Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Description	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.
Time Frame	Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Afamelanotide
Arm/Group Description
Measure Participants	13
Day 60	-5
Day 120	-4.5
Day 180 or ET	-5

Adverse Events

Adverse Event Reporting Description	The adverse event reported below is an adverse event assessed to be related to the study drug by the investigator.
Arm/Group Title	Afamelanotide
Arm/Group Description
All Cause Mortality
	Afamelanotide
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Afamelanotide
	Affected / at Risk (%)	# Events
Total	0/16 (0%)
Other (Not Including Serious) Adverse Events
	Afamelanotide
	Affected / at Risk (%)	# Events
Total	1/16 (6.3%)
Nervous system disorders
Headache	1/16 (6.3%)	1

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.