Multicentre Phase III Erythropoietic Protoporphyria Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04053270
• Date of registration: 08/08/2019
• Prospective Registration: No
• Primary sponsor: Clinuvel Pharmaceuticals Limited
• Public title: Multicentre Phase III Erythropoietic Protoporphyria Study
• Scientific title: A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutanenous Bioresorbable CUV1647 Implants in Patients With Erythropoietic Protoporphyria (EPP)
• Date of first enrolment: May 2007
• Target sample size: 100
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04053270
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Contacts

• Name: Head of Clinical Development
• Affiliation: CLINUVEL PHARMACEUTICALS LTD

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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 Criteria:

- 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 ß-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.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Erythropoietic Protoporphyria

Intervention(s)

Drug: Afamelanotide

Drug: Placebo

Primary Outcome(s)

Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) [Time Frame: 0-360 days or Early Termination]

The Mean Number of Phototoxic Reactions (Study Efficacy Population) [Time Frame: 0-360 days or Early Termination]

Secondary Outcome(s)

Change in Quality of Life Using SF36 Questionnaire (Mental Component Score) for Study Completers Population [Time Frame: Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination]

Skin Melanin Density (Study Completers Population) [Time Frame: Day0, Day14, Day30, Day60, Day74, Day90, Day120, Day150, Day180, Day210, Day240, Day270, Day300, Day330, Day360 or Early Termination]

Change in Quality of Life Using SF36 Questionnaire (Physical Component Score) for Study Completers Population [Time Frame: Day0, Day60, Day120, Day180, Day240, Day300, Day360 or Early Termination]

Cumulative Number of Days With Sunlight Exposure (Study Efficacy Population) [Time Frame: 0-360 days or Early Termination]

Secondary ID(s)

CUV017

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/10/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04053270