Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 5 of 6 for:    Neurodegeneration with Brain Iron Accumulation (NBIA)

Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration (TIRCON-EXT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02174848
Recruitment Status : Completed
First Posted : June 26, 2014
Results First Posted : July 17, 2019
Last Update Posted : July 17, 2019
Sponsor:
Information provided by (Responsible Party):
ApoPharma

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.

Condition or disease Intervention/treatment Phase
Pantothenate Kinase-Associated Neurodegeneration Drug: Deferiprone oral solution Phase 3

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 68 participants
Intervention Model: Single Group Assignment
Intervention Model Description: All participants in this study received the same intervention.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
Study Start Date : June 2014
Actual Primary Completion Date : March 16, 2018
Actual Study Completion Date : March 16, 2018


Arm Intervention/treatment
Experimental: Deferiprone
All patients will receive deferiprone oral solution.
Drug: Deferiprone oral solution
Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day
Other Name: DFP




Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: 18 months ]
    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.


Secondary Outcome Measures :
  1. Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study [ Time Frame: Baseline and Month 18 of each study ]
    The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of both the initial study (during which one group received placebo and the other received deferiprone) and the extension study (during which both groups received deferiprone).

  2. Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies [ Time Frame: Baseline and Month 18 of each study ]
    The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of each study.

  3. Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies [ Time Frame: Baseline and Month 18 of each study ]
    The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of the study.

  4. Proportion of Patients With Improved or Unchanged BAD Score [ Time Frame: Month 18 of each study ]
    Patients were deemed to be responders if their BAD total score either improved or remained unchanged from baseline, with baseline being the start of each study for the placebo-DFP group and the start of the initial study for the DFP-DFP group

  5. Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies [ Time Frame: Month 18 of each study ]
    The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.

  6. Patient Global Impression of Improvement (PGI-I) Comparison of DFP-DFP Patients Across Studies [ Time Frame: Month 18 of each study ]
    The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.

  7. Proportion of Patients With Improved or Unchanged PGI-I Score [ Time Frame: Month 18 of each study ]
    Patients were deemed to be responders if their PGI-I score at the end of the study indicated that they felt they had either improved or remained unchanged from baseline



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02174848


Locations
Layout table for location information
United States, California
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Germany
Klinikum der Universität München
Munich, Germany, 80336
Italy
Foundation Neurological Institute C. Besta
Milan, Italy, 20133
United Kingdom
Newcastle University Institute of Human Genetics
Newcastle Upon Tyne, United Kingdom, NE1 3BZ
Sponsors and Collaborators
ApoPharma
Investigators
Layout table for investigator information
Principal Investigator: 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
  Study Documents (Full-Text)

Documents provided by ApoPharma:
Statistical Analysis Plan  [PDF] April 9, 2018
Study Protocol  [PDF] August 20, 2014


Layout table for additonal information
Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT02174848     History of Changes
Other Study ID Numbers: TIRCON2012V1-EXT
2012-000845-11 ( EudraCT Number )
First Posted: June 26, 2014    Key Record Dates
Results First Posted: July 17, 2019
Last Update Posted: July 17, 2019
Last Verified: June 2019
Keywords provided by ApoPharma:
Pantothenate Kinase-Associated Neurodegeneration
PKAN
Neurodegeneration with Brain Iron Accumulation
NBIA
Deferiprone
Ferriprox
Additional relevant MeSH terms:
Layout table for MeSH terms
Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
Brain Diseases
Pathologic Processes
Basal Ganglia Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action