Condition category
Nervous System Diseases
Date applied
08/11/2013
Date assigned
08/11/2013
Last edited
11/11/2013
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Mrs joanne Morrison

ORCID ID

Contact details

Institute of Health and Society 4th Floor William Leech Building
Framlington Place
Newcastle Upon Tyne
NE2 4HH
United Kingdom
joanne.morrison@ncl.ac.uk

Additional identifiers

EudraCT number

2012-000845-11

ClinicalTrials.gov number

Protocol/serial number

15679

Study information

Scientific title

Acronym

TIRCON

Study hypothesis

To evaluate the change in severity of dystonia (BAD scale) in patients with PKAN treated with deferiprone for 18 months compared to placebo.
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

13/YH/0171

Study design

Randomised; Interventional; Design type: Treatment

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

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

Intervention

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

Other

Phase

Phase III

Drug names

Primary outcome measures

1. 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

01/07/2013

Overall trial end date

01/07/2015

Reason abandoned

Eligibility

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

Patient

Age group

Child

Gender

Both

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

01/07/2013

Recruitment end date

01/07/2015

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Institute of Health and Society 4th Floor William Leech Building
Newcastle Upon Tyne
NE2 4HH
United Kingdom

Sponsor information

Organisation

Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)

Sponsor details

New Victoria Wing
Queen Victoria Road
Newcastle Upon Tyne
NE1 4LP
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

European Commission

Alternative name(s)

EC

Funding Body Type

government organisation

Funding Body Subtype

Federal/National Government

Location

Belgium

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes