Pantothenate Kinase-Associated NeurodegenerationPhase 3TIRCON-EXTNCT02174848
Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
A Phase 3 study testing Deferiprone oral solution in people with Pantothenate Kinase-Associated Neurodegeneration, run by ApoPharma at 4 sites in 4 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Pantothenate Kinase-Associated Neurodegeneration
- What is being tested?
- Deferiprone oral solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 68 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Jun 2014to 16 Mar 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed study TIRCON2012V1
Who cannot join
- 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
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
United States1 site
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
United Kingdom1 site
Newcastle University Institute of Human Genetics
Newcastle upon Tyne, NE1 3BZ
About this study
Official title: Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
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.
Read the 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.
- Being tested
- Deferiprone oral solution
- Keywords
- Pantothenate Kinase-Associated Neurodegeneration, PKAN, Neurodegeneration with Brain Iron Accumulation, NBIA, Deferiprone, Ferriprox
- Medical subject headings
- Pantothenate Kinase-Associated Neurodegeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone oral solutionDrug
Also called DFP
Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day
| Study group | Role | What they receive |
|---|---|---|
| Deferiprone | Experimental | All patients will receive deferiprone oral solution. Receives: Drug: Deferiprone oral solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 18 months
Secondary outcomes (5)
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over 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).
Measured: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies
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.
Measured: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies
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.
Measured: Baseline and Month 18 of each study
Proportion of Patients With Improved or Unchanged BAD Score
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
Measured: Month 18 of each study
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies
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.
Measured: Month 18 of each study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2014 (estimated)
- Study startJun 2014
- Primary completion16 Mar 2018
- Study completion16 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pantothenate Kinase-Associated NeurodegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All participants in this study received the same intervention.
- Masking
- None (open label)
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elliott Vichinsky, MD, Principal investigator, UCSF Benioff Children's Hospital OaklandThomas Klopstock, MD, Principal investigator, LMU KlinikumNardo Nardocci, MD, Principal investigator, Foundation Neurological Institute C. BestaPatrick Chinnery, MD, Principal investigator, Newcastle University Institute of Human Genetics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02174848
- Sponsor's protocol ID
- TIRCON2012V1-EXT
- Other IDs
- 2012-000845-11
- Acronym
- TIRCON-EXT
- Status last verified
- Aug 2020
- First submitted
- 5 Jun 2014
- Last updated
- 25 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
- ClinicalTrials.gov ID
- NCT02174848
- Acronym / sponsor ID
- TIRCON-EXT / TIRCON2012V1-EXT
- Keywords
- Pantothenate Kinase-Associated Neurodegeneration, PKAN, Neurodegeneration with Brain Iron Accumulation, NBIA, Deferiprone, Ferriprox
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- Jun 2014
- Primary completion
- 16 Mar 2018 (actual)
- Study completion
- 16 Mar 2018 (actual)
- First posted
- 26 Jun 2014 (estimated)
- Results
- Posted 17 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All participants in this study received the same intervention.
| Arm | Type | Receives |
|---|---|---|
| Deferiprone | Experimental | Drug: Deferiprone oral solutionAll patients will receive deferiprone oral solution. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Completed study TIRCON2012V1
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deferiprone oral solutionAlso: DFPDeferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day | Drug | Deferiprone |
Primary outcome
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.
5 secondary outcomes
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study
Time frame: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies
Time frame: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies
Time frame: Baseline and Month 18 of each study
Proportion of Patients With Improved or Unchanged BAD Score
Time frame: Month 18 of each study
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies
Time frame: Month 18 of each study
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Neurodegenerative Diseases Progression Markers (MARKERS-NDD)
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TIRCON International NBIA Registry
Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elliott Vichinsky, MD principal investigator
UCSF Benioff Children's Hospital Oakland
Thomas Klopstock, MD principal investigator
LMU Klinikum
Nardo Nardocci, MD principal investigator
Foundation Neurological Institute C. Besta
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deferiprone oral solution in people with Pantothenate Kinase-Associated Neurodegeneration, run by ApoPharma at 4 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Germany, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2020.