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

Phase 1

• Conditions: Pantothenate kinase-associated neurodegeneration (PKAN); Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-03-30
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• 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 two months prior to the screening visit and every effort should be made to maintain the stable setting for the duration of the study:
• Enrollment of non-DBS patients will be given priority, to maximize the proportion that can undergo imaging;
5. 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 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

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 clinically significant abnormal creatinine levels at screening visit;
5. Disorders associated with neutropenia (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 the ANC has fully recovered at the screening visit;
6. Pregnant or nursing females, females planning to become pregnant, and females of childbearing potential who are sexually active and are unwilling, or unable, to use an acceptable method of contraception
according to local requirements;
7. Initiation or discontinuation of treatment with baclofen, trihexyphenidyl, clonazepam, tizanidine within 30 days prior to baseline; treatment with botox within 60 days of 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 visit;
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 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 beginningof the study.

Study & Design

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