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Phase III Confirmatory Study in Erythropoietic Protoporphyria

NCT01605136 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 93

Last updated 2019-09-19

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). Between 75 and 100 eligible patients will be enrolled. Patients 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

The number and severity of phototoxic reactions, the type and duration of sun exposure, treatment-emergent adverse events and the use of concomitant medication will be recorded by patients in study diaries between Days 0 and 180. Quality of life will be measured using the DLQI and EPP-QoL at Days 0, 60, 120 and 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.

A subset of patients will be photoprovoked on the lower back and dorsal surface of the hand and the minimal symptom dose (MSD) will be determined on Days 0, 30, 60, 90 and 120.

Conditions

Interventions

DRUG

Afamelanotide

One 16mg subcutaneous implant every 2 months for 6 months.

DRUG

Placebo

One placebo subcutaneous implant every 2 months for 6 months

Sponsors & Collaborators

  • Clinuvel Pharmaceuticals Limited

    lead INDUSTRY

Principal Investigators

  • Robert Desnick, MD · Mt. Sinai Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-05-31
Primary Completion
2013-07-31
Completion
2013-07-31

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 NCT01605136 on ClinicalTrials.gov