Pantothenate kinase-associated neurodegeneration (PKAN)
Conditions
Interventions
Product Name: deferiprone 80 mg/mL oral solution
Pharmaceutical Form: Oral solution
INN or Proposed INN: DEFERIPRONE
CAS Number: 30652-11-0
Concentration unit: mg/ml milligram(s)/millilitre
Concentrat
Sponsors
ApoPharma Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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; To evaluate global improvement over time in patients with PKAN treated with deferiprone.;Primary end point(s): 1. Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product. 2. Serious adverse events (SAEs): Frequency, severity, time to onset, duration, and relatedness to study product. 3. Number of discontinuations due to AEs. 4. Hematology assessments. 5. Blood chemistry assessments. 6. ECG assessments.;Timepoint(s) of evaluation of this end point: Safety assessments will be conducted at the following time points: Hematology: Weekly up to Visit 4 (End of Study) or early termination. Blood chemistry: Visits 1, 2, 3, and 4 or early termination. ECG: Visits 1 and 4 or early termination. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. 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. 2. Proportion of patients with improved or unchanged BAD scale total score between baseline and Visit 4 (responder analysis). 3. 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. 4. Change in score on the Patient Global Impression of Improvement (PGI-I) from baseline to Visit 4. 5. Proportion of patients showing an improvement on PGI-I at Visit 4 (responder analysis). The time points for these efficacy endpoints are defined as follows: (1) For patients who received deferiprone in the earlier study, the baseline visit of that study will be treated as the baseline visit of TIRCON2012V1-EXT as well. Thus, Visit 1 of the extension study (Week 0) will be Year 1.5, and Visit 4 (Week 78) will be Year 3. (2) For patients who received placebo in the earlier study, Visit 1 of the extension study (Week 0) will be the baseline visit. Thus, Visit 4 (Week 78) will be Year 1.5. Patients who received placebo in initial study: For this group only, for each measure, the changes seen between the start and completion of study TIRCON2012V1 (i.e., following 18 months on placebo) will be compared against the changes seen between the start and completion of study TIRCON2012V1-EXT (i.e., following 18 months on deferiprone), as follows: 1. Comparison of change in BAD total score from baseline to completion of the initial study vs. change in BAD total score from baseline to completion of the extension study. 2. Comparison of the proportion of patients with improved or unchanged BAD scale total score between baseline and completion of the initial study vs. the proportion with improved or unchanged BAD scale total score from b | — |
Countries
Germany, Italy, United Kingdom, United States
Contacts
Public ContactJohn Connelly
ApoPharma Inc.
Outcome results
None listed