MedTrace Logo

Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

NCT01741532 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 89

Last updated 2019-07-05

Study results available
· View outcomes & findings →

Summary

A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of 18 months of treatment with deferiprone versus placebo in patients with PKAN.

This investigator-initiated trial was funded by the European Commission's Seventh Framework Programme (FP7/2007-2013, HEALTH-F2-2011, grant agreement No. 277984) to the TIRCON consortium (Treat Iron-Related Childhood-Onset Neurodegeneration) and by the FDA Office of Orphan Products Development (OOPD) (Dr. Elliott Vichinsky).

Conditions

  • Pantothenate Kinase-Associated Neurodegeneration

Interventions

DRUG

Deferiprone oral solution

Deferiprone 80 mg/mL oral solution will be administered twice daily (b.i.d.) for 18 months. An initial dose of 5 mg/kg b.i.d. will be administered for 6 weeks. The dose will then be escalated to 10 mg/kg b.i.d. and finally to 15 mg/kg b.i.d.

DRUG

Placebo

A deferiprone matching placebo oral solution will be given twice daily for 18 months.

Sponsors & Collaborators

  • Food and Drug Administration (FDA)

    collaborator FED

  • ApoPharma

    lead INDUSTRY

Principal Investigators

  • Fernando Tricta, MD · ApoPharma Inc.

  • Thomas Klopstock, MD · Friedrich-Baur-Institute, Department of Neurology, University of Munich Ziemssenstr

  • Elliott Vichinsky, MD · Children's Hospital & Research Center at Oakland Hematology/ Oncology, Pediatric Rehabilitation

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-12-13
Primary Completion
2016-10-21
Completion
2017-01-11

Countries

  • United States
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT01741532 on ClinicalTrials.gov