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

NCT ID: NCT02174848

Last Updated: 2020-08-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 68 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-06-30
• Study Completion Date: 2018-03-16

Brief 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.

Detailed Description

TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 (NCT01741532) are eligible to take part. In the initial study, patients were randomized in a 2:1 ratio to receive 18 months of treatment with either the iron chelator deferiprone or placebo, respectively. In this extension study, all participants will receive deferiprone for 18 months. Thus, depending on which product was received earlier, patients will be on deferiprone for a total of either 1.5 years or 3 years. As in the earlier study, assessments will be carried out every six months to look at the safety of the drug and to see if patients are showing any improvement in dystonia and other symptoms of PKAN.

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Deferiprone

All patients will receive deferiprone oral solution.

Group Type: EXPERIMENTAL

Deferiprone oral solution

Intervention Type: DRUG

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

Interventions

Deferiprone oral solution

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

Intervention Type: DRUG

Other Intervention Names

DFP

Eligibility Criteria

Inclusion Criteria

• Completed study TIRCON2012V1

Exclusion Criteria

• Withdrew from the study TIRCON2012V1 for reasons of safety
• Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study

• Minimum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ApoPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Elliott Vichinsky, MD

Role: PRINCIPAL_INVESTIGATOR

UCSF Benioff Children's Hospital Oakland

Thomas Klopstock, MD

Role: PRINCIPAL_INVESTIGATOR

Klinikum der Universität München

Nardo Nardocci, MD

Role: PRINCIPAL_INVESTIGATOR

Foundation Neurological Institute C. Besta

Patrick Chinnery, MD

Role: PRINCIPAL_INVESTIGATOR

Newcastle University Institute of Human Genetics

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Klinikum der Universität München

Munich, , Germany

Foundation Neurological Institute C. Besta

Milan, , Italy

Newcastle University Institute of Human Genetics

Newcastle upon Tyne, , United Kingdom

Countries

United States; Germany; Italy; United Kingdom

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

2012-000845-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TIRCON2012V1-EXT

Identifier Type: -

Identifier Source: org_study_id