NCT02174848

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
68

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jun 2014

Typical duration for phase_3

Geographic Reach
4 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2014

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

June 5, 2014

Completed
21 days until next milestone

First Posted

Study publicly available on registry

June 26, 2014

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 16, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 16, 2018

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

July 17, 2019

Completed
Last Updated

August 25, 2020

Status Verified

August 1, 2020

Enrollment Period

3.8 years

First QC Date

June 5, 2014

Results QC Date

May 1, 2019

Last Update Submit

August 12, 2020

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Keywords

Pantothenate Kinase-Associated NeurodegenerationPKANNeurodegeneration with Brain Iron AccumulationNBIADeferiproneFerriprox

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Adverse Events

    Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product.

    18 months

Secondary Outcomes (5)

  • Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study

    Baseline and Month 18 of each study

  • Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies

    Baseline and Month 18 of each study

  • Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies

    Baseline and Month 18 of each study

  • Proportion of Patients With Improved or Unchanged BAD Score

    Month 18 of each study

  • Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies

    Month 18 of each study

Study Arms (1)

Deferiprone

EXPERIMENTAL

All patients will receive deferiprone oral solution.

Drug: Deferiprone oral solution

Interventions

Deferiprone oral solutionDRUG

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

Also known as: DFP
Deferiprone

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Completed study TIRCON2012V1

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Klinikum der Universität München

Munich, 80336, Germany

Location

Foundation Neurological Institute C. Besta

Milan, 20133, Italy

Location

Newcastle University Institute of Human Genetics

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

MeSH Terms

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Interventions

DeferiproneIsoflurophate

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeuroaxonal DystrophiesMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

PyridonesPyridinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsOrganofluorophosphonatesOrganophosphonatesOrganophosphorus CompoundsOrganic Chemicals

Results Point of Contact

Title
Fernando Tricta, MD
Organization
Chiesi Canada Corp.
f.tricta@chiesi.com
416--558-6342

Study Officials

  • Elliott Vichinsky, MD

    UCSF Benioff Children's Hospital Oakland

    PRINCIPAL INVESTIGATOR

  • Thomas Klopstock, MD

    Klinikum der Universität München

    PRINCIPAL INVESTIGATOR

  • Nardo Nardocci, MD

    Foundation Neurological Institute C. Besta

    PRINCIPAL INVESTIGATOR

  • Patrick Chinnery, MD

    Newcastle University Institute of Human Genetics

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: All participants in this study received the same intervention.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 5, 2014

First Posted

June 26, 2014

Study Start

June 1, 2014

Primary Completion

March 16, 2018

Study Completion

March 16, 2018

Last Updated

August 25, 2020

Results First Posted

July 17, 2019

Record last verified: 2020-08

Locations

IT(1)US(1)GB(1)DE(1)