A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP)

Phase 3

Completed

• Conditions: EPP; Protoporphyria; 10027424

• Registration Number: NL-OMON31710
• Lead Sponsor: Clinuvel Pharmaceuticals Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 13 May 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 25

Inclusion Criteria

-Male or female subjects with a diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes)

Exclusion Criteria

-Allergy to CUV1647 or the polymer contained in the implant or 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 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-Jaegher 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 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).

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>•Number and severity of phototoxic reactions</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>•Melanin density (measured by spectrophotometry)<br /><br>•Duration of sunlight exposure, as recorded in patient diary<br /><br>•Quality of life measured with SF36 questionnaire<br /><br>•*Time taken to develop provoked symptoms* following phototesting (in a subset<br /><br>of patients only)<br /><br>•Treatment-emergent adverse events (coded as MedDRA Preferred Terms)<br /><br>•Changes in hematology, serum chemistry and urinalysis measurements from<br /><br>Screening to Study Days 1, 61, 121, 181, 241 and 301.</p><br>