Status:

COMPLETED

Multicentre Phase III Erythropoietic Protoporphyria Study

Lead Sponsor:

Clinuvel Pharmaceuticals Limited

Conditions:

Erythropoietic Protoporphyria

Eligibility:

All Genders

18-70 years

Phase:

PHASE3

Brief Summary

This was a phase III, multicentre, randomised, double-blind, placebo-controlled study, to evaluate the safety and efficacy of subcutaneous bioresorbable afamelanotide implants in patients with Erythro...

Detailed Description

Afamelanotide is a man-made drug being studied for use as a preventative medication for Erythropoietic Protoporphyria (EPP) sufferers. It is a synthetically produced analogue of human alpha melanocyte...

Eligibility Criteria

Inclusion

  • Male or female patients with a diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) of sufficient severity that they have requested treatment to alleviate their symptoms.
  • Aged 18-70 years.
  • Written informed consent prior to the performance of any study-specific procedure.

Exclusion

  • Any allergy to afamelanotide or the polymer contained in the implant or to lignocaine or other local anaesthetic used during the administration of 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 other photodermatosis such as PLE, DLE or solar urticaria.
  • Diagnosed with HIV/AIDS or hepatitis.
  • 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).
  • History of disorders of the gastrointestinal, hepatic, renal, cardiovascular, respiratory, endocrine (including diabetes, Cushing's syndrome, Addison's disease, Peutz-Jeagher syndrome), neurological (including seizures), haematological (especially anaemia of less than 10 g/100 mL) or systemic disease judged to be clinically significant by the Investigator.
  • Major medical or psychiatric illness
  • Patient assessed as not suitable for the study in the opinion of the investigator (e.g. noncompliance history allergic to local anaesthetics, faints when given injections or giving blood).
  • Female who was 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 clinical trial of an investigational agent within 30 days prior to the screening visit.
  • Use of regular medications as specified in protocol Section 5.4 Prior and Concomitant Therapy.
  • Any factors that may affect skin reflectance measurements.

Key Trial Info

Start Date :

May 1 2007

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 9 2009

Estimated Enrollment :

100 Patients enrolled

Trial Details

Trial ID

NCT04053270

Start Date

May 1 2007

End Date

December 9 2009

Last Update

October 10 2019

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