An international trial of deferiprone in patients with iron storage brain disorders

Phase 1

• Conditions: Pantothenate Kinase-Associated Neurodegeneration (PKAN); MedDRA version: 16.0 Level: PT Classification code 10053643 Term: Neurodegenerative disorder System Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2012-000845-11-GB
• Lead Sponsor: ApoPharma Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-09-19
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 90

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 non-DBS 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 study-related activities are undertaken;

8.Ability and willingness to adhere to the protocol including appointments and evaluation schedule.

Are the trial subjects under 18? yes
Number of subjects for this age range: 72
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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 half-lives (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 co-operation.

13.Baclofen pump placement less than two months prior to the beginning of the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified