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Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration

NCT02174848 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2020-08-25

Study results available
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Summary

Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months, are eligible to enroll.

Conditions

  • Pantothenate Kinase-Associated Neurodegeneration

Interventions

DRUG

Deferiprone oral solution

Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day

Sponsors & Collaborators

  • ApoPharma

    lead INDUSTRY

Principal Investigators

  • Elliott Vichinsky, MD · UCSF Benioff Children's Hospital Oakland

  • Thomas Klopstock, MD · Klinikum der Universität München

  • Nardo Nardocci, MD · Foundation Neurological Institute C. Besta

  • Patrick Chinnery, MD · Newcastle University Institute of Human Genetics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-06-30
Primary Completion
2018-03-16
Completion
2018-03-16

Countries

  • United States
  • Germany
  • Italy
  • United Kingdom

Study Locations

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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 NCT02174848 on ClinicalTrials.gov