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

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ClinicalTrials.gov Identifier: NCT04578496
Recruitment Status : Completed
First Posted : October 8, 2020
Results First Posted : November 2, 2020
Last Update Posted : November 19, 2020
Sponsor:
Information provided by (Responsible Party):
Clinuvel Pharmaceuticals Limited

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

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Study Type : Interventional  (Clinical Trial)
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 : July 11, 2011
Actual Primary Completion Date : February 27, 2014
Actual Study Completion Date : February 27, 2014


Arm Intervention/treatment
Experimental: Afamelanotide Drug: Afamelanotide



Primary Outcome Measures :
  1. Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). [ Time Frame: 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) [ Time Frame: 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04578496


Sponsors and Collaborators
Clinuvel Pharmaceuticals Limited
Investigators
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Study Director: Pilar Bilbao Clinuvel Pharmaceuticals Limited
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Responsible Party: Clinuvel Pharmaceuticals Limited
ClinicalTrials.gov Identifier: NCT04578496    
Other Study ID Numbers: CUV037
First Posted: October 8, 2020    Key Record Dates
Results First Posted: November 2, 2020
Last Update Posted: November 19, 2020
Last Verified: November 2020
Additional relevant MeSH terms:
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Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias
Metabolic Diseases
Afamelanotide
Dermatologic Agents