Pantothenate Kinase-Associated NeurodegenerationNCT02635841
Compassionate Use of Deferiprone in Patients With PKAN
A study testing Deferiprone in people with Pantothenate Kinase-Associated Neurodegeneration, run by Chiesi Canada Corp.
Trial summary
Patients with pantothenate kinase-associated neurodegeneration (PKAN) who have completed the ApoPharma-sponsored study TIRCON2012V1-EXT and who wish to continue to take deferiprone will be offered the opportunity to receive it on a compassionate basis. Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Pantothenate Kinase-Associated Neurodegeneration
- What is being tested?
- Deferiprone
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 7 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Completed study TIRCON2012V1-EXT
- Able to adhere to program appointments and evaluation schedules
- Females of childbearing potential must have a negative pregnancy test result at the start of the program, unless the treating physician determines there is no reasonable risk of pregnancy because of significant incapacity. If applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the program and for 30 days following the last dose of deferiprone, OR participate in a non-heterosexual lifestyle, OR have a male sexual partner who has been sterilized, OR be abstinent during the program and for at least 30 days after its completion.
- Fertile sexually active heterosexual males must agree to use an effective method of contraception during the program and for at least 30 days after its completion
- Patients and/or their authorized legal representatives must provide signed and dated written informed consent, and minors must additionally sign an assent form as per local regulatory requirements.
Who cannot join
- Patients will be excluded from taking part in the compassionate use program if the treating physician determines the presence of any medical, psychological, or psychiatric condition which in his/her opinion would cause participation in the program to be unwise
About this study
Official title: The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration
Patients with pantothenate kinase-associated neurodegeneration (PKAN) who have completed the ApoPharma-sponsored study TIRCON2012V1-EXT and who wish to continue to take deferiprone will be offered the opportunity to receive it on a compassionate basis. Patients will be followed locally by their own neurologist or other appropriate specialist.
Read the detailed description
Patients with PKAN who have completed both the randomized, blinded, placebo-controlled trial TIRCON2012V1 and its follow-up, the single-arm open-label extension study TIRCON2012V1-EXT, may enroll in this compassionate-use/expanded access program in order to continue receiving the iron chelator deferiprone. Participants in this program will be followed locally by their own neurologist or other appropriate specialist. Patients will take deferiprone oral solution 80 mg/mL at the same dose they were prescribed in the TIRCON2012V1-EXT clinical trial. Safety monitoring of the patients will be performed. At a minimum, the program will continue until the analysis of the currently blinded TIRCON2012V1 trial is completed (approximately Q4 2016), at which time it will be re-evaluated.
- Being tested
- Deferiprone
- 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.
DeferiproneDrug
Also called Ferriprox
Ferriprox (deferiprone) 100 mg/mL oral solution
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Chiesi Canada Corp
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02635841
- Sponsor's protocol ID
- TIRCON2012V1-COMP
- Status last verified
- Nov 2022
- First submitted
- 17 Dec 2015
- Last updated
- 14 Nov 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration
- ClinicalTrials.gov ID
- NCT02635841
- Acronym / sponsor ID
- TIRCON2012V1-COMP
- Keywords
- Pantothenate Kinase-Associated Neurodegeneration, PKAN, Neurodegeneration with Brain Iron Accumulation, NBIA, Deferiprone, Ferriprox
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Chiesi Canada CorpIndustry (pharma, biotech, devices)
- First posted
- 21 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with pantothenate kinase-associated neurodegeneration (PKAN) who have completed the ApoPharma-sponsored study TIRCON2012V1-EXT and who wish to continue to take deferiprone will be offered the opportunity to receive it on a compassionate basis. Patients will be followed locally by their own neurologist or other appropriate specialist.
Detailed description
Patients with PKAN who have completed both the randomized, blinded, placebo-controlled trial TIRCON2012V1 and its follow-up, the single-arm open-label extension study TIRCON2012V1-EXT, may enroll in this compassionate-use/expanded access program in order to continue receiving the iron chelator deferiprone. Participants in this program will be followed locally by their own neurologist or other appropriate specialist. Patients will take deferiprone oral solution 80 mg/mL at the same dose they were prescribed in the TIRCON2012V1-EXT clinical trial. Safety monitoring of the patients will be performed. At a minimum, the program will continue until the analysis of the currently blinded TIRCON2012V1 trial is completed (approximately Q4 2016), at which time it will be re-evaluated.
Study design
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Completed study TIRCON2012V1-EXT
- Able to adhere to program appointments and evaluation schedules
- Females of childbearing potential must have a negative pregnancy test result at the start of the program, unless the treating physician determines there is no reasonable risk of pregnancy because of significant incapacity. If applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the program and for 30 days following the last dose of deferiprone, OR participate in a non-heterosexual lifestyle, OR have a male sexual partner who has been sterilized, OR be abstinent during the program and for at least 30 days after its completion.
- Fertile sexually active heterosexual males must agree to use an effective method of contraception during the program and for at least 30 days after its completion
- Patients and/or their authorized legal representatives must provide signed and dated written informed consent, and minors must additionally sign an assent form as per local regulatory requirements.
Key exclusion criteria (1 listed)
- Patients will be excluded from taking part in the compassionate use program if the treating physician determines the presence of any medical, psychological, or psychiatric condition which in his/her opinion would cause participation in the program to be unwise
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 |
|---|---|---|
| DeferiproneAlso: FerriproxFerriprox (deferiprone) 100 mg/mL oral solution | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2022.
Related trials for Pantothenate Kinase-Associated Neurodegeneration
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Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN)
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Deferiprone in people with Pantothenate Kinase-Associated Neurodegeneration, run by Chiesi Canada Corp.
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 5 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 14 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
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 14 Nov 2022.