|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
EUCTR |
|
Last refreshed on:
|
19 March 2012 |
|
Main ID: |
EUCTR2008-003059-56-IT |
|
Date of registration:
|
18/07/2008 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND
|
|
Scientific title:
|
Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND |
|
Date of first enrolment:
|
29/07/2008 |
|
Target sample size:
|
|
|
Recruitment status: |
Not Recruiting |
|
URL:
|
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-003059-56 |
|
Study type:
|
Interventional clinical trial of medicinal product |
|
Study design:
|
Controlled: no
Randomised: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
Italy
| | | | | | | |
|
Contacts
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
| |
|
Key inclusion & exclusion criteria
|
Inclusion criteria:
Written IC;
Clinical diagnosis of NBIA (neurodegeneration with brain iron accumulation);
Presence of ?Eye of the tiger? sign on brain MRI;
Genetic confirmation of PKAN diagnosis, with presence of mutation in the PANK2 gene;
Age 6 to 40 years;
Effective contraception (i.e. hormonal or IUD) during the study (women in childbearing age);
No conditions known to be contraindications to the use of Deferiprone (history of recurrent episodes of neutropenia; history of agranulocytosis).
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
Neurological disorders other than PKAN, or major co-morbidities (e.g. psychiatric disease other than OCD, overt heart, kidney or lung failure, overt malignancy, HIV infection).
Known haemathological disorders (presence of any disorders associated with neutropenia [ANC < 1.5 x 109/L] or thrombocytopenia [platelet count <50 x 109/L) during the 12 months prior to screening.
Treatment with medicinal products known to be associated with neutropenia or agranulocytosis during the three months prior to screening.
Any condition preventing from brain MRI assessment (i.e pace-maker, metal prostheses or other metallic devices). Patients with intrathecal infusion pump can be included in the study but will undergo the following procedures before the radiological examination: the pump is turned off, then it is empted, secured to the patient?s abdomen with a cotton belt and drug infusion suspended for the entire duration of MRI exposure (Medtronic Inc.);
Female subjects who are pregnant or lactating;
Participation in a clinical trial with experimental drugs during the three months prior to screening.
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
|
|
Health Condition(s) or Problem(s) studied
|
PKAN
MedDRA version: 9.1
Level: HLT
Classification code 10042259
Term: Structural brain disorders NEC
|
|
Intervention(s)
|
Trade Name: FERRIPROX*100CPR RIV 500MG
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Deferiprone
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 25-
|
|
Primary Outcome(s)
|
|
Secondary Objective: Secondary efficacy endpoints are changes in the following clinical scales: Burke-Fahn -Marsden Dystonia Rating Scales (BFMDRS) (Burke et al, 1985) and health-related quality of life (assessed with the 36 item Short Form questionnaire [SF-36] in patients aged 14 year or older (Ware 1992; Apolone et al, 1998), and with the Child Health Questionnaire Parent Form (CHQ-PF50) in 6 -13 year old children (Landgraf et al, 1996) and will be assessed every two months.
|
|
Main Objective: The primary efficacy outcome will be a reduction of iron concentration in the exposed patients at six months, assessed by measuring the iron concentration in both globus pallidus on MRI.
|
|
Primary end point(s): The predefined primary outcome criterion is the effect of Deferiprone treatment on MRI-determined iron concentration in both globus pallidus (mean R 2* changes at six months).
|
|
Source(s) of Monetary Support
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|