A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN)
| ISRCTN | ISRCTN72904618 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN72904618 |
| EudraCT/CTIS number | 2012-000845-11 |
| ClinicalTrials.gov number | NCT01741532 |
| Secondary identifying numbers | 15679 |
- Submission date
- 08/11/2013
- Registration date
- 08/11/2013
- Last edited
- 09/08/2019
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Mrs joanne Morrison
Scientific
Scientific
Institute of Health and Society
4th Floor William Leech Building
Framlington Place
Newcastle Upon Tyne
NE2 4HH
United Kingdom
| joanne.morrison@ncl.ac.uk |
Study information
| Study design | Randomised; Interventional; Design type: Treatment |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | TIRCON: A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) |
| Study acronym | TIRCON |
| Study objectives | 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 patients global impression of conditions improvement in patients treated with deferiprone for 18 months compared to placebo (PGI-I). |
| Ethics approval(s) | 13/YH/0171 |
| Health condition(s) or problem(s) studied | 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 |
| 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 study start date | 01/07/2013 |
| Completion date | 01/07/2015 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 4 Years |
| Sex | Both |
| Target number of participants | Planned Sample Size: 90; UK Sample Size: 8 |
| Key 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 partners 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 |
| Key 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 |
| Date of first enrolment | 01/07/2013 |
| Date of final enrolment | 01/07/2015 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
Institute of Health and Society
Newcastle Upon Tyne
NE2 4HH
United Kingdom
NE2 4HH
United Kingdom
Sponsor information
Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)
Hospital/treatment centre
Hospital/treatment centre
New Victoria Wing
Queen Victoria Road
Newcastle Upon Tyne
NE1 4LP
England
United Kingdom
"ROR" |
https://ror.org/05p40t847 |
|---|
Funders
Funder type
Government
European Commission
Government organisation / National government
Government organisation / National government
- Alternative name(s)
- European Union, Comisión Europea, Europäische Kommission, EU-Kommissionen, Euroopa Komisjoni, Ευρωπαϊκής Επιτροπής, Европейската комисия, Evropské komise, Commission européenne, Choimisiúin Eorpaigh, Europskoj komisiji, Commissione europea, La Commissione europea, Eiropas Komisiju, Europos Komisijos, Európai Bizottságról, Europese Commissie, Komisja Europejska, Comissão Europeia, Comisia Europeană, Európskej komisii, Evropski komisiji, Euroopan komission, Europeiska kommissionen, EC, EU
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | 09/08/2019 | No | No | ||
| Results article | results | 01/07/2019 | 09/08/2019 | Yes | No |
| HRA research summary | 28/06/2023 | No | No |
Editorial Notes
09/08/2019: ClinicalTrials.gov number, publication reference and basic results added.
09/08/2017: No publications found, verifying study status with principal investigator.
