Condition category
Nervous System Diseases
Date applied
Date assigned
Last edited
Retrospectively registered
Overall trial status
Recruitment status
No longer recruiting
Publication status

Plain English Summary

Not provided at time of registration

Trial website

Contact information



Primary contact

Mrs joanne Morrison


Contact details

Institute of Health and Society
4th Floor William Leech Building
Framlington Place
Newcastle Upon Tyne
United Kingdom

Additional identifiers

EudraCT number

2012-000845-11 number


Protocol/serial number


Study information

Scientific title

TIRCON: A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN)



Study hypothesis

1. To evaluate the change in severity of dystonia (BAD scale) in patients with PKAN treated with deferiprone for 18 months compared to placebo.
2. To evaluate the patient’s global impression of condition’s improvement in patients treated with deferiprone for 18 months compared to placebo (PGI-I).

Ethics approval


Study design

Randomised; Interventional; Design type: Treatment

Primary study design


Secondary study design

Randomised controlled trial

Trial setting


Trial type


Patient information sheet

Not available in web format, please use the contact details to request a patient information sheet


Topic: Medicines for Children Research Network; Subtopic: All Diagnoses; Disease: All Diseases


Main Intervention, Haematology
UPDRS (Unified Parkinson's Disease Rating Scale)
PK Sample collection (in a subset of patients)
Genetic Sample (only taken in patients who experience neutropenia)

Intervention type



Phase III

Drug names

Primary outcome measure

Change in the BarryAlbrightDystonia Scale (BAD) total score from baseline to month 18 in patients

Secondary outcome measures

Not provided at time of registration

Overall trial start date


Overall trial end date


Reason abandoned (if study stopped)


Participant inclusion criteria

1. Males and females 4 years of age and older at screening visit
2 .Patients must have PKAN, confirmed by genetic testing
3. Patients having a BAD total score > = 3 at the screening visit
4. Patients who have Deep Brain Stimulation (DBS) systems or baclofen pumps in place will be eligible for the study, but they must have had a stable setting for at least 2 months prior to the screening visit and stimulation parameters /pump settings must remain stable for the duration of the trial. Enrollment of nonDBS patients will be given priority in order to ensure the majority can undergo imaging
5. Potentially sexually active female patients of childbearing potential must have a negative pregnancy test result at Screening Visit (if applicable; in cases where the Investigator determines there is no reasonable risk of pregnancy because of significant incapacity, pregnancy testing will not be performed)
6. Fertile potentially sexually active males must use an effective method of contraception or must confirm partner’s use of effective contraception
7. Informed consent/assent obtained before any studyrelated activities are undertaken
8. Ability and willingness to adhere to the protocol including appointments and evaluation schedule

Participant type


Age group




Target number of participants

Planned Sample Size: 90; UK Sample Size: 8

Participant exclusion criteria

1. Evidence of iron deficiency defined by Fe:TIBC ratio <15%, or serum ferritin < 12 ng/mL
2. Treatment with deferiprone in the past 12 months
3. Previous failure of treatment with deferiprone, or previous discontinuation of treatment with deferiprone due to adverse events
4. Evidence of abnormal liver or renal function (serum liver enzyme level(s) > 3 times upper limit of normal at screening) or abnormal creatinine levels at screening visit
5. Disorders associated with neutropenia (absolute neutrophil count (ANC) < 1.5 x 109/L) or thrombocytopenia (platelet count < 50 x 109/L) in the 12 months preceding the initiation of the study medication. Exception: for patients whose neutropenia was attributed by the treating physician to episodes of infection or to drugs associated with a decline in the neutrophil count and in whom ANC has fully recovered at the screening visit
6. Pregnant, breastfeeding, or planning to become pregnant during the study
7. Initiation or discontinuation of treatment with baclofen, trihexyphenidyl, clonazepam, tizanidine within 30 days prior to baseline; and initiation or discontinuation of treatment with tetrabenazine within 90 days prior to baseline
8. Treatment with an investigational drug within 30 days or 5 halflives
(whichever is longer) preceding the baseline
9. Currently taking iron chelators
10. Patients who, in the opinion of the physician, represent a high medical or psychological risk
11. History of or active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements
12. Patients and patient's legal representative (if applicable) with a mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
13. Baclofen pump placement less than two months prior to the beginning of the study

Recruitment start date


Recruitment end date



Countries of recruitment

United Kingdom

Trial participating centre

Institute of Health and Society
Newcastle Upon Tyne
United Kingdom

Sponsor information


Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)

Sponsor details

New Victoria Wing
Queen Victoria Road
Newcastle Upon Tyne
United Kingdom

Sponsor type

Hospital/treatment centre



Funder type


Funder name

European Commission

Alternative name(s)

European Union, EC, EU

Funding Body Type

government organisation

Funding Body Subtype

National government


Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Basic results (scientific)

See: (added 09/08/2019)

Publication list

2019 results in: (added 09/08/2019)

Publication citations

Additional files

Editorial Notes

09/08/2019: number, publication reference and basic results added. 09/08/2017: No publications found, verifying study status with principal investigator.