An 18-month extension of an international trial of deferiprone in patients with iron storage brain disorders

Phase 1

• Conditions: Pantothenate Kinase-Associated Neurodegeneration (PKAN); MedDRA version: 21.1Level: PTClassification code 10053643Term: Neurodegenerative disorderSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2014-001427-79-IT
• Lead Sponsor: APOTEX EUROPE B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2018-03-16
• Study Start: 2021-06-04
• Last Update Posted: 4 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 68

Inclusion Criteria

1. Completed study TIRCON2012V1

2. Sexually active females of childbearing potential, including those who are peri-menopausal (defined as less than 2 years since last menstrual period) must have a negative pregnancy test result at Visit 1 (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). In addition, if applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the study and within 30 days following their last dose of study medication, OR; Participate in a non-heterosexual lifestyle, OR; Have a male sexual partner who has been sterilized (supporting evidence required).

Approved methods of contraception will consist of the following or must follow local requirements: Oral contraceptive used in conjunction with condom, diaphragm, or spermicide; Hormonal implant used in conjunction with condom, diaphragm, or spermicide; Injectable contraceptive used in conjunction with condom, diaphragm, or spermicide; Diaphragm or condom used with spermicide.

If a hormonal contraception is used, it should have a Pearl index <1%.

Female patients who meet any of the following criteria are not of childbearing potential and therefore do not need to practice contraception: Post-menopausal (last menstrual period > 2 years ago); Had a tubal ligation (supporting evidence required); Had a hysterectomy or oophorectomy (supporting evidence required).

3. Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication.

4. Patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and patients must be able to adhere to study restrictions, appointments, and evaluation schedules. Patients who are minors must sign an assent form as per local regulatory requirements.
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 52
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Withdrew from the study TIRCON2012V1 for reasons of safety.

2. Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study.

3. Presence of any medical, psychological, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.

4. Pregnant, breastfeeding, or planning to become pregnant during the study period.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety and tolerability of deferiprone in patients with PKAN.;Secondary Objective: • To evaluate the change in severity of dystonia over time in patients with PKAN treated with deferiprone. <br>• To evaluate global improvement over time in patients with PKAN treated with deferiprone. ;Primary end point(s): •Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product<br>•Serious adverse events (SAEs): Frequency, severity, time to onset, duration, and relatedness to study product <br>•Number of discontinuations due to AEs<br>;Timepoint(s) of evaluation of this end point: 18 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): •Change in the Barry-Albright Dystonia Scale (BAD) total score from baseline (defined as prior to the start of deferiprone therapy) to Visit 4, as assessed by central evaluation of videotapes; Proportion of patients with improved or unchanged BAD scale total score between baseline and Visit 4 (responder analysis); •Change from baseline to Visit 4 in BAD scale score per body region (eyes, mouth, neck, trunk, and each upper and lower extremity), as assessed by central evaluation of videotapes; Change in score on the Patient Global Impression of Improvement (PGI-I) from Visit 1 to Visit 4; Proportion of patients showing an improvement on PGI-I at Visit 4 (responder analysis);Timepoint(s) of evaluation of this end point: Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visit 4