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Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

NCT01097044 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2019-10-28

Study results available
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Summary

This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen:

* Group A will be administered afamelanotide implants on Days 0, 60 and 120
* Group B will be administered placebo implants on Days 0, 60 and 120

To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured using the EPP specific questionnaire (EPP-QoL) every 60 days and the DLQI questionnaire every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.

Conditions

Interventions

DRUG

Afamelanotide

One 16mg subcutaneous implant every 2 months for 6 months. (3 implants in total)

DRUG

Placebo

One placebo subcutaneous implant every 2 months for 6 months. (3 implants in total)

Sponsors & Collaborators

  • Clinuvel Pharmaceuticals Limited

    lead INDUSTRY

Principal Investigators

  • Robert Desnick, MD · Mt. Sinai

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-04-30
Primary Completion
2011-04-30
Completion
2011-04-30

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01097044 on ClinicalTrials.gov