E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving
Dialysis |
|
E.1.1.1 | Medical condition in easily understood language |
Secondary Hyperparathyroidism and Chronic Kidney Disease |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10020708 |
E.1.2 | Term | Hyperparathyroidism secondary |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To characterize the long-term safety and tolerability of cinacalcet in pediatric subjects with CKD receiving dialysis |
|
E.2.2 | Secondary objectives of the trial |
To characterize the long-term effect of cinacalcet in pediatric subjects
receiving dialysis on laboratory parameters associated with chronic kidney disease-mineral bone disease (CKD-MBD) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All Subjects
- Subject’s legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any day 1 study-specific activities/procedures being initiated.
- Completed treatment through week 20 in the parent study, 20130356
- Dry weight ≥ 12.5 kg at day 1
- Dialysate calcium concentration ≥ 2.5 mEq/L at day 1
Subjects Randomized to the 20130356 Standard of Care Arm Only
- iPTH value ≥ 300 pg/mL (week 19 in 20130356)
- Corrected calcium value ≥ 8.8 mg/dL (week 19 in 20130356) |
|
E.4 | Principal exclusion criteria |
General
- Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(s), other than Amgen Study 20130356.
- Other investigational procedures while participating in this study are excluded.
- Malignancy except non-melanoma skin cancers, cervical or breast ductal carcinoma in situ within the last 5 years.
- Subject has known sensitivity to any of the products to be administered during dosing
- Subject likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, electronic patient diary [ediary]) to the best of the subject and investigator’s knowledge
- History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.
- Subject previously has entered this study.
- If sexually active, subject is not willing to use highly effective contraception during treatment and for at least 9 days after the end of treatment
- Subject is pregnant or breast feeding, or planning to become pregnant during the study or within 9 days after the end of treatment
- History of congenital long QT syndrome, second or third degree heart block, ventricular tachyarrythmias, or other conditions associated with prolonged QT interval
At Day 1 Study Visit-All Subjects
- Subject has an ongoing adverse event from study 20130356 that is considered related to IP and:
- Is ≥ CTCAE (v 4.0) grade 3, and/or
- Considered clinically significant in the opinion of the investigator
- Central laboratory values (cCa, iPTH) were not obtained/are not available for week 19 in the 20130356 study
- Corrected QT Interval (QTc) > 500 ms, using Bazett’s formula
- QTc ≥ 450 to ≤ 500 ms, using Bazett’s formula, unless written permission to enroll is provided by the investigator after consultation with a pediatric cardiologist
- Use of grapefruit juice, herbal medications, CYP3A4 inhibitors (eg, erythromycin, clarithromycin, ketoconazole, itraconazole), or CYP2D6 substrates (eg, flecainide, propafenone, metoprolol, desipramine, nortriptyline, clomipramine)
- Use of concomitant medications that may prolong the QTc interval (eg, ondansetron, albuterol)
At Day 1 Study Visit-Subjects Randomized to the 20130356 Cinacalcet Arm Only
- Cinacalcet dose withheld > 1 month |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of treatment emergent adverse events of interest |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From the date of the first dose through 30 days following the last dose of investigational product |
|
E.5.2 | Secondary end point(s) |
- Percent change from day 1 in iPTH to week 11 and week 28
- Serum cCa values at day 1, week 11, and week 28
- Serum phosphorus values at day 1, week 11, and week 28 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- iPTH: from day 1 to week 11 and week 28
- Serum cCa: at day 1, week 11, and week 28
- Serum phosphorus: at day 1, week 11, and week 28 |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Mexico |
New Zealand |
Russian Federation |
Ukraine |
United States |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
The end of study is the primary completion date. This is defined as the time when the last subject is assessed or receives an intervention for the purposes of final collection of data. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 4 |