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); Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2014-001427-79-DE
• Lead Sponsor: ApoPharma Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-03-27
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 90

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: 36
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 54
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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